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