But regardless of the uncertainty surrounding its value, the PRV program has had some impact on decision-making around neglected disease product development in the private sector, as seen in recent statements by companies such as PaxVax. Investors, CEOs, and other industry executives are familiar with the program, but there has been no in-depth analysis to better understand and document the perceptions of these stakeholders, including their estimates of the PRV’s value. To address this evidence gap, BVGH recently conducted a survey of companies with active drug or vaccine programs in one of the 16 PRV-eligible neglected tropical diseases. Our results are fresh off the online journal presses today in PLoS Neglected Tropical Diseases.

Our study focused on understanding three areas: 1) the influence of the PRV in setting priorities within biopharmaceutical companies developing medicines for neglected tropical diseases; 2) the perceived monetary value of the PRV; and 3) barriers toward improving the PRV as an incentive for the development of new neglected tropical disease medicines. We received responses from 12 companies, representing 27 unique neglected tropical disease drug or vaccine programs—7 responses from smaller companies (<60 full time employees, or FTEs), and 5 responses from larger biopharmaceutical companies (>500 FTEs).

Through the survey and follow-up interviews, we found that the majority of respondents did consider the PRV incentive when deciding whether or not to pursue neglected tropical disease R&D programs. In fact, two respondents specifically said the PRV was the controlling factor in the initiation or continuation of their respective neglected tropical disease programs. When compared to other possible motivating factors, however, the PRV ranked lower than other identified incentives, such as potential market value in the developing world or emerging markets. This result was not surprising. It is clear that the PRV is not a panacea, but one of a number of incentives meant to encourage biopharmaceutical companies to engage in neglected disease R&D.

In addition to the impact of the PRV on portfolio prioritization, we also compiled data on the perceived monetary value of the voucher. Responses spanned a wide range, suggesting there is no industry consensus on the value of the PRV. This finding is not surprising since a PRV has not actually been sold, nor has there been a dollar amount attached to a voucher’s use.

The bottom line is that without a better understanding of the actual market for a voucher, a PRV’s value is uncertain and difficult to assess. This was clear in our follow-up interviews with survey respondents. The respondents underscored, first and foremost, the need for a demonstrated sale of a voucher, with the purchase price disclosed. They also cited a need for the FDA to demonstrate support of the program, and a need for revision of implementation rules governing the program. We hope these constructive suggestions will be considered by the FDA and other industry stakeholders. These documented suggestions should also be examined in light of the recently-passed Creating Hope Act legislation, which creates a parallel three-voucher pilot PRV program for rare pediatric diseases.

Despite these uncertainties, we remain convinced that the PRV represents a valuable incentive for neglected disease research. The PRV program will ultimately generate a number of vouchers -- as more PRV-eligible drug candidates are approved -- which will reinforce the utility as well as the value of the PRV program. Those approved drugs will have the potential to make a real difference in the fight against neglected tropical diseases.

The full paper can be found here.

Rianna Stefanakis is Manager, Research and Policy, at BIO Ventures for Global Health

Intrigued by the findings of the March report, we teamed up with the Biotechnology Industry Organization (BIO) to delve deeper into the biotech sector’s contributions to neglected disease product development. The result is a new report, Biotechnology: Bringing Innovation to Neglected Disease Research and Development. What it uncovers is just as intriguing.

As expected, our research reinforces the findings of the March report. Around the world, biotechnology companies are participating in 39% of all projects in development for new drugs, vaccines, and diagnostics for neglected diseases such as malaria, tuberculosis, Chagas disease, dengue fever, and others. Additional data emerge that shed light on just how these companies are participating. For instance, 64% of all products in development by biotechnology companies across the pipeline for neglected diseases involve partnering. The report finds that the biotech sector’s most frequent partner is academia (57% of projects), followed by product development partnerships — unique public-private partnering mechanisms designed to increase industry participation in neglected disease research and development — (52%), government agencies (30%), other biotech companies (16%), and large pharmaceutical companies (7%).

It was when we began to examine the biotech companies themselves that things got interesting. Across 191 neglected disease products, we found 134 unique biotechs participating — a much bigger number than we expected from an industry facing so many barriers in its efforts to innovate. But when we took a step back, we saw that these companies comprise only around 5% of the biotechnology industry globally — a figure made more provocative when considering that 90% of all biotechs focus on health research and development.

To begin to solve the unmet needs of the developing world, we believe that both biotech companies and global health groups must ramp up commitment and involvement. In the report, we give specific recommendations for how biotech companies can increase their commitment and investment in neglected disease research and development through partnering. We also discuss how neglected disease stakeholders from academia, governments, nonprofits, and foundations can engage these innovative biotech companies.

In light of the data stemming from our research, we are more encouraged than ever by the biotech sector’s level of innovation and willingness to take significant financial risks. We can’t help but wonder, though...if just 134 biotechs can accomplish so much, what could 3,000 do?

Robert Schendle is Manager, Knowledge Systems, at BIO Ventures for Global Health.

In Brazil, the active assistance of government in policy making and spending, the growing capability of private and government capabilities, and the general resurgence of the Brazilian economy have contributed to real improvements in global health. According to Carlos Gadelha, Secretary of Science, Technology and Strategic Products in the Ministry of Health, Brazil recently passed the United Kingdom to become the world’s eighth largest pharmaceutical market. Brazil has greatly increased its vaccine production capacity, he noted, and is spending heavily on producing -- along with international partners at local facilities -- many of the needed vaccines. Brazil’s 2011 budget for vaccine purchasing exceeds US$1 billion.

India and China represent even larger opportunities in vaccine development. Dr. G.S. Reddy, Chief General Manager of Indian Immunologicals, a major manufacturer of human and veterinary vaccines, noted that his country continues to grow rapidly both as a seller’s market and as a supplier to the global vaccine market. He noted that increasing prosperity and a rising middle class have raised the level of the private vaccine market significantly, which is important to attract producers for the public market opportunity.

China, with its state-controlled economy and enormous population challenges, has also made real progress in addressing vaccine development. Julie Milstien, of the University of Maryland, observed that China’s version of the FDA has recently been certified by the World Health Organization (WHO) as a functional regulatory authority, meaning that vaccine products approved by that agency are eligible for the WHO prequalification process. That, combined with increases in capacity and capabilities in China, means that China may well emerge as a low-cost supplier of vaccine products at levels which will challenge India’s role as one of the leading emerging market suppliers. This could have an impact not only on supplies but on prices, one of the key topics of the meeting. This ties in with the GAVI Alliance’s increased purchasing of vaccine products manufactured in developing countries, a trend which will only accelerate as capacity and capabilities increase, particularly in China.

BIO Ventures for Global Health recently published a report, Developing New Drugs & Vaccines for Neglected Diseases of the Poor, which indicates the growing importance of developing world participants, even on the research and development (R&D) side. For example, of the R&D projects we track for neglected tropical diseases, roughly 23% of the participants in those projects are headquartered in the developing world (with just over half of those in the BRICS countries). 

Yet, challenges -- significant challenges -- continue to loom large. Reconciling and refining the inter-related roles and responsibilities of governments, NGOs such as the GAVI Alliance and others, industry partners both large and small, and other stakeholders remains complex and sometimes contentious. Even in our meetings, the tension was apparent between the competing values of affordability and access to these critical products, and sustainability in revenue for the producers and developers who take the risk and bear the cost of development.  Several speakers, including Kim Bush, Deputy Director, Industry Partnerships at the Bill & Melinda Gates Foundation, emphasized that innovation not only in science, but in financing the science will be critically important in the coming months and years.

And we agree. We at BIO Ventures for Global Health will continue to analyze and provide data illuminating the progress and challenges in vaccine development so that we can all work together to drive innovation in the developing world.

