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Getting biomedical advances from the research pipeline to the marketplace is an important step in meeting the need for new drugs, according to actor – and Parkinson’s patient – Michael J. Fox.
Fox addressed translational research and its implications during his keynote speech at Bio 2007 in Boston recently. Fox, who was diagnosed with Parkinson’s disease in 2000 and has established The Michael J. Fox Foundation for Parkinson’s Research, said translational research requires a “new kind of stakeholder.”
He said the foundation hears from smaller companies involved in early-stage preclinical work that investors have “little appetite for the high-risk studies necessary to really determine whether an idea has value or not.”
“It seems that more and more industry capital is focused on repurposing ideas and compounds that have been kicking around for years,” Fox said. “Again, I get it, but here’s a question: Who’s funding genuine innovation today?”
The foundation has funded more than $90 million of research either directly or through partnerships, supporting more than 300 Parkinson's research projects throughout the world. Last month, the foundation launched Biomarkers 2007, a two-year, $2-million funding program for the discovery of an objective biomarker of Parkinson's disease. The program is in its third funding round targeted at biomarkers, which the Parkinson's field currently lacks.
“I know we can find common ground where maximizing the effectiveness of an idea complements your need to maximize the value of your assets,” Fox said.
The following is a transcript of Michael J. Fox’s keynote address:
Thanks, Jim, for that gracious introduction, and for inviting me here today. I’d also like to thank the folks at Genzyme for sponsoring this luncheon. It’s an honor to speak to this group. I’m grateful for the opportunity to share my thoughts as both a patient and the founder of a disease research foundation.
I thought I’d open by passing along a statistic Mike Milken shared with me a couple of weeks ago:
There are 30,000 known human diseases; but we have treatments for only 10,000.
I’d be willing to make a couple of bets about the people in this room. First, I’d wager we’d all agree on the massive unmet need for new treatments for disease. There’s still a lot of work to be done - but that also means there are a lot of opportunities.
The thousands of people at this meeting represent the major stakeholders in the therapeutics development process. We run the gamut of public and private funders, researchers, patients, pharma and biotechs.
Here’s a second bet: Every single one of us would say we’re working as urgently as we can to steer our resources toward developing new therapies. It’s just not that easy to do.
In fact, you could look at the whole drug development pipeline as a big game of Texas Hold ‘Em.
You’re dealt a few cards, and you have to make a bet based on how promising are your hand looks. With each new card you’re dealt, you get a little more information - and of course, you also have to put more money on the table. Big money. And the stakes get exponentially higher with each round.
The stakes get so high, in fact, that the game can take on a life of its own. But of course that depends on your appetite for risk and the potential pay-off. (By the way, how obvious is it that I just got back from Vegas?)
But listen - the pot is significant - and it’s hard to know which hands are worthy of doubling down on. But all this boils down to... How do we define winning?
Answering this question may be one of our greatest challenges. What does achieving success - winning - mean to each of us and our organizations?
The drug development process is a constantly changing environment. Access to capital waxes and wanes, and industry’s risk appetite shifts. All of us do the best we can with the cards we’re dealt, placing our bets and trying to maximize value as we see fit.
I’d like to take a crack at explaining how I define success in the drug development process.
Let’s start at the beginning of the “research pipeline,” where you have the government’s huge investment in discovery, funded mainly through NIH grants. In this system, success means more NIH grants - ongoing funding for a lab.
NIH grantees are typically academic scientists - brilliant, well-intentioned researchers. But to guarantee additional funding, they must publish and secure tenure. And a publish-or-perish system encourages exploring elegant questions whose answers are intellectually interesting, but often low-risk. It favors basic research over clinical, a far cry from the out-of-the-box flyers that are the hallmark of truly innovative results: translating discovery into new treatments.
American taxpayers are funding the greatest discovery engine in the world, yet we fail to provide incentives for our scientists to convert their relevant findings into improvements in human health.
At the other end of the research pipeline, the commercialization end, there’s the even larger pool of capital controlled by pharma and biotech companies and their backers.
We understand that a company’s measure of “success is shareholder profit. To many companies, this means “IP positions or “patentable assets offering potential markets in the billions of dollars.
Look: I get it. That’s why we have hired business leaders to run our Foundation. We’re realistic about how for-profit companies are incented to compete.
But we hear from small companies involved in the earliest-stage pre-clinical work that investors have little appetite for the high-risk studies necessary to really determine whether an idea has value or not. It seems that more and more industry capital is focused on repurposing ideas and compounds that have been kicking around for years. Again, I get it... but here’s a question...Who’s funding genuine innovation today?
