Breaking out of the 'academic comfort zone' for higher returns on medical research philanthropy

Last week I spoke on a couple of panels at the BIO Global Convention in San Diego. At one point I found myself seated next to Joshua Boger, the CEO of Vertex Pharmaceuticals, which recently announced the development of a new drug for cystic fibrosis. He told the audience, in as many words, that without the Cystic Fibrosis Foundation (CFF) as a 10-year funding partner, this $70 million therapy would not exist. That's a head-turning statement to say the least, both for people with CF and for Vertex's shareholders.

Yet The Michael J. Fox Foundation and CFF were among only a few medical research philanthropies who chose to come and be counted among BIO's 20,000-plus attendees. I fielded huge curiosity about how industry could best obtain foundation funding to pursue "high risk/high reward" work. But I heard from countless industry reps that MJFF and CFF are the exception, not the rule, in partnering with for-profits.

Why the low penetration of this approach in spite of the obvious and tangible benefits and the efforts of groups like Michael Milken's FasterCures to build momentum? Maybe it's got something to do with the idea, eloquently refuted in a recent Boston Globe Op-Ed by David A. Shaywitz and Dennis A. Ausiello, that academic researchers who partner with industry are "intrinsically tainted." Maybe other factors are at work. But in disease research funding circles, there is endless talk about the broken medical research enterprise and the pressing need for new funding paradigms. That's why over the past 10 years (starting with Bob Beall's efforts at CFF), a handful of foundations have explored different ways of driving toward cures -- and evidence continues to accumulate that funding companies could be a very big wave to ride.

I can speak only for MJFF's experience, but I think it's pretty compelling. With 21 active industry partnerships at the time of this writing, we're funding the pursuit of multiple disease-modifying PD treatments that likely otherwise would have been shelved for lack of capital. Our investments are frequently leveraged, with companies attracting both external and internal follow-on funding once we've signed on. We routinely seek out the opinions of industry decision-makers -- both on the science and business development sides -- to inform our opinion of the field. Their expertise on drug development is a force multiplier for interpretable outcomes from our academic and industry investments alike.

No one advocates a complete shift away from funding academic research. But medical research philanthropy, funded largely by patients themselves, is uniquely motivated to bridge the gap between discoveries in university labs, drug development efforts within companies, and therapies in the clinic. And to do that effectively, we must work as intellectual and financial partners with industry, from tiny biotechs to Big Pharma.

So what will it take to make this happen on a larger scale? I'd argue the field needs two things in greater measure: cross-talk among leaders and boards of different foundations, and honesty with donors about what their contributions are achieving.

Private philanthropists need to break out of the collective comfort zone of a traditional, "academics only" approach. Identifying a prize-winning researcher or a celebrated institution to support is simply not an effective way to get to the life-transforming therapies that are so desperately needed. But that wouldn't be obvious to an individual donor. It doesn't become clear until you get a comprehensive view of the field -- the bird's-eye-view of an MJFF or a CFF, reviewing hundreds of grant proposals in our respective indications each year, working day in and day out with a who's-who of academic and industry scientists toiling in the field.

So MJFF forges ahead with the experiment. It's early days yet, and we're still part of a lonely minority. But we believe the payoff in terms of field-advancing results is likely to convince far more organizations -- and their donors -- that this is an approach foundations can't afford not to take.

Comments

[indigogo]

I'm glad Katie is at the MJFF helm; innovative thinking is our only way out, and a cure surely possible one day. I just wish more attention was being given to funding innovative models of care for current PD patients.

While MJFF searches for a cure, money should also be found for education and awareness of the new developments in patient care and health management. That"s difficult. Katie Hood"s job to find a cure for PD is hard, but raising the money is relatively easy. Cures sell. Care is another matter.

The front line warriors in the battle to keep patients going are the doctors and allied health professionals who see people suffering in their clinics everyday. Clinicians are developing new models of PD care; they know that an integrated program of drugs, exercise, and a variety of speech, occupational, physical and mental health therapies can improve the lives of most of those living with PD. I know mine has.

Unfortunately, this groundbreaking work is difficult to fund in the chase for medical research dollars. It"s just not sexy. But it is important, and it"s available now.

A million-plus people living with Parkinson"s in the U.S. could have a better quality of life while waiting for (or joining a clinical trial), MJFF to find the answer; the future is bright. We need a greater commitment to improving the lives of those of us living with PD today, a commitment to the innovations in both cure and care.

[lclilly]

Re: tompoe

Can you please define -
1. the secret nonproprietary automated clinical research data collection system that would do a lot of things, chief among them, lower the cost of pharmaceutical research by 70% or more.
2. the standard approach to the conduct and management of human clinical trials

You said: "I'll bet the patients would appreciate knowing their medicine was supported by actual reliable and accurate data."
Very true, this quest for honesty has been active for a number of years. A venture should be planned with the patients at the table from the beginning. There are actually patient experts on GDNF , for example.

We woud all learn alot -
lclilly

[tompoe]

Interesting post by the author. She obviously does not know about the secret nonproprietary automated clinical research data collection system that would do a lot of things, chief among them, lower the cost of pharmaceutical research by 70% or more.

This secret system would ensure that the "data speaks for itself", providing reliable and accurate data. Companies, big and small, would use the standard approach to the conduct and management of human clinical trials. It would enable the government(s) to protect their populations and avoid needless deaths that number over 100,000 in U.S. hospitals alone.

It would also have a dramatic impact on the pharmaceutical industry as it exists, today. Companies would have to opt for lower profit margins over a longer period of time, namely, years. Of course, such a change would improve their overall profit structure, but there would be an increase in competition as well.

Maybe the private philanthropists should hedge their bets, and limit their participation to opportunities that agree to use the secret system. I'll bet the patients would appreciate knowing their medicine was supported by actual reliable and accurate data.

There is only one downside to this approach. No one would own the nonproprietary automated clinical research data collection system. No royalties would be paid to an owner, and no owner could control what medicines are, or are not, made available to the world.