Don Joseph is the CEO of BIO Ventures for Global Health.

• There are 104 biotechnology companies participating in 41% of all projects. We were not expecting these high numbers given that small companies face larger financial hurdles for participation in neglected disease R&D than larger pharmaceutical companies.
• Although 13 of the 20 large pharmaceutical companies are participating in neglected disease R&D, the majority of products with pharmaceutical company participation are in development by a minority of companies.
• Academic and research institution participation is broad and deep, but often under recognized. This is an area of potential growth as translational research becomes an increased focus of key funders, such as the U.S. National Institutes of Health.
• PDPs are participating in the development of 40% of products in the pipeline. The percentage of projects with no PDP development partner is surprising considering the focus of investment in neglected disease R&D through the PDP model. It’s important to keep in mind, however, the many indirect benefits PDPs provide to the broader product development community that aren’t captured in this analysis – such as development of new animal models, standardized protocols, target product profiles, and clinical trials capacity building for neglected diseases.

So when it comes to global development, success cannot be achieved through developing and investing in projects. Instead, we must develop and invest in people, and we must do this by discovering new solutions and innovating current tools to appeal to people’s interests and harnesses the power of their skills.

This message was delivered by Bradley Broder, executive director of the Kenya Education Fund, and reiterated by experts from all corners of global development at FHI360’s event, Human Development: The 360 degrees Perspective, on Friday. At the event, experts and practitioners with backgrounds in education, the arts, health, and economic development shared insights into how to spark progress throughout the world. Despite their diverse backgrounds, each of the speakers shared stores about the importance of reaching people individually and involving them in the creation of global development programs that then reach larger populations — composed of individuals.

One of those speakers, Patricia Mechael, executive director of the mHealth Alliance, spoke about how mobile phones, which people already use to make personal connections and share stores, can help improve the health and livelihood of people in the developing world. About 64% of mobile phone users live in the developing world. Leveraging mobile health — or mHealth — technology, health care experts can send messages reminding patients to take their medications, show up for appointments, and encourage healthy behaviors in a way that fits into their individual lives.

But mHealth initiatives could do even more to harness the capabilities of mobile technology, Mechael said, by not just collecting and sharing data, but building off of the device — truly innovating — to create point-of-care diagnostics, chronic disease monitoring devices, and more.

To do that, it will take private sector participation, as well as leaders who design mHealth projects not just as pilots, but as long-term initiatives with business plans, scale-up strategies that would help reach hundreds of thousands of leaders, followers, teachers, and connectors, who spread the word and build a healthier world together.

As a newly minted member of the mHealth Alliance, we at BVGH couldn’t agree more. Just as we work to find ways to leverage the expertise and experience of the private sector in the development of new drugs, vaccines, and diagnostics for neglected diseases, we are now working to find ways to bring together experts in mobile technology with mHealth innovators to build new, and improve existing, mHealth programs. By coming together from all perspectives — as individuals and industries — we believe mobile technology can bring health care to ever individuals’ home.

Lindsay Moore is Associate, External Affairs, at BIO Ventures for Global Health.

 We heard this over and over again during yesterday's Global Health Technologies Coalition (GHTC) briefing. The message is clear: millions of lives have been saved across the globe because of American innovation. But global health is more than that: spurring innovation to create new tools that fight against neglected tropical diseases helps Americans travelling abroad and the U.S. military. It also guards Americans against pandemic diseases. And the briefing speakers pointed to additional benefits: investing in research leads to the creation of new American jobs as well as diplomacy advancements. Perhaps most importantly, a healthy global population leads to stable economies, which in turn leads to stable governments, resulting in good partner countries and a more secure American nation. 

The purpose of this briefing was to present GHTC’s 2012 policy report, focusing on the need to create and sustain U.S. policies that spur global health innovation. BIO Ventures for Global Health (BVGH) is a member of the GHTC and has endorsed the report. 

During the briefing, an expert panel of speakers spoke about steps their organizations have taken to continue the U.S legacy of advancing innovation to save lives around the world. Katherine Monahan, Deputy Executive Director of the Global Health Initiative, stressed that one of the Initiative’s core principles is to promote research and innovation and that global health is in the national interest. 

Kevin de Cock, Director of the Center for Global Health at the Centers for Disease Control and Prevention (CDC), echoed that notion and went a step further. At the CDC, he said, global health is indivisible from domestic health. Further strengthening this stance, Colonel Peter Weina, Deputy Commander of the Walter Reed Army Institute of Medicine (WRAIR), said that at WRAIR, soldier health is world health. 

We at BVGH agree. And it’s clear that others do as well. One of the projects highlighted in the GHTC report is WIPO Re:Search, a consortium of pharmaceutical companies and research institutions that have come together to make their intellectual property and know-how available for neglected tropical disease researchers in all regions of the world. By providing a searchable, freely accessible database of available intellectual property assets, information, and resources, WIPO Re:Search members hope to facilitate new partnerships with organizations that conduct research on treatments and diagnostics for neglected tropical diseases, malaria, and tuberculosis. 

We hope that, as the report recommends, the U.S. Government will ramp up its efforts in the global health arena and participate in projects -- as the National Institutes of Health does in WIPO Re:Search -- to help ensure progress on lifesaving tools to save lives around the globe, as well as at home.

Molly Polen is Director of Communications at BIO Ventures for Global Health

Our view at BIO Ventures for Global Health is that rumors of the PRV’s demise are greatly exaggerated. In fact, use of the voucher may yet prove to be an advantage to Novartis for Ilaris, compared to a normal review time. More broadly, FDA has so far played by the rules, which is good news for others developing PRV-eligible products or considering doing so.

The PRV program, created by legislation in 2007, was intended to provide an incentive to the pharmaceutical industry to develop new therapeutics for a specified list of neglected tropical diseases, such as malaria, leishmaniasis, dengue fever and others. Any entity obtaining FDA approval of a new chemical or molecular entity for one of the listed neglected diseases would receive a voucher providing priority (meaning expedited) review of an application for any other drug or biologic, regardless of indication. The effect of this voucher, when used, would be to shorten the FDA review time from the normal ten months or more, to six months (assuming the drug did not qualify for its own accelerated or priority review, in which case the voucher would not be used). This could have the effect of accelerating revenue for the new product by at least four and perhaps many more months, resulting in increased revenues in the tens or even hundreds of millions of dollars for a blockbuster product.

In 2009, Novartis obtained the first and, so far, only PRV issued upon obtaining FDA approval of Coartem (artemether/lumefantrine) for malaria. While Coartem clearly was not the result of the PRV program incentive—it had been on market around the world for several years, but never approved in the US—issuance of the first PRV to Novartis was appropriate under the terms of the legislation. (Pending legislation, which BVGH has helped to craft, and supports, would remove this possibility by restricting PRVs to drugs approved within a relatively short period of time of approvals outside the US.)

To begin, the jury remains out on the actual result in the Ilaris situation. While FDA normally follows the recommendation of its Advisory Committees, several outcomes remain possible. The Advisory Committee strongly endorsed the efficacy of Ilaris for the proposed indication, but opined overall against approval in light of safety risks associated with side effects seen in the new use. According to Novartis’s press release, however, Committee members raised the potential for use in a narrower population of gouty arthritis patients. This suggests one alternative to outright non-approval of the sBLA: allowing Novartis to submit additional data in support of a modified dosing or use in that subpopulation.

Such a decision—which has not yet occurred, and the sBLA remains under review at this writing—could still result in significant accelerated revenue to Novartis if that data were to be required, obtained, submitted, and ultimately approved. Certainly that revenue would not occur as soon (and perhaps not as much) as Novartis would like, but such an outcome remains possible. (We assume that the likelihood of FDA approving the drug on present data, over the recommendation of the Panel, is very small.) In other words, the faster Ilaris review decision resulting from the PRV could still generate accelerated revenue as long as a path to approval remains open and could prove highly valuable.

