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Título: A Bipartisan Victory for Medical Research in Congress
Fecha: 16-07-2015
With exquisite timing, House Republicans last week stepped into a public-relations debacle. The House was working to pass an appropriations bill for the Department of the Interior, but the longstanding bipartisan process on spending bills has been shattered, meaning that there would be no Democratic votes for the highly partisan bill. That, in turn, meant that Republican leaders had to corral the vast majority of their own members, including many who reflexively vote against spending bills. And that meant accommodating Southern members who wanted to have more opportunities to showcase Confederate flags on federal sites. That proved so controversial that the bill ultimately had to be shelved. Not surprisingly, the stirring story of South Carolina facing history and bringing down its Confederate flag on its state capitol grounds competed for media attention with the embarrassing story in the U.S. House of Representatives.

But almost lost in the firestorm was another story, at the opposite end of the continuum. On Friday, the House passed by overwhelming margins the 21st Century Cures Act. It beat back a poison-pill amendment by Republican Representative Dave Brat of Virginia. Some Democrats withdrew their own amendments that might have complicated passage after a promise by House Energy and Commerce Chairman Fred Upton to find a way to accommodate their concerns. It was a remarkable spectacle of bipartisan cooperation.
The 21st Century Cures Act had earlier passed through the committee on a 51-0 vote. Almost nothing except the most innocuous non-binding resolution gets unanimity in the sharply polarized Congress. But this bill is a substantive and substantial one, making the committee action truly notable. It happened because Chairman Upton and his Democratic partner on the bill, Colorado’s Diana DeGette, worked for months to find common ground, and did it the right way—through deliberation and give-and-take, and by bringing in other committee members, keeping them in the loop, and incorporating their ideas. And it happened because the substantive area—expediting research and development on debilitating diseases, and making it easier to get important treatments to the patients who need them—was a natural for bipartisan cooperation.
Almost nothing except the most innocuous non-binding resolution gets unanimity in the sharply polarized Congress. But this bill is a substantive and substantial one.
The Act does many things, including making research collaborations easier; promoting therapies like biomarkers to enhance personalized drug treatments, targeted at individuals and not just broadly at diseases; reforming and streamlining clinical trials and making it less challenging and expensive for companies to bring drugs to market; creating incentives for developing drugs for uncommon but deadly diseases; creating an Innovation Fund to encourage young scientists to do path-breaking research; and putting more money into both the National Institutes of Health and the Food and Drug Administration to make these innovations work.

But getting to “yes” was not so easy. First, it required a bundle of money—at a time when federal dollars are particularly difficult to come by. Many Republicans do not want to expand government of any variety, and the zeitgeist is to slash more, not spend more. The Brat Amendment reflected that view, and would have cut the innovations in the Act off at the knees. Second, the Act tries to reduce the daunting costs of bringing promising treatments through the lengthy and difficult process of laboratory work, animal trials, and several phases of human clinical trials—something that is opposed by many people who fear that any shortcuts could be dangerous, and who do not want to do anything that might reward pharmaceutical companies. Thus, such luminaries as David Kessler, the former head of the FDA, opposed the bill.
I, on the other hand, rooted for it enthusiastically. First, any effort of this sort—serious and important legislation to improve the life and health of Americans—that tries and succeeds at finding that common ground through a model legislative process needs to be encouraged and applauded. Second, there are few more cost-effective ways to spend taxpayer dollars than on medical and scientific research. That approach is now more important than ever, when the cuts in research dollars and the assaults on federal agencies have taken away many of the incentives for young, brilliant scientists to go into medical research. Third, the need to enhance research in areas like Alzheimer’s is especially acute and urgent. Newt Gingrich has pointed out that if we do not make progress combatting the scourge of Alzheimer’s, the costs to the country as Baby Boomers age will be $20 trillion or more—which does not include the human costs, just the money to care for patients.
A change in the process that might mean that a few more promising treatments do not get abandoned prematurely.
Third, I have seen from the inside the costs and difficulties of bringing novel treatments for debilitating diseases to market. For the past several years, I have been on the board of UCB, a biopharmaceutical company based in Brussels.I have no stock in the company and don't stand to profit directly from these regulatory changes, but they would certainly help UCB and companies like it expedite beneficial drugs to market. UCB specializes in finding treatments and cures for diseases like epilepsy, rheumatoid arthritis, Crohn’s, Parkinson’s, lupus, and osteoporosis. In our scientific discussions, we have heard about hundreds of promising molecules that have to be narrowed down to dozens and then to a handful that can move through proof-of-concept and first-in-man studies, and then the three necessary stages of clinical trials. Because of the costs, which can go up to hundreds of millions or more, many very promising treatments end up abandoned. Others move forward but cannot reach the finish line. Of every 5,000 to 10,000 promising treatments, one or two end up making it all the way through to actually becoming drugs on the marketplace. A change in the process might mean that a few more promising treatments do not get abandoned prematurely, or that might change the ratio of 1 in 5,000 to 3 in 5,000, could make a huge difference in the lives of people with awful diseases.
Of course, no one—including the authors and co-sponsors of the 21st Century Cures Act—wants to reduce the costs or streamline the process in a way that means ineffectual or dangerous treatments make it through. But there are ways in the bill to make it less expensive and less daunting, which will also mean that many people suffering from awful diseases will have treatments available earlier than they are now. And, critically, the Act provides more money to an underfunded FDA to make sure the streamlining can work well.
The overwhelming House passage does not mean that the 21st Century Cures Act will inevitably become law. The Senate has to act—not always easy for anything in the Senate—and overcome the same political hurdles that faced the House. If we are all lucky, the Senate will do so, and do it in the same, model bipartisan way the House did. And if we are especially lucky, this model will work next on the desperately needed reform of the mental-health system.
Fuente: endovascular.es

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