Written and submitted by Sharon Terry
This guest blog post is by Sharon Terry, President and CEO of Genetic Alliance and Founding CEO of PXE International. Sharon spoke on the TEDMED stage in 2016, and you can watch her talk here.
We are told frequently that we’re fortunate to live in a new golden age of medicine, that advances taking place in genomic research, immunology and other biomedical research fields are bringing us closer to a horizon that will see more effective treatments and even cures for the diseases that have plagued humankind for generations.
And yet, the reality, to this point, is somewhat less glowing than the sales pitch. A study cited in the New York Times found that we’re spending more money than ever on drug development with fewer positive results to show for it. The study found that, for every billion dollars spent on pharmaceutical research and development since 1950, the number of new drugs reaching the marketplace has fallen every nine years by roughly half. So, we remain in a state in which there are thousands of diseases, but only hundreds have treatments.
There are a number of reasons to which we can point for this declining productivity in the drug development pipeline, from the higher costs of conducting clinical trials to the need for Food and Drug Administration reforms. From my own experience, however, I would strongly suggest that the biomedical research infrastructure needs to take a hard look at itself and ask, first, whether a hypercompetitive, non-collaborative approach to biopharmaceutical science is conducive to achieving optimal progress and, second, why it is not actively engaging some of its most valuable resources—patients and family members.
In my 2016 TEDMED talk, I discuss my family’s personal experiences with the medical research community after my two children were diagnosed with PXE, a rare genetic disorder that results in a slow advance of premature aging. After the diagnosis, we were contacted by researchers asking for blood samples for a research project focused on identifying the gene that causes PXE. Only two days later, another research center also wanted blood samples. Not wanting my young children to be stuck with needles twice, I asked if they could share the samples taken by their Boston counterparts. They looked at me as if I was suggesting using leeches and spells to cure this disease.
That’s when we realized that the world of biomedical research is an alien landscape to those of us who are unfortunate enough to be dependent on it. There is intense competition between scientists and academic institutions. Ok, I know that competition can generate great progress in an economic paradigm. But, in academic research, where information is critical to medical breakthroughs, and tax payer dollars are supporting the work, the hoarding of data in order to get more funding, more published articles, a better tenure track, and faster promotions is antithetical to what we need for our, and our children’s, health and wellbeing.
This experience also drove home the “us versus them” nature of researchers’ relationships with research participants and their families. There is a great deal of conversation and rhetoric around participant and patient engagement. In most cases, though, that phraseology is often code for recruiting more clinical trial participants to increase sample sizes. In seeing patients only as data points instead of true partners, biomedical researchers are missing a key opportunity to advance and accelerate their work.
My organization Genetic Alliance, in partnership with Private Access, is putting this theory into practice through an undertaking called Platform for Engaging Everyone Responsibly (PEER). The individuals participating in PEER are much more than statistics in a limited disease silo. They share health information and “patient reported outcomes,” from their lived experience, which warrant attention and resource commitment. In one example, we’ve worked closely with a number of sickle-cell patient advocacy organizations, learning from individuals about the aspects of the condition that matter most to them. Combining this with socioeconomic data, we were able to provide the FDA—for its patient-focused drug development program—valuable data from individuals who can’t come to Washington, DC for a hearing, and who are unlikely to use the agency’s web-based system to record comments.
This effort to better engage patients as true experts, is an initiative for which we should have the support of the full biomedical research establishment, instead of resistance. Too many times the experience of people living with a condition is dismissed or overlooked. There should not be an “us versus them” dynamic to pursuing treatments and cures. It remains, since my children were first diagnosed with PXE in 1994, a mystery as to why we’re not all in this together, pulling in the same direction.
It is clear that the status quo, when it comes to drug development, is not working. What will it take for us to wake up and realize that individuals and their families are not only demanding a change, but we’re leading the way to change.