Drawing any conclusion about the PRV’s value for Ilaris is therefore premature. In fact, as to the implications for the PRV more broadly, Novartis has said that FDA has so far complied with the steps necessary to complete an expedited review. The sBLA was submitted in the first quarter of 2011; the Advisory Committee was convened and issued its findings in late June; and a decision by FDA is expected in the third quarter of 2011. Novartis gave the required year’s advance notice to apply the PRV to Ilaris, and paid the $4.6 million user fee. So far, other than the challenge to Novartis of deciding a year in advance of submission, there seem to have been no bumps in the process. Concerns that FDA would not fulfill its obligations in review of a PRV-related submission therefore do not seem warranted based on this initial experience; of course, FDA must still issue its action letter on time, but the signs point to that occurring as required.

It is important to keep in mind that the PRV program was never designed to produce a different outcome upon FDA review, but simply a faster outcome. One of the inherent risks of the PRV is its use on a drug which ultimately is not approved. (Another, as noted, is the requirement that the PRV be submitted at least a year in advance, in order to allow FDA to focus the resources necessary to provide the expedited review.) The success or value of the PRV as an expediter of the FDA process should not be judged by approval or disapproval, and especially on the basis of one voucher. Even as to Ilaris, the full story has not yet been written. Our surveys and discussions with industry, and our monitoring of the PRV program since its inception, indicate that there are a significant number of PRV-eligible products in various stages of development. In our view, the only PRV issued to date has, so far, done exactly what it was designed to do: expedite the review of a product not otherwise entitled to that priority review.

That said, the eyes of those with PRV-eligible programs, and those considering entering the field to earn a PRV, will be on FDA and its actions not only in the decisions made, but in the timing of those decisions. FDA has an opportunity to solidify its commitment to the program and to the benefits to global health by continuing to comply with the PRV rules and timelines.

Don Joseph is the Chief Operating Officer at BIO Ventures for Global Health.

• To identify parts of the human genome that encode host factors that affect the bacterial or parasitic infection, for the purposes of targeting therapeutics at those factors
• To more quickly validate predicted host factors as compared to traditional gene knockout studies

Read Barder's full blog post here.

For decades children in poor countries have received vaccines only after they had been on the market for 20 years or more; when they were off patent and thus available at commodity pricing. If you apply this standard wait time to pneumococcal disease, which kills about a million children a year, 20 million poor children would die needlessly while children in rich countries were protected by very good vaccines.  

There are many barriers that create this inequity. For vaccines like pneumococcal vaccines where the research and development can be justified by the commercial opportunity, the biggest barriers are building the manufacturing capacity to supply the developing world (110 million live births vs. 10 million in developed countries) and ensuring that there is money to purchase the vaccines on behalf of poor countries. A well-designed AMC can address both of these concerns by creating a return on investment where market pull is otherwise insufficient.

It took vision and the work of many people and organizations to make this AMC a reality. Prof. Michael Kremer deserves considerable credit for championing this idea from the start. Dr. Ruth Levine’s project at The Center for Global Development turned a concept into a specific incentive structure ready for debate, analysis, and action by donors. Dr. Orin Levin of Johns Hopkins deserves enormous credit for putting pneumo vaccines on the global agenda, creating the evidence base that turned inertia into momentum, and for his unwillingness to accept “it can’t be done” as an answer when millions of children are dying needlessly. The donors to the AMC were courageous in willing to stake their credibility (and of course their funds) on a novel incentive mechanism. The Bill & Melinda Gates Foundation deserves special credit for sponsoring analytic and policy work to lay a foundation for a well-designed AMC and then for kicking in a substantial cash commitment. (Sadly, the Bush Administration did not join leaders from Italy, the UK, and others in the commitment.)

In an environment where donors often find it compelling to pick their own favorite approaches with “push” funding, it is refreshing to see new market-based solutions succeed by “pulling” successful innovation though the pipeline all the way to the people who need our help the most. I hope that 2011 will see commitments to another market-based solution for global health. They work.

J. Leighton Read, MD, is a General Partner at Alloy Ventures and is on the Board of BIO Ventures for Global Health. He is also a member of the UC Berkeley School of Public Health Policy Advisory Council.

There are thousands of patents in the Pool for Open Innovation, and contributors include GlaxoSmithKline, Alnylam Pharmaceuticals, Massachusetts Institute of Technology, Medicines for Malaria Venture, the University of California, Berkeley, and the California Institute of Technology. These patents cover a wide swath of intellectual property for use against neglected tropical diseases ranging from small molecules and their formulations, uses, and processes to RNAi technology and contributions around malaria research.

As the non-profit administrator of the Pool for Open Innovation, one of our responsibilities is to deepen the Pool by adding new contributors and patents. But even more important is our responsibility to ensure that the resources available are accessed by the capable innovators – the product development partnerships, academic researchers, and companies that can take what is in the Pool and turn it into life-saving treatments.

As a user, if you find a resource that you would like – whether a patent or related know-how – you can submit a request to BIO Ventures for Global Health (BVGH) through the site. BVGH will review each request, looking at the potential user’s scope of work, the nature of its resources and capabilities, and other relevant factors. Additionally, users must agree to abide by the Pool’s core principles. The Pool is guided by two key principles – licenses for patents and know-how will include therapeutics to treat the 16 neglected tropical diseases identified by the World Health Organization, and must be royalty free for sales in the world’s least developed countries. Additionally, if the know-how or patents sought by the end user are not listed as available, a general request can be sent to BVGH staff and they will work with the resource providers to see if they will add the requested resources to the Pool.

Shortly, we will be adding a search function to the site that will allow potential users to search the database of patents by patent number and keywords in the title and abstract (many of which include specific compound names, classes, and neglected disease relevance), or simply browse.

From a practical approach, free access to patents and know-how can clearly save time and money. Many of the resources in the Pool have had millions of dollars invested in their development. Access to these patents and know-how for non-neglected disease products could easily have fees and royalties in the ten to hundreds of millions of dollars. This is an unprecedented opportunity to build on these investments to develop the next generation of drugs that are so desperately needed by the world’s poor today.

We encourage you to visit the site and take a look around. If we can help you better understand what’s there, please let us know.

To learn more about the Pool, its contents, and how to access them, visit www.ntdpool.com. Contact us at ntdpool@bvgh.org with any questions or concerns.

Jennifer Manganello is Associate, External Affairs, at BIO Ventures for Global Health.
 

• Adds Chagas disease to the list of eligible NTDs, to align with the World Health Organization’s list of NTDs
• Allows for multiple transfers/sales of the voucher, once it is awarded 
• Allows for the withdrawal of a PRV by the sponsor before full review so the sponsor retains the rights to the voucher. This helps reduce the risk for industry to use the voucher because of the inherent uncertainty of clinical trial results, thereby increasing the estimated value
• Includes the ability of the FDA to make an “early designation” that a product would qualify for a voucher, at the request of a sponsor
• Closes the window for products that are in widespread use but have not gone through the FDA review process to be eligible for a PRV. We support the concept of closing the window for products that are in widespread use abroad because the PRV is meant to encourage new R&D, therefore it is of great value to include specific language around defining an “innovative treatment.” Specifically, the bill defines an innovative treatment as “a drug that has not been approved for commercial marketing for any tropical disease indication by a government authority outside of the United States for more than 24 months before the tropical disease product application is submitted.” While we believe this language supports the motivation for the incentives, we also want to make it clear that the first PRV award to Coartem (an anti-malarial treatment that was already in widespread use abroad) offers an important early test case for the program. To learn more about our thoughts on this, read our blog post on the subject here.

