Rare diseases have often been the starting point for breakthroughs in medicine and biology: Oliver Sachs, for one, has made a career out of writing about rare neurological conditions as a window to a deeper understanding of how the human brain works.Can business models built around rare diseases similarly serve as a starting point for a breakthrough in understanding or even reinventing the biopharmaceutical industry business model?
If a thought provoking forum hosted by the Institute of Medicine June 23 is any indication, the answer may be yes.
Entitled "Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies," the workshop took an in-depth look at creative approaches to drug development. We were fascinated by approaches like the sustainable non-profit vision of the Institute for OneWorld Health, the patient advocate-driven research agenda crafted by the Cystic Fibrosis Foundation, Peter Corr's new venture with Celtic Therapeutics, and the orphan-drug as blockbuster model created and maybe perfected by Genzyme. And that was just the first panel!
Rare and neglected diseases may not sound like a promising opening for pharma execs struggling to make up for looming losses of blockbuster brands. But a little perspective is useful. Most in industry would agree that the future will bring many more specialty products focusing on relatively smaller patient populations, for a whole host of reasons--scientific advances in understanding the basis of disease, regulatory concerns about safety, payor insistence of value versus standard therapy, etc. etc.
In other words, the question may not be whether Big Pharma needs to focus more on rare diseases, but rather how Big Pharma can best prepare for a world in which, in effect, every disease is rare.
That, at least, is the argument that Tim Cote, head of the Food & Drug Administration's Office of Orphan Products Development, is making. He made it in person during the Pharmaceutical Research & Manufacturers of America trade meeting earlier this year, and repeated some of the points during the IoM forum. Cote wants Big Pharma to take more interest in the orphan drug program, since, as he points out, it has been largely the province of biotech companies.
Cote also highlighted some earlier work by IoM in the area that may now be a must-read for everyone in drug development: a 2001 report on "Small Clinical Trials."
Talk about a problem most Big Pharma R&D heads haven't had to think about! In an era where the central question has been how to keep the cost of Phase III trials below the billion dollar mark, most executives probably haven't spent a lot of time wrestling with the challenges of generating a robust data package from a study involving dozens--or even fewer--patients.
But that just might be the central regulatory challenge for the personalized medicine era.
It may feel like we've gone to another world here, but that is only appropriate. As Cote pointed out, the 2001 IoM report was commissioned by the National Air & Space Administration, which wanted to understand as much as possible about the effects of zero gravity on human health. NASA, as Cote points out, has "a lot of data, but very few astronauts."
One small step....
May I suggest that the text be modified slightly? We should call "personalized medicines for rich countries and people".
ReplyDeleteNIH and now Bill and Melinda Gates Foundation are funding research on Malaria and infectious diseases. Millions have been spent and we are still analyzing. "Analysis Paralysis".
Had these diseases been in developed countries, the problem would have been solved.
Should we be putting higher emphasis on these diseases which are coming back? In the global village, diseases can traverse faster than we know.
I guess curing malaria or other infectious diseases does not pay well compared to a lifestyle disease and/or self inflicted diseases that are covered by various health plans.
Thanks to IoM for bringing more mainstream attention to these new models, and thanks to IN VIVO for blogging on them. FasterCures has been involved with many of these nonprofits for a number of years as part of a project we call TRAIN (The Redstone Acceleration and Innovation Network). As an organization concerned about accelerating progress in medical research across all diseases, we originally looked to nonprofits like Cystic Fibrosis Foundation, Multiple Myeloma Research Foundation, and Michael J. Fox Foundation for Parkinson's Research for innovations in the conduct of research that held the promise of speeding up the process. Clearly, government and industry funders are spinning their wheels in a system that costs too much and takes too long. Nonprofits may not bring as much capital to the table as those sectors, but they do a bring a unique sense of urgency, a singular focus on patients, and speed, flexibility and a willingness to take risk. We believe they can be an enormous force for positive change in the medical research establishment -- and not only in rare diseases.
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ReplyDeleteThank you both for your comments. I certainly was impressed by the creativity, vision and sophistication of the business models (and these are truly business models) employed by patient organizations and non-profits discussed during the IoM meeting.
ReplyDeleteBig Pharma clearly could learn a lot by studying those carefully.
To me, the question for industry remains: how do you get there from here?