Levodopa is the gold-standard treatment for Parkinson’s, the disease I live with. Don’t get me wrong...I’m grateful for my meds. L-dopa helps PD patients manage some of the symptoms, but not all... and for only some period of time. I may be luckier than most - I’ve been reasonably well-treated with available drugs for over 15 years. But it’s a little frustrating that the best drug we’ve got is one that’s been around for 40 years. 40 years! And away from some tweaking here and there, PD patients are still faced with tough choices and no current treatment that can actually modify the course of the disease. Do we really need another dopamine agonist?
But, hey, credit where credit is due - there are a lot of innovations happening. One that comes to mind is anti-depressants for dogs. Which makes things a little better for me -I mean, my symptoms aren’t improving, but at least I have some options to help my dog deal with it.
So let’s add it all up... Between government and commercial investments in medical research, we’re talking a hundred billion dollars a year pumped into the drug discovery process. That is a boatload of money. Yet there is much talk about where to find more money for these endeavors.
I’d argue that filling the unmet needs of patients across all diseases isn’t about more money - it’s about spending the money more effectively.
And though it’s modest in comparison, add to that $100 billion another couple of billion in private philanthropic capital, donated largely by patients themselves. Here’s a group with a compelling interest in speeding up the development of new therapies.
Yet these funders, too, could raise their own bar for giving, which is too often dominated by the “grateful patient effect- a predisposition to fund one’s own doctors out of gratitude, loyalty or a lack of other perceived options.
The tough truth is that the research pipeline - the therapeutic development system - breaks down in the exact spot where we need it to work the hardest: the place where it should be strategically driving the higher-risk, higher-reward work of converting basic discoveries into new therapies. We are failing to incent the high-risk innovators who could really make a significant difference more quickly.
Looking around this room, filled with an incredible reserve of brainpower, scientific prowess, and excellent intentions, I’ve got to ask: How can we get this right?
Because I can’t help thinking about the cost of failing to get it right. The importance of these outcomes can’t be measured only on a corporate balance sheet or by being featured on the cover of Science or Nature. You have to measure it in patients’ lives.
I’m not naive. I’m not wringing my hands, I’m not here to plea. I appreciate that my being here today talking to you from the patient perspective - by itself - doesn’t change the factors that go into your investment decisions. And I’m not expecting a groundswell of newfound interest in PD research.
But it seems to me that regardless of how each of us separately defines a winning hand, we can all achieve success by re-deploying our resources creatively and strategically in ways that benefit everyone.
Our Foundation, and a handful of like-minded, proactive, disease groups, stand ready to work with you. But I’m sure you understand that we do measure the return on our investment differently from other stakeholders.
I’m here today to make the point that we think translation requires a new kind of stakeholder.
We hope to help bridge the gap between value and effectiveness...because that creates a win-win for all participants. And we’re focused on finding ways to change the game so that you get to turn some of the cards over earlier, when the antes are smaller and you can afford to fold without losing your shirt.
Circling back to the poker table... We’re in a high- stakes game and - although you don’t often see this in Vegas - our Foundation is working to improve the players’ odds. For patients like me, the risk is folding too early and not having a chance to win the big prize. But the goal here is for everybody to win.
We want to work with companies that have exciting ideas at various stages of development...from target validation to phase II. We’d rather put $200,000 in the pot early on if it will help answer crucial questions about drug targets. This, in turn, may reduce the risk and increase the odds of an industry player investing $10 million in a promising treatment down the line.
We’re continually looking for new ways to custom-fit promising ideas with whatever resources we can provide - financial, scientific, collaborative or otherwise. This is better for you, and it can also benefit patients.
- In 2006, across seven different grant programs, we made a total of $7.5 million in commitments to 16 companies - a number we expect to grow this year.
- Our Foundation has funded industry since we awarded our first grants in 2001... but just last year we took it to the next level. We launched the Therapeutics Development Initiative, our first program open exclusively to industry researchers, just to hammer home the point that we want and need to work with you.
I’m eager to hear what else we can to do to convince you how serious we are about working with you. Just let me know and we’ll do it.
I’m an optimist. I know we can find common ground where maximizing the effectiveness of an idea complements your need to maximize the value of your assets.
I truly believe that if we spur innovation... work together to reduce the risk of early-stage ideas... and seed the drug development pipeline with the next generation of therapies, we can assure every stakeholder of transformative results - high returns on financial capital and on human health.
When all’s said and done, we’re thrilled that you’re out there doing the work that you’re doing. Thanks for being here, and thank you for having me here today. Thank you.
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