Welcome to my first blog post as the new Chief Operating Officer of BIO Ventures for Global Health! I’m very excited to be on board. A few introductory comments. . .  

My commitment to global health comes first from my industry experience and the desire for an evolving career path. I left law firm life more than 15 years ago to join a small biotech company, to become involved in life sciences and help make a difference in the way people live.  Since then I’ve been very fortunate to be with cutting-edge companies, technologies, and products, always with the goal of meeting unmet medical needs for the developed world.  Over time I realized that I wanted to take my industry knowledge and apply it to the unmet needs of the developing world—needs that are of course far more basic, yet are being left behind as the technological advances of the developed world continue. This gap results from many factors, including hugely disproportionate resources, inadequate incentives, the requirements of business models, and limits on access and distribution of current treatments.

I have been energized by the advent of “venture philanthropy” and a novel, results-driven approach to philanthropic effort.  This has created a new impetus for addressing not just global health but any number of social issues which are critical for the future of the planet:  education, literacy, and environmental conservation are just a few of what are now perceived as solvable rather than intractable, inevitable challenges.  The Bill & Melinda Gates Foundation has taken a giant leadership role in this regard (full disclosure:  they are a significant funder of BVGH’s activities).

So BVGH offered me a unique opportunity:  to use my industry background and training to help bring biotechnology innovations to the unmet needs of the developing world.  We address some of the “gap” factors described above in ways that are consistent with the business models, missions, and priorities not only of the biopharma community, but of global health organizations, PDPs, and funders. We intend to connect, communicate, and create information, ideas, and incentives to bridge the innovation gap.

I also joined BVGH through inspiration from my kids, whose world view is shaping my own.  They see the world without limits, without borders or boundaries, in their friendships; their communities; and their responsibilities.  I hope to live up to that ideal too.  More soon!

Don Joseph is the COO of BIO Ventures for Global Health

In order to most effectively connect innovators in global health, academia, and the biopharmaceutical industry, the conference is divided into two sections. The first section includes one day of high-level panels and plenary sessions and the second includes three days of one-on-one partnering meetings to uncover potential for collaborating on new technologies, new product development, and new funding. In the Forum’s one day of panel sessions, we’ve focused on a number of key topics that we think will provide insight, inspiration, and a concrete call to action for the biopharmaceutical and global health communities.

The first panel of the day will take a look at the drug development pipeline, particularly how it varies for developing world products as opposed to developed world products. We will hear from representatives from each stage of the pipeline – from academia to big pharma – to look at some of the challenges of translational medicine for global health, as well as some potential solutions.

Our second panel will bring BIO Ventures for Global Health’s recent publication Global Health Innovators: A Collection of Case Studies live to the stage. We’ll take a deep dive into one of the business models featured in the report and one from an upcoming study. Presenting companies and their partners will provide insight into their partnership structures, how global health product development has supported their core commercial strategies, and the strategic value that each gains from this effort.

The third panel will allow us a peek inside the boardroom, with an interactive discussion amongst senior executives from biotech and pharma to determine which of the current and proposed research and development incentives for global health are the most compelling. They will share what impact each incentive — individually or in tandem — would have on a company’s decision-making process.

We’ll round out the day with a fourth panel focused on the booming field of emerging markets. While emerging markets aren’t brand new, more and more companies are seeking opportunities to support their commercial strategies by working with developing world companies and manufacturers. We will hear some lessons learned from companies actively engaged in that space, as well as from some emerging market representatives on their experiences with U.S. and European companies.

These panels should prime attendees for what we consider to be the main event – three full days of partnering meetings at the BIO Business Forum. The partnering portion of the meeting is where the real action will happen, where people will sit down together and discuss real ways to move their technologies, products, or expertise forward. Our hope is that the call to action repeated throughout the programming on Monday will be firmly answered in the days that follow.

To register or learn more about the Partnering for Global Health Forum, visit the Web site: pgh.bio.org.

Jennifer Manganello is Development Associate at BIO Ventures for Global Health.

How much is the priority review voucher worth? In a 2009 paper, my colleagues and I estimated that the voucher could be worth several hundred million dollars. The value derives from three factors: shifting sales earlier, longer effective patent life due to earlier entry, and competitive benefits from earlier entry relative to rivals. In addition to the voucher value, the developer of the treatment is eligible for orphan drug tax credits equal to half of development costs.

Value by Class

In September 2009, Waseem Noor of IMS Health showed that the voucher value varies by therapeutic area, because FDA review times vary by therapeutic area. Noor analyzed approvals between 2002 and 2007. For treatments for the nervous system, the median difference between priority and standard review is about 9 months (3 quarters) whereas for respiratory system the median difference is zero. Likewise, the value of applying a voucher to a blockbuster treatment for the nervous system could be $120 million, whereas the voucher might have negligible value if applied to a treatment for the respiratory system.

Noor's estimates account for several sources of uncertainty, including whether the product will be a blockbuster and whether the product will receive priority review without a voucher (because of the product's own novelty). For example, he estimates that the value of a voucher for type 2 diabetes medication Januvia (Sitagliptin) would be $162 million if the alternative were standard review. There is, however, an estimated 23% probability for priority review in Januvia's therapeutic class, so he estimates that the voucher value would be $125 million if applied to Januvia ex ante. This assumes that the value of the voucher is zero if the product receives priority review on its own merits. The voucher will have some value, however, if it can be transferred to a different product.

Value to Society

In the original paper where we proposed the priority review voucher, my colleagues and I focused on the value to the voucher holder, but the voucher potentially creates much more value to society if it motivates new cures for neglected diseases, and if consumers in the United States value earlier access to drugs beyond the price they pay. We estimate that the additional value to consumers of earlier access is half the value of the voucher. Hence, if the voucher is worth $100 million, then the consumer surplus is $50 million from faster review. The value of the new cure for a neglected disease could be many times greater, given that, for example, tens of millions of disability adjusted life years are lost each year to malaria.

- David Ridley is an Assistant Professor at Duke University. David, with Henry Grabowski and Jeffrey Moe, proposed the priority review voucher in a 2006 paper.

To read more:

• Henry G. Grabowski, David B. Ridley, and Jeffrey L. Moe. "Priority Review Vouchers to Encourage Innovation for Neglected Diseases.” In: K. Eggleston, ed. Prescribing Cultures and Pharmaceutical Policy in the Asia-Pacific. Brookings Institution Press. 2009.
http://faculty.fuqua.duke.edu/~dbr1/research/priority.pdf
• Waseem Noor. "Placing Value on FDA's Priority Review Vouchers." In Vivo. September 2009.
http://www.imscorprep.com/tl_programs/documents/IMS0909iv.pdf
• David B. Ridley, Henry G. Grabowski, and Jeffrey L. Moe. "Developing Drugs for Developing Countries." Health Affairs. 2006. Vol. 25, No. 2: 313-24.
http://content.healthaffairs.org/cgi/content/abstract/25/2/313
• BVGH Priority Review Voucher Web site: prvinfo.org
 

As 2009 comes to an end, BIO Ventures for Global Health is excited about what lies ahead in the coming year. Each day, we see enthusiasm growing around the important role that biotech can play in solving some of the world’s most pressing health issues, and each day we work to convert that passion and enthusiasm to action. While we cherish the opportunity to work on life-saving issues, our focus on results reminds us that our actions must lead to commercially viable product development.

