Yesterday, I posted a response to the portion of the President’s February 28 address to Congress where he used the example of a young woman suffering from Pompe Disease, and her father’s heroic efforts to develop a treatment for the disease, as fodder for an attack on the FDA. I was motivated by the striking disconnect between the problem – developing effective treatments for rare diseases and making them accessible to patients – and the President’s proposed solution (gut the FDA). It was as if he had a solution in mind and then went looking for a dramatic problem that could justify his solution. The pieces just did not fit.
Since the President’s speech, others have dug deeper into this dubious connection between problem and solution, questioning the truth of the President’s claim that government bureaucracy was responsible for keeping new treatments from reaching patients who needed them. Their reports demonstrate that, contrary to what the President stated, the FDA process is not the problem in getting accessible and effective treatments to patients with rare diseases:
Herper, M. (2017, March 1). Would Trump’s FDA deregulation create an age of miracles? Don’t bet on it. Forbes. Retrieved from https://www.forbes.com/sites/matthewherper/2017/03/01/would-trumps-fda-deregulation-create-an-age-of-miracles-dont-bet-on-it/#4c1a81443883
Huron, J. (2017, March 1). NORD issues statement on President Trump’s Address to Congress. National Organization for Rare Disorders. Retrieved from https://rarediseases.org/nord-issues-statement-president-trump-address-congress/
Johnson, C. (2017, Feb. 28). The backstory behind the rare disease patient Trump highlighted to Congress. The Washington Post. Retrieved from https://www.washingtonpost.com/politics/2017/live-updates/trump-white-house/real-time-fact-checking-and-analysis-of-trumps-address-to-congress/the-backstory-behind-the-rare-disease-patient-trump-highlighted-to-congress/?utm_term=.0c32e803b570
Raymond, L. (2017, March 1). Trump’s disingenuous attack on the FDA highlights the precarious future of medical research funds. ThinkProgress. Retrieved from https://thinkprogress.org/if-trump-really-cared-about-medical-advances-hed-increase-funding-not-slash-regulations-e59a78e30647#.ri1r1dk7f
What most of these authors did conclude, however, as I concluded yesterday, is that various other features of the President’s agenda may pose more of a danger to patients with rare diseases than FDA procedures. Health care policy is complicated. People’s health, welfare, and lives are at stake. We should be wary of the oversimplification, linear thinking, and easy scapegoating that serve a purely political agenda.