A “Sputnik Moment”? Hopes for Renewed Drug Development With A Little Help From Our NIH Friends
Filed under: Drugs & Medical Devices, Public Health, Research
In his State of the Union Address, President Obama tried to spark the “creativity and imagination” of the American people when he proclaimed
[t]his is our generation’s Sputnik moment. Two years ago, I said that we needed to reach a level of research and development we haven’t seen since the height of the Space Race. And in a few weeks, I will be sending a budget to Congress that helps us meet that goal. We’ll invest in biomedical research, information technology, and especially clean energy technology… an investment that will strengthen our security, protect our planet, and create countless new jobs for our people.
Now I don’t know what came first — the chicken or the egg — but President Obama’s speech about investing in biomedical research and technological innovation follows National Institutes of Health (NIH) Director Francis Collins’ proposal to create a National Center for Advancing Translational Sciences (NCATS) to encourage drug discoveries and facilitate translational research in compounds overlooked by or abandoned by pharmaceutical manufacturers. So does Dr. Collins’ proposal qualify as a “Sputnik moment”?
You might not have realized it, given those never-ending TV and magazine advertisements for prescription products (whose side effects sometimes sound worse than their benefits, but that’s another blog post), but pharmaceutical manufacturers have reduced their investment in researching and developing drugs. According to the New York Times, pharmaceutical manufacturers spend over $1 billion developing a drug, with some “typically spend[ing] twice as much on marketing as on research” though that’s “a business model that is increasingly suspect.” (For a brief overview of the research and development process, click here.) The Pharmaceutical Research and Manufacturers of America (PhRMA) estimates that its members spent $45.8 billion in 2009 in research alone.
Even so, the number of drugs approved by the Food and Drug Administration (FDA) has dropped over the last 15 years and that’s not due to a lack of scientific information or higher FDA standards. In November 2010, Forbes health blogger Matthew Herper reported on the annual meeting of the American Society of Human Genetics at which Dr. Collins
implored his colleagues in genetics to work to develop new treatments for rare diseases. His point was that the NIH and the Food and Drug Administration are increasingly able to handle preclinical and early clinical drug development, and that with these first steps taken medicines are more likely to be brought to market by large pharmaceutical companies.
Mr. Herper also noted that a few organizations, such as the Cystic Fibrosis Foundation and the Multiple Myeloma Research Foundation, have taken a similar route in pushing along research until a pharmaceutical manufacturer picks up the slack. Then a month later, Arthur H. Rubenstein, dean of the University of Pennsylvania School of Medicine and chair of the NIH Translational Medicine and Therapeutics Working Group, told the Wall Street Journal Health Blog, “[b]asic science has exploded but it has not translated into benefit for the public. The question was what to do about it.”
In stepped NIH to ease, in the words of the WSJ Health Blog, the “mounting frustration that a wealth of new information about the molecular basis of diseases hasn’t produced more new therapies.” On December 7, 2010, NIH’s Scientific Management Review Board (SMRB) voted 12-1 in favor of adding NCATS to NIH. Dr. Collins notified Health and Human Services Secretary Kathleen Sebelius of the decision. Then on January 14, 2011, Secretary Sebelius sent a letter to Congress. However, the decision to add NCATS meant dismantling one of the 27 NIH centers and institutes, per the requirements of a 2006 law (“27″ is the “magic number”). The lone SMRB dissenter, Jeremy Berg, director of the National Institute of General Medical Sciences, said he was “concerned that the implications for the rest of NIH hadn’t been adequately discussed.”
And therein lies some of the controversy. NIH envisions NCATS as a link between basic discovery research and therapeutics care by:
- providing a visible, central locus for access to resources, tools, and expertise related to translational medicine;
- streamlining and improving the process of therapeutics development;
- serving as a catalyst, resource, and convener for collaborative interactions by supporting novel and innovative partnerships between multiple key stakeholders, including academia, government, industry, venture capitalists, and non-profit organizations;
- expanding the pre-competitive space by, among other things, enabling and providing incentives for greater sharing of scientific information and publication of negative results;
- supporting and strengthening translational medicine and therapeutics research, including providing access to services and resources for high-throughput screening, assay development, medicinal chemistry, and preclinical modeling;
- training translational research investigators; and
- enhancing communication among all stakeholders.
PhRMA Senior Vice President David E. Wheadon supports NCATS because
[c]ollaboration — including industry, NIH and academia — is one element driving innovation in drug development, particularly early stage — and ‘bold and ambitious’ proposals, such as Dr. Collins’, will be key to how we collectively progress in discovering novel compounds for addressing patients’ unmet medical needs….
The fact remains that biopharmaceutical research companies today and in the future will play a pivotal role: Our companies create the vast majority of new medicines from start to finish and, for the remainder, in close collaboration with academia and NIH, fulfill the critical final phase that transforms promising molecules into actual medicines for patients.
The WSJ Health Blog notes that NCATS isn’t NIH’s first foray into developing drugs. In 2009, NIH created the Therapeutics for Rare and Neglected Diseases (TRND) program for basic research on rare diseases. This early stage of drug development, WSJ Health Blog notes, is “expensive, time-consuming… prone to failure” and often not worth the effort for pharmaceutical manufacturers since the related market is small.
However, this time NIH must restructure several programs to accommodate NCATS, including the Molecular Libraries screening program, TRND, and the National Center for Research Resource’s (NCRR) Clinical and Translational Science Awards. NCATS would house all three programs, along with the in-the-works Cures Acceleration Network (a drug-development program created by the Patient Protected and Affordable Care Act).
Staff members and researchers connected with the NIH community aren’t too thrilled over the restructuring, particularly with respect to NCRR. If you visit Feedback NIH, the online forum for public commentary on NIH initiatives, you can read through the 1,100+ lengthy NCATS-related comments. You’ll also see the “Separating Fact & Fiction” post by Dr. Francis Collins, which begins:
[b]y now, many of you have read the recent New York Times article or related news coverage, about NIH’s plan to establish the National Center for Advancing Translational Sciences (NCATS).
While we are pleased that the news media have recognized NIH’s efforts as a significant development for translational research, the Times article contains some misleading statements that we would like to clarify. Those statements suggest that a much larger shakeup of NIH is underway than is actually contemplated.
So, to set the record straight, we want to share with you what we know at this point in time…
(internal links removed). The “we” includes Members of the Institute and Center Directors NCATS Working Group. The post attempts to clear up concerns about budget cuts (House Republicans have already promised to cut the discretionary spending that supports NIH), the security of existing programs, and the misconception that NCATS will be a drug company.
Despite the negative responses, Dr. Collins remains optimistic. In an interview with ScienceInsider, he emphasized that
the NIH director is called upon to look for scientific opportunities that aren’t being met and to figure out how to make them happen, and that sometimes requires moving things forward at a rapid pace, and that affects a lot of people.
And of course change is always distressing, especially if people aren’t quite sure where it’s going. So I understand the anxiety that currently exists.
But let’s wait a year and see when this has all taken shape how people feel at that point. Will they say at that point that projects or programs at NCRR got dealt a bad deal? I bet they won’t. Will they say they’re excited about the translational science opportunities that are taking shape in the form of this new center? I bet they will.
Perhaps if purse strings weren’t so tight and the healthcare reform debate was settled — or if NIH wasn’t limited to 27 centers and institutes — the creation of NCATS might not upset so many people. Yet has Dr. Collins displayed a little of that “Sputnik moment” spirit by accelerating the development of drugs for diseases and other areas overlooked by pharmaceutical manufacturers? Sure. Just don’t expect him to take us to the moon.