We took some important steps forward in 2009. We released two reports “Global Health Innovators: A Collection of Case Studies” and the 2009 edition of the “Global Health Primer.” We’ve moved incentives for research and development on drugs, vaccines, and diagnostics for the developing world ahead, particularly the FDA’s priority review voucher program. We have also made significant progress in the design of new research and development incentives for drugs for Chagas disease as well as point-of-care diagnostics. We also launched a Corporate Leadership Council to bring together biotech executives with an interest in global health, and debuted a new blog.

With these successes under our belt, here are just a handful of new projects we will be working on in 2010:

• Global Health Connect, a network that connects industry, nonprofits, governments, and the academic community to exchange expertise that will help in the creation of new products.

• Global Health Markets, a program that will help biotech companies navigate markets, supply chains, and processes and define exit strategies for global health products that differ from traditional pharmaceutical partnering models.

• 2010 Partnering for Global Health Forum, a meeting in conjunction with the BIO Annual Convention in May. Keep an eye out for an upcoming announcement.

• Diagnostics Innovation Map, a report that analyzes existing and upcoming diagnostic technologies that can be applied in the developing world.

• New Incentives Initiative, a program that helps biotech companies with the financial risk of developing new drugs, vaccines, and diagnostics for the world’s poor.

• A New and Improved Web site

Our goals for this year are ambitious, so we hope you will join us in making a commitment to ensure that we reach them. Your support is critical to our success. Please consider making a donation using the big red donation button below or by visiting our Web site.

Thank you for your support and best wishes for a happy new year,

Melinda Moree
CEO

What ties together someone who works on a disease like Chagas, leishmaniasis, or lymphatic filariasis and someone who works on Rett syndrome or multiple myeloma? Your first thought might be ‘Not much.’ But an interesting group called FasterCures brought 600 of us together in New York last week to find out.

I mention Rett syndrome because I sat next to two parents of girls suffering from the disease during one of the meeting’s lunches. The symptoms of this rare disorder appear after an early period of apparently normal or near normal development until six to 18 months of life, when there is a slowing down or stagnation of skills. A period of regression then follows when the child loses communication skills and purposeful use of her hands. Over time, motor problems may increase, but eye contact and communication generally improve.

I sat there at lunch feeling the pain of these two parents and yet also knowing that the diseases that BIO Ventures for Global Health address result in hundreds of millions of cases of illness, millions of deaths a year, and untold amounts of human misery and poverty. But for all our disparities, the meeting’s attendees shared a feeling that the biomedical enterprise is not focused in a powerful enough way, or perhaps not designed, to cure diseases. The “aha” moment for new drugs, vaccines, and diagnostics may come from academic labs, research foundations, or government labs—but the machinery to turn them into products that can make a difference in peoples’ lives lies almost exclusively within industry.

The business model that has brought us needed drugs, vaccines, and diagnostics for as long as we can remember is an industry in crisis. This presents both a challenge and an opportunity. Opportunities previously seen as “niche” markets make more economic sense to companies if foundations, patient advocacy groups, and governments have created the infrastructure to “de-risk” the endeavor for companies. The lure of emerging markets is making the globe smaller and making companies rethink how they will need to play in these markets and still profit. Success surely will not come through ignoring diseases associated with inequity and poverty.

In the end, what drew the attendees together was the imperative for patient need to be at the center of the search for new drugs, vaccines, and diagnostics; the recognition that we are all players in the search for cures and that we all have a need to find creative and flexible solutions; and an utter lack of patience with bureaucracy and inaction. There is an illusion that not doing something is the safest path to take. But in biomedical research, inactivity and passivity results in death.

Melinda Moree is the interim CEO of BIO Ventures for Global Health

Last week’s Global Forum for Health Research meeting in Havana featured many talks about technology innovation. Buzzwords such as “capacity building,” “technology transfer,” and “innovation networks” were bandied about. Surprisingly, however, true product innovators were dramatically underrepresented at the meeting. There were a few basic scientists and almost no representatives of the most innovative sectors of the biopharma and diagnostics industries—the small to medium-sized companies whose lifeblood is turning basic discoveries into products that meet pressing health care needs. Product development experts, meaning professionals who can understand and assess technology but also have the bruises that come from interacting with regulators and customers, could have added to the rich discussions that were already occurring.

One leitmotif of the Forum might be paraphrased as: “We’ve got so many people afflicted by easily treatable or preventable illnesses who are not being helped. Why should we be making any investments in long-term and expensive technological innovations when we aren’t taking advantage of the tools we have today?” And these critics, a minority but a vocal group at the Forum, have an important point: we need to make sure we are doing what we can to alleviate human pain and suffering if a good solution exists today. But in all too many cases—the treatment and diagnosis of tuberculosis, prevention of HIV transmission, vaccination against malaria, and treatment of Chagas disease, for example—the available tools are inadequate, have too many side effects, or simply don’t exist. Thus we need to commit to innovative research and development to solve these and many other medical problems.

And so another theme, expressed powerfully by Melinda Moree, Interim CEO of BIO Ventures for Global Health, and Maria Freire, President of the Lasker Foundation, was that the choice between delivering products now or developing products for the future is a false dichotomy. We need both. Sometimes a good solution is available now but it is ensnared in financing, delivery, or intellectual property issues. We should without hesitation support the use of resources to address health problems that are currently solvable. Sometimes, however, the development of new and imaginative technologies stimulates the global health community with the art of the possible. Sometimes, the ability to treat disease with a novel approach is so transformative — as in the innovations of antiretroviral therapy for HIV — that it leads to revolutionary changes in patient health while strengthening health delivery systems in the process. (On a hopeful note, the initiation of phase 3 testing of a malaria vaccine, developed by GSK Biologicals in collaboration with the Malaria Vaccine Initiative, promises to be such a transformative technology.)

In several instances, academic or policy speakers at the Forum gave vacuous descriptions of product development. Product development was reduced to the transfer of academic research discoveries to manufacturing partners who could then make and sell a product. This caricature of product development does injustice to the many disciplines required to develop products as well as the unpredictable nature of biomedical innovation. So one way to strengthen subsequent meetings would be to include a subset of policy-minded product developers and entrepreneurs who can discuss what it actually takes to finance, organize, and mobilize a product development organization. It is only by bringing together a broad range of disciplines that we will discover new and innovative approaches to finding solutions for pressing global health problems.

David Cook is the Vice President of Business Development at BIO Ventures for Global Health.

I’m just returning from the Global Forum for Health Research meeting in Havana, Cuba. The theme of this year’s meeting was “Innovating for the Health of All.” As a veteran of the biotechnology industry I thought I knew something about innovation. After all, innovation has been the engine behind the emergence of biotechnology worldwide from a cottage industry in the late 1970s to a vital force for improving health care and building national wealth. But I encountered a new and puzzling use of the term innovation, in the form of “social innovation” to address global health needs.

To understand social innovation in the context of global health, I spoke with Rakgadi Mohlahlane, a senior researcher at the University of Pretoria in South Africa, whose focus is HIV medical education in Africa. She described one of the problems that she is passionate about addressing: how can one design programs for HIV therapy for residents of remote African villages? In South Africa, it is estimated that 5.2 million individuals are living with HIV among a population of 27 million people. The country has one of the world’s largest HIV treatment programs with about 5 million people taking antiretroviral (ARV) therapy, but it primarily reaches urban residents and has not penetrated into more remote, isolated settings.

Why does this problem require “social innovation?” Providing ARVs isn’t just a matter of sending pills along with instructions for use. First, the knowledge that someone is HIV-positive is profoundly transforming for the individual, for their family, and for their community contacts. And while testing is an essential first step in controlling the epidemic, it’s a step approached fearfully and is often avoided by individuals who may be HIV-positive. Infection is still a cause for social opprobrium, which is perhaps even more intense in small communities where it is difficult to find the refuge of anonymity. Second, there is an intense need for culturally appropriate medical education—on the value of knowing one’s HIV status, on the complex management of this disease, and on the appropriate precautions for both HIV-positive and HIV-negative individuals. Third, implementation plans have to take into account the dearth of medical professionals who can provide oversight of patients to stay on chronic therapies or make adjustments to regimens. ARVs can have serious side effects; regimens can be complicated; and poor compliance can result in the development of drug resistance. How do you effectively support a patient in an isolated village of 600 people who must take a chronic therapy for the rest of his or her life?

