- Patient commitment may not only facilitate the development of new therapies, but may also be required
- Many patient groups feel increasingly obliged to take treatment research into their own hands
- Relying on what is essentially a patronage system for medical research is uncomfortable
Patients who take a close look at medical science in search of treatments are often appalled by what they discover. On the one hand, there's academic research, a self-contained and self-absorbed universe of its own where data may be internally consistent (on a good day) and robustly reproducible (on a very good day), yet often has depressingly little relevance to real-world clinical conditions.
Meanwhile, many medical products companies, faced with an increasingly unstable environment characterized by high failure rates and huge headwinds from regulators and payors, seem to be jumping in and out of therapeutic areas -- and R&D models -- like a virus desperately mutating in the face of negative selective pressure, or perhaps like the computer in War Games, hurtling toward the seemingly inevitable conclusion that the only winning move is not to play.
It's hardly surprising, then, that many patient groups feel increasingly obliged to take matters into their own hands. The Myelin Repair Foundation (MRF) now conducts its own translational research, and one cystic fibrosis patient's family was motivated to fund new therapies, channeled through the CF Foundation. Groundbreaking efforts by Jamie Heywood's exceptional initiative, PatientsLikeMe, initially focused on ALS, a condition afflicting Jamie's late brother, but has now expanded to a broader array of medical conditions. Recent articles have also discussed the role of motivated -- and affluent -- families in driving research in schizophrenia (and other psychiatric disorders), and in the treatment of a devastating degenerative disease, spinal muscular atrophy.
These patient groups are absolutely right to be striking out on their own; what's perhaps scarier is the concern that such patient commitment may not only facilitate the development of new therapies, but may also be required. The costs and uncertainties of medical product development are becoming so prohibitively high that many drug companies may strategically elect to pursue conditions for which the development path has already been relatively de-risked, and where much of the advance work has been done. Patient groups that successfully deliver this -- an explicit goal of the MRF, for example -- are likely to garner the attention of drug makers, and ultimately catalyze the development of new therapeutics.
A particularly important area for patient involvement is the regulatory requirements for new products, such as the determination of what constitutes acceptable endpoints for clinical studies. This will be especially important in the always-fraught area of identifying suitable measures for patient-reported outcomes; I suspect having invested patients, and their advocates, across the table from regulators would stimulate a particularly constructive dialogue. If patient advocacy groups are able to develop and achieve regulatory acceptance of clear clinical trial endpoints and assessment instruments, this would also significantly accelerate drug development.
To be sure, patient-supported translational efforts alone won't cut it for every condition. Consider Alzheimer's disease, an area that has received a remarkable amount of investment from both the public and private sectors, and yet has seen depressingly little progress, highlighting the wide gaps in our fundamental scientific understanding.
Another problem with relying increasingly on patient advocates is the likelihood that diseases that kill quickly (sepsis, say, or pancreatic cancer) are less likely to catalyze the formation of an active patient advocacy group than conditions that are more chronic (such as multiple sclerosis or muscular dystrophy) -- even though the opportunities for therapies to be life-altering are profound in both cases.
Finally, there's an inherent discomfort with relying on what is essentially a patronage system for medical research -- is the presence of a well-heeled sponsor truly the best criterion for an extensive translational effort? Should the development of cures depend upon the randomness of who gets sick? Also, does the real possibility of affecting medical progress paradoxically create an unspoken burden on the affluent who are sick, and their families -- will there soon be an expectation not only to manage your disease but also to figure out how to cure it?
I suspect in the end, sponsorship will be only one of several mechanisms for developing new medicines; in areas where the science seems obviously compelling (i.e. targeting PCSK9) or where the unmet need seems so high (Alzheimer's), medical products companies will continue to take risks. But to get them to venture beyond this limited comfort zone will likely require a palate of translational activities that patient advocacy groups may be uniquely positioned to deploy.
With luck, the efforts of patient advocacy groups will be successful, and ultimately provide insights and catalyze progress extending far beyond the original patients. But you need to start somewhere -- and it's hard to imagine a more powerful, compelling, and impassioned force for good than courageous patients and their determined families.