Once again officials within the FDA are proposing to put other interests over the needs of dying patients, predominantly children.
For more than three decades a group of FDA civil servants aligned with Ralph Nader’s Public Citizen has fought accelerated review of breakthrough drugs for devastating diseases. In the mid-1980s AIDS activists were the first to loosen the grip of this unholy alliance. Without the persistent campaigns of ACT UP and other activists, the FDA would not have approved the first AIDS treatment, AZT, in 1987. More significantly, the FDA’s accelerated approval of AZT stimulated the biotechnology industry to invest in better drugs; these drugs have not cured the disease, but they have saved millions of lives and made the disease manageable for many people.
The FDA’s old guard reacted to this success by trying to find a way to declare AIDS unique, so that it could continue the risk-averse approval process it preferred—a process more devoted to data collection than helping the desperately ill. When I was general counsel at the Department of Health and Human Services, the Council on Competitiveness executive director David McIntosh and I convinced FDA commissioner David Kessler that there was no moral difference between a patient dying of AIDS and a patient dying of any other disease for which there was no treatment. With that principle established, in 1992 the FDA promulgated its first regulations to allow for accelerated drug approval more broadly.
The frontline employees of the FDA tried to undermine the implementation of the new regulations, but they were overruled time and time again by their supervisor, David Finbloom. His courage and respect for the accelerated approval regulations saved the lives of millions of people with multiple sclerosis, cystic fibrosis, lysosomal storage diseases, and many other fatal conditions.
When Finbloom died in the late 1990s, the old guard led a successful retreat on rare diseases. The FDA responded to competing breakthrough products for Fabry disease and muscular dystrophy by slowing down approvals in order to create a fictitious “level playing field” that would allow them to pick the best in class. Excessive clinical trial requirements and blatant disregard for its own conflict-of-interest rules slowed down approvals even further.
Perhaps there is hope to speed things up once more. The FDA’s new commissioner, Scott Gottlieb, has a record of being more sympathetic to the ideas of David Finbloom than those of Ralph Nader. The problem is that the old guard has created a sneaky tactic to try to box Commissioner Gottlieb into policies whose inevitable result will be avoidable suffering and deaths.
The FDA and its European counterpart are proposing a new regulatory regime for Gaucher’s disease, a rare illness in which an enzyme that cleans out toxins is missing from cells. That regime would explicitly seek to stifle competition and turn most clinical trial design decisions over to the two regulatory agencies. The stunning thing about this announcement is that it is more than a quarter of a century late. In 1991 the FDA approved a naturally derived enzyme missing in Gaucher’s patients as the first treatment. It subsequently approved three recombinant forms of that enzyme and two small molecules with a different mechanism of action. Now, with the problem for patients being primarily one of cost not therapeutic benefit, the FDA wants to “help.”
Far-left politics, not science or patient needs, is driving this proposal. In the long term, gene editing and gene therapy may create better and cheaper treatments for Gaucher’s disease, but there are no promising treatments on the immediate horizon. The FDA and its European counterpart cynically picked Gaucher’s disease solely because it has become a rallying point for left-wingers who want to control drug pricing. The plan, then, is for the FDA old guard to jam this heavy-handed proposal through for a politically vulnerable disease in order to set a precedent for applying progress-killing principles to all experimental treatments for rare diseases.
The Europeans have already released their version of the proposal, and the FDA has indicated that it will be releasing its version for public comment in the next few months. The mere release of this document will make things worse. As happened during the “Hillarycare” debate in 1993-1994, venture capitalists will hold back funding and biotech companies will shelve programs until the regulatory landscape becomes clear.
Contrary to the expectations of those who opposed Commissioner Gottlieb’s nomination, he has slapped down pharmaceutical companies that needed to be slapped down. He has also made significant progress toward cleaning up the Obama administration’s generic drug review backlog, a backlog that unnecessarily aggravated health care inflation.
A typical FDA commissioner would let this Gaucher’s proposal become the subject of public comment, then put a finger in the air to decide whether to push back against the agency’s hardliners. A great commissioner would shut down the agency’s disingenuous power-grab now, then start a genuine discussion about how the agency can best help the desperate and dying.
Michael Astrue is a former general counsel for the Department of Health and Human Services and biotechnology CEO. He sits on the board of a rare disease advocacy organization, but the views expressed here are entirely his own.