Think of the challenge of developing an HIV testing and treatment program for a rural and scientifically unsophisticated community in Africa. And then multiply by the tens of neglected diseases and hundreds of different community, religious, and social contexts around the globe. And that’s how I came to understand the need for social innovation in the context of global health.

David Cook is the Vice President of Business Development at BIO Ventures for Global Health.

Dateline: Havana

Can product development partnerships (PDPs) deliver? This was the question tackled during a session at this week’s Global Forum for Health Research in Havana. The presentations actually spoke little to the topic—perhaps because PDPs are taking on some of the biggest scientific challenges of our time such as developing a HIV vaccine, a tuberculosis vaccine, and broad-spectrum anti-virals. But it is a question worth asking.

PDPs are a relatively new concept. The earliest were started just over 10 years ago and are either free standing nonprofit organizations or several different product programs housed together under one nonprofit. The basic concept when they began was that industry has expertise and capabilities that are needed to make new vaccines, drugs, and diagnostics for neglected diseases affecting poor countries but this expertise and capability is not being applied to diseases primarily affecting the developing world. PDPs were created to bridge the gap between global health need (public sector) and an innovation system that responds to market demand (private sector).

Given the timelines for developing novel drugs, vaccines, and diagnostics, it may be too early to say if PDPs have indeed delivered upon their promise. What is stunning is that no one has developed evaluation metrics to measure either effectiveness or efficiency, and no PDP has been evaluated by such metrics. Billions of dollars have been invested in these mechanisms by philanthropic organizations such as The Bill & Melinda Gates Foundation and bilateral donor governments such as the U.S. Agency for International Development and the U.K. Department for International Development.

The companies who make products are evaluated daily by their shareholders or at least quarterly by their venture capitalists or investment bankers. Companies also use internal metrics to improve their business processes and improve profits. One company was able to reduce their time to peak sales in half in a short period of time, greatly improving their profits. Should we not be taking the same approach in the public sector—greatly improving the speed of development and uptake of new products so that more lives can be saved?

My guess is that most PDPs would come out well in an evaluation — the pipelines are filling up with new drugs, vaccines, and diagnostics targeted toward neglected diseases. But for some diseases, a single PDP is virtually the only mechanism for funding research on that specific disease intervention. We should assure ourselves as a global health community that these investments are operating efficiently and effectively.

We as a community stand the strong chance of donor fatigue because the funding for PDPs often comes from agencies who measure outcomes in election cycles. It may take decades for new chemical entities, novel vaccines, and appropriate diagnostics to be developed because in addition to market barriers, many of the neglected diseases being tackled are scientifically very challenging. Adopting robust evaluation schemes now may help to show interim progress that points towards timelines for future products with health outcomes that can be counted in hundreds of millions of lives saved.

Melinda Moree is the interim CEO of BVGH

Havana? Yes, I’m blogging from Havana, where I am attending the Global Forum for Health Research Annual Conference. This year’s theme—“Innovating for the Health of All”—is perfectly in line with our goals at BVGH to bring capable innovators and technological know-how to global health research and development. Even more exciting, the Forum’s location in Cuba is drawing attention to Latin America at exactly the right moment.

Why does Latin America deserve all eyes and ears right now?

First, a number of diseases endemic to Latin America and the Caribbean are, at long last, gaining important publicity. The poster child example of this is Chagas disease, which was discovered in 1909 by Carlos Chagas, a medical doctor in Brazil. The disease now affects more than 8 million in Latin America and approximately 300,000 in the United States. For decades, the disease was systematically ignored by researchers and patients were offered little in the way of effective treatment. The only drugs that exist today, nifurtimox and benznidazole, both have high toxicities and long treatment times. There is no point of care diagnostic and no test of cure. However, this neglect is beginning to change. Médecins Sans Frontières (MSF) and the Drugs for Neglected Diseases initiative (DNDi) have embarked on an advocacy campaign around the issue. DNDi is also building a pipeline of drugs to test against the parasite that causes Chagas disease and to help identify clinical trials capacity for drug trials. BVGH is working behind the scenes to make sure that important compounds make it to the relevant players in drug development. The synergy in the global health community around the need to find new and better drugs and diagnostics for Chagas disease—and to treat those patients already affected—makes now an important time for the global health community to gather in Latin America to focus on the need for innovation.

More importantly, Latin America now has a thriving biopharmaceutical sector. Brazil’s well-known generics industry has a long history of success, and public sector research institutions, such as Fiocruz, have strong vaccine capabilities. Fiocruz is building a new translational research facility, and already has an alliance with Genzyme on Chagas disease. In addition, Brazilian President Lula has made innovation a pillar of public policy during his term as president. While Brazil has long been recognized for its growing technological capabilities, other countries in Latin America stand out as well. Mexico is among one of the top ten drug producers in the world, and remains the largest drug exporter in Latin America ($1.5 billion in exports in 2007). In both Brazil and Mexico, there is a growing interesting in innovative pharmaceuticals.

Sadly, less than USD 1 million was spent last year on research and development (R&D) for new drugs for Chagas. But there is a real opportunity here for capable innovators with compounds that could be tested for activity against Chagas to join in the movement that will only grow. These large, mid-size, or small companies could, in partnership, reduce their risks of development. An R&D incentive would also certainly help to fill up the pipeline quickly with a newer generation of drugs. The innovation process that creates novel compounds from good research ideas is still largely not found in the developing world, as was discussed in a very interesting forum held Monday by the Pan American Health Organization. The space is open for drug hunting companies and diagnostic companies to step into. Our Board chair Carl Feldbaum, our VP of Business Development David Cook, former BVGH CEO Chris Earl, and I spent a fair amount of time discussing the value that BVGH could add to solving the problem of Chagas, which causes so much suffering in Latin America. Fueled by cigar smoke (Yes—I did!) we see the opportunity. If you want to see it too—please be in touch with us.

In the developing world, a basic relationship exists between the need for diagnosis of infectious agents and their treatment. Diagnostics — which provide important information to aid physicians in the diagnosis, management, and treatment of myriad diseases — are critical for identifying communicable diseases that cause colossal morbidity and mortality. Properly designed diagnostics will enable the selection of medicines for treating infectious diseases such as HIV, TB, and malaria, along with a wide range of parasites, viruses, and bacteria; just as important, diagnostics thwart the over-use of medicines due to misdiagnosis, which is critical in the fight against the emergence of drug-resistant bugs.

The greatest need in the developing world is for molecular diagnostic tests that meet the requirements of cost, portability, and ruggedness for low resource environments. Molecular tests detect the presence of nucleic acids—DNA or RNA—from specific infectious agents. They are exquisitely sensitive and a successful platform could find broad applicability in the developing world, since the nucleic acid signature of each infectious agent is already known. So what prevents the many companies with such technology from developing a point of care (POC) molecular diagnostic platform?

One fundamental barrier is the lack of clearly identifiable market demand in the developed world. Companies are reluctant to spend tens of millions of dollars in product development if they do not have a reasonable forecast of recovering their investment and making a profit. Another barrier is cost. Portable technologies are available for selected indications in the developed world today, but the cost per test is too high for widespread utilization in the developing world.

One reason why molecular tests are common among rich nations is that there is a large infrastructure of centralized laboratories and sample transportation to support the diagnostics testing industry. The existing investment in central lab infrastructure biases future investments to take advantage of that sunk cost. But developing world markets have not made such a large investment, and just as they have for mobile phone technology instead of landlines, developing countries may jump over the step of infrastructural investment in favor of a network of decentralized laboratories that provide broader access to patients.

At BVGH, we believe that a financial incentive should be created to remove some of the barriers in order to engage innovative companies in product development for POC diagnostics that meet the needs of portability, ruggedness, and cost in the developing world. The financial risks that companies take on could be minimized by incentives that reward them for creating platforms and products that meet requirements appropriate for resource poor settings. In order to qualify for these incentives, companies would need to agree to basic principles ensuring access to these products in the developing world at a reasonable cost. One great benefit of the developing world is that there is a huge potential volume of product required, and large volumes can enable suppliers to reduce unit costs by developing automated manufacturing lines and amortizing the capital investment over large numbers of units. And reasonable cost needs to be defined by the health benefit the diagnostic enables as well as a final cost that supports a sustainable market.

What would an innovator get in return for such incentive funding? The successful company would have created a POC molecular platform that is robust, portable, and low cost—precisely the attributes that would enable new indications in the developed world markets. Such a platform could enable tests for malaria, Chagas, and onchocerciasis in low income countries—while at the same time serving as a basis for running tests for cancer and swine flu in middle and high income countries. We believe that CEOs and strategic thinkers within companies will support an appropriately designed incentive as something that benefits their overall corporate mission while serving the needs of the impoverished citizens of the developing world. And bringing companies with a successful track record of product development into global health is ultimately the best way to ensure that diagnostics are available to save millions of lives in the developing world.

David Cook is the Vice President of Business Development at BIO Ventures for Global Health.

There have been no FDA-approved or designated treatments for Chagas disease since 1983. That’s more than 25 years. Diseases like Chagas, which are relatively uncommon in America (but affect a large part of the global population), are considered rare by the FDA and drugs, vaccines, and diagnostics targeting them are termed “orphan,” because biotechnology and pharmaceutical companies are not interested in owning or producing these treatments. So how do we create systems that ensure more funding for Chagas and other neglected diseases of the developing world?

Last Friday, this very question was debated at the Tufts Center for the Study of Drug Development’s forum, “Neglected Diseases in the Developing World.” During the forum, representatives from the FDA, NIH, USAID, developing country governments, non-profit organizations, and leading academic institutions discussed how to incentivize drug development and ensure that new drugs, technologies, and information make it to people who need them.

Among different strategies to encourage research and product development for neglected diseases, Tim Coté, Director of the Office of Orphan Product Development at the FDA, highlighted and strongly endorsed the priority review voucher (PRV) program. This program awards a voucher to a company that develops a drug or vaccine that targets a neglected disease. The voucher can be “cashed in” for use with a future product of the company’s choosing. It makes that future product eligible for a “priority” review—a process that is generally 4-10 months shorter than a standard review, but is no less rigorous. The benefit of the program is that earlier market entry of the future drug will allow for longer market exclusivity and possible gains that come from beating competing drugs to market.

Sixteen diseases currently define the list of qualified neglected diseases. This list has been debated, and the FDA held a public comment meeting in December 2008 asking for suggestions for expanding the list. Chagas disease – which is not on the current list – was recommended by BVGH for inclusion during this time.

We will continue to advocate for Chagas’ inclusion, but in the meantime it is important to show that the PRV program works. And for the program to truly act as an incentive, the economic benefit to the company has to be as big as possible. To a drug company with development costs exceeding hundreds of millions of dollars, the larger the windfall, the better the incentive. Only a huge success will truly convince drug companies to invest in new research and development for diseases of the developing world.

To learn more about PRVs, please visit BVGH’s Web site on the program: prvinfo.org.

Anna Heard is the Director of Policy and Advocacy at BVGH.

Chagas disease is caused by a parasite transmitted through the bite of an insect. It is endemic to Latin America and most commonly afflicts the poor living in rural villages. Due to the high prevalence of infected individuals, blood transfusion is now the second leading cause of transmission in places like Brazil. Estimates vary, but there are probably 10 million people who are carriers of this disease, and approximately 30 percent of infected individuals will develop heart failure (cardiomyopathy) and die prematurely as a result of their infection.

BIO Ventures for Global Health is committed to bringing the tools of biotechnology and the expertise of the biotech industry—which have created dramatic improvements in the treatment of chronic diseases such as autoimmunity, heart disease, HIV, and cancer—to bear on the problem of neglected diseases such as Chagas disease. So an important question is: how can the global health community marshal the resources necessary for serious, innovative drug and diagnostics research and development (R&D) to address this health scourge? One mechanism is to create appropriately sized incentives to make it feasible for companies to invest in global health; the quid pro quo for this financial support is that companies must agree to principles allowing the poorest populations of the world to access these novel medicines at low or no profit. But incentives don’t have to be the sole solution.

In listening to the speakers at last week’s UCLA symposium “Chagas Disease in the Americas: Improving Access and Tools for Patient Diagnosis and Treatment,” I was struck by similarities between Chagas disease and a viral infection common in the U.S., hepatitis C virus (HCV). It turns out that there are about 3 million Americans who are infected with HCV, comparable to the 3 million people infected with Chagas in Brazil and Mexico combined. Like Chagas, HCV is a silent infection that causes long term damage to a major organ only after several decades. HCV is a leading cause of liver failure and liver cancer worldwide, while Chagas causes heart failure. Like the treatments for Chagas, the currently licensed therapy for HCV is difficult to tolerate (it’s described as feeling like having the flu for 48 weeks!) and, in the case of patients with genotype I virus (the major type in the U.S.), only about 50% effective.

Yet the parallels between Chagas and HCV only go so far. Less than $1 million was spent in 2008 on developing new medicines to treat Chagas. This is a paltry sum when one considers that it costs $300 to $800 million to develop a novel drug! But it reflects the reality that patients who have Chagas are among the poorest in the Western Hemisphere and have little voice politically or economically. While Chagas is an orphan, there is a monumental effort by pharmaceutical and biotech companies to develop novel therapies for HCV: one recent assessment noted that there are more than 40 novel drugs being tested at various stages of clinical trials! These therapies promise to transform this potentially deadly viral infection into one that can be cured with pharmaceutical intervention.

So why this dichotomy between Chagas and HCV? The main difference is that drug development companies can identify a robust market for HCV therapy. The market is currently well over $2 billion in the U.S. and Europe, and it stands to grow substantially upon approval of the next generation of drugs. In contrast, medicines for Chagas are sold at essentially no profit and the market is not an inducement to innovation. But one under-recognized fact regarding Chagas is that about 300,000 people in the U.S. are actively infected with perhaps another 60,000 infected individuals in Europe. These are mostly Latino immigrants who are now living outside their native countries. It stands to reason that these individuals, in the absence of a safe and effective therapy, will become a significant economic and medical burden if their disease goes untreated and they develop heart disease at some point in the future. So there may actually be a compelling financial–as well as humanitarian–reason to engage in drug discovery to create safer and more effective treatments to cure Chagas disease. While the market opportunity alone may not be sufficient to induce significant R&D investment, BVGH hopes that a combination of incentives and the recognition of a real commercial opportunity in the U.S. and Europe may lead more companies to take steps to invest in Chagas disease drug discovery.

David Cook is the Vice President of Business Development at BIO Ventures for Global Health.

We’ve come a long way, baby. In 2001, 42 pharmaceutical companies shocked the global health community when they took Nelson Mandela and the South African government to court to protest the importation of cheap, generic drugs for AIDS patients. To the global health community — who argued that saving lives was more important than profits — the move seemed questionable at best. Why would corporations take Nelson Mandela to task for trying to provide critical treatment to a disintegrating society?

The answer, of course, is that pharmaceutical companies felt that the law violated patent agreements and unfairly targeted large drug manufacturers. Patents protect developers — allowing them to invest risky capital in research and development (R&D), with the anticipation of profits without competition until the patent expires. And economists and pharmaceutical industry experts agree that intellectual property (IP) stimulates innovation. Pharmaceutical companies asked the global health community: what would happen if there was no blockbuster drug from which to create a generic?

Yet, neglected diseases are labeled as such because they are just that – neglected. Neither patent protection, nor anything else, has proved to be a silver bullet to encourage new R&D. This is beginning to change.

Fast forward eight years and we have the creation of patent pools, providing wider access to IP in the hopes of spurring the type of innovation needed for neglected diseases. In February 2009, GlaxoSmithKline (GSK) announced the formation of one such pool as part of their larger commitment to global health. They donated the proprietary research for 16 neglected diseases to this pool and were quickly joined by Alnylam Pharmaceuticals, a Boston-based biotechnology company. And other organizations are pursuing similar ideas. The World Health Organization recently called for the creation of patent pools, and UNITAID, a purchaser of AIDS treatments, has expressed interest in creating a pool focused on AIDS therapeutics.

In addition to large patent pools, some biotechnology companies have achieved one-off success in negotiating IP agreements with partners. This has proved critical to the effectiveness of partnerships when creating new interventions for neglected diseases. BVGH released a report in August 2009, Global Health Innovators: A Collection of Case Studies, that looked at six partnerships in global health R&D. In each of the case studies presented, IP agreements were reached without major conflict. For instance, in a partnership between Genzyme Corporation (Genzyme), Medicines for Malaria Venture (MMV), and the Broad Institute of MIT and Harvard aimed at malaria drug discovery, Genzyme committed from the beginning to contribute any IP related to malaria to the public good. This promise facilitated the collaboration and leading candidates are expected to enter phase 1 clinical trials in 2011.

While the debate over IP continues, it is important to recognize that access to medicines is not possible when there is no medicine to deliver. Of the 19 diseases examined in the 2009 edition of BVGH’s Global Health Primer, nine lacked a drug, vaccine, or diagnostic. Patent pools and creative interpretations of IP rights are an important incentive to companies that work in global health, but IP is one part of the larger picture that includes incentives like Advanced Market Commitments and Priority Review Vouchers. Ultimately, we must continue to unearth new ways to engage companies in global health R&D and save lives in the developing world.

Both Global Health Innovators: A Collection of Case Studies and the 2009 Global Health Primer are available for download on the BVGH Web site. Please contact info@bvgh.org to request a hard copy of the documents.

I’m more than enthusiastic about this moment at BVGH. Thanks to hard groundbreaking work by others over the last four and a half years, the biotech industry is now poised for significant involvement in neglected diseases of the developing world. Getting to this point was no mean feat for BVGH. The good intentions and will were there–many biotech CEOs come from academia, non-profits, and other organizations where they witnessed first-hand the health burden borne by the poorest among us. But there were (and still are) barriers between that goodwill and committing a technology-rich but often cash-strapped company to the quest for a new drug for populations that simply cannot afford them.

I represented the biotech industry in Washington, D.C. for almost 12 years, but I confess I never fathomed the internal dynamics of an individual company until I left Washington and began to serve as a director on a couple of biotech boards. That’s where I witnessed first-hand the economic and other pressures that cause companies and their shareholders to (excuse the hackneyed expression) “go for the gold,” i.e. to develop the best drug, a “blockbuster” for an expansive population that can pay, typically in North America, Western Europe, and/or Japan. For the record, I did not invent this system, and won’t apologize for it. BVGH has thus far worked rather successfully with the system as it is — diplomatically, but with no sugarcoating.

Chagas, dengue, and leishmaniasis are hardly household words in the U.S., and there are so many other killers abroad — in addition to HIV/AIDS, tuberculosis, and malaria– which we assume Americans have heard and care about. The latent strength of the biotech industry to deal with these diseases lies in what I’ll call its discovery diversity–there are 4400 companies out there at last count. BVGH has now identified many of the barriers to new drug development for neglected diseases, created an innovation map and specific business cases, and detailed the direction, guideposts, and milestones needed to get us further down this road.

The “dots” are now there to follow, but it won’t be easy–it’s more akin to putting together a 1000-piece puzzle. We can do it. I believe we’re on the verge of a transformation, and I feel lucky as the new chairman to both ride and help guide this fast-building wave.

Carl Feldbaum, Chairman, BVGH Board of Directors

Carl is well loved and respected around the world for his ardent belief that biotechnology can improve the lives of those suffering from diseases, and especially those suffering from diseases that primarily affect the poor. BVGH was spun out of BIO during Carl’s tenure as President based on this vision.

Carl is “retired” but remains active in the biotech industry by serving on the Board of Directors of Actelion, LTD and Exelixis, Inc., both biotechnology companies, and the Biotechnology Institute. Along with Carl’s extraordinary depth of knowledge regarding the biotechnology industry, he brings to the role a distinguished policy background. He came to Washington, D.C. in 1973 as an assistant special prosecutor for the Watergate special prosecution force. He served as chief of staff to Senator Arlen Specter (D-PA) and was president and founder of the Palomar Corporation, a national security “think tank” in Washington, D.C. Before founding Palomar Corporation, Carl was assistant to the Secretary of Energy, and served as the Inspector General for defense intelligence in the U.S. Department of Defense.

Carl succeeds Rob Chess, Chairman of the Board of Nektar Therapeutics, who served as BVGH board chair since the organization’s inception. Under Rob’s leadership, BVGH has grown from a concept to an organization focused intensively on bringing more biotech companies into the global health arena and achieving tangible outcomes. Rob will continue to serve on the BVGH Board.

BVGH is fortunate to have the time and commitment of its board members who are steeped in the financing, policy, and operations of the biotech industry. Beware Friends of Carl—your next phone call may very well be a request to help BVGH in accomplishing its very important mission.

Melinda Moree, interim CEO, BVGH

In last night’s speech on health care reform, President Obama outlined his plan to make affordable health care available to all U.S. citizens, an important and worthy undertaking. Important organizations and individuals are working to ensure that the efforts to restrain and cut costs do not come at the expense of our scientific leadership, which has made the U.S. the leading innovator in developing new drugs, vaccines, and diagnostics that improve health and save lives. But perhaps less well known is the critical benefit this innovation has not just on U.S. citizens, but on people around the globe.

For decades, people all over the world have depended on U.S. ingenuity in the development of new drugs, vaccines, and diagnostics. The first AIDS drug was developed by U.S. scientists in 1987, just four years after the HIV virus was identified. Since that first drug was identified, 31 medicines to treat HIV/AIDS have been approved. The increased availability and utilization of newer prescription medicines has helped to reduce the U.S. death rate from AIDS substantially in recent years. Moreover, there are now more than two million people in poor countries who are being treated with anti-retrovirals. This is the type of broad impact that American innovation is having.

Research!America has tracked consistently strong public support for the U.S. to maintain its leadership role in health and medical research. Indeed, an overwhelming 88% of Americans think that the U.S. should be a leader in research to improve health not only in the U.S. but around the world. Along with improving health systems, medical innovation is key.

This is a case where the American people can help themselves continue to receive new life-saving medicines and also help people around the world who are suffering from ill health. As the horse-trading around health care reform continues, we hope the positive impact the U.S. can make around the globe through the development and use of new drugs, vaccines, and diagnostics is taken into account.

Melinda Moree, interim CEO, BVGH