Trump’s disingenuous attack on the FDA highlights the precarious future of medical research funds

Trump used a survivor of a rare disease to attack the FDA. But her story actually undermines his policy proposals.

President Donald Trump addresses a joint session of Congress on Capitol Hill in Washington, Tuesday, Feb. 28, 2017, as Vice President Mike Pence and House Speaker Paul Ryan of Wis., applaud. CREDIT: Jim Lo Scalzo/Pool Image via AP
President Donald Trump addresses a joint session of Congress on Capitol Hill in Washington, Tuesday, Feb. 28, 2017, as Vice President Mike Pence and House Speaker Paul Ryan of Wis., applaud. CREDIT: Jim Lo Scalzo/Pool Image via AP

President Donald Trump has long relied on a certain kind of showboating to make specific points. He cherry-picks a few stories, sometimes ones of genuine tragedy, and parades them before an audience to craft a misleading narrative about which dire threats the country faces.

Tuesday night’s Joint Address to Congress was no exception. Attacking FDA regulations as the culprit behind slow advances in medicine, Trump pointed to one of his special guests, college student Megan Crowley.

“If we slash the restraints, not just at the FDA but across our government, then we will be blessed with far more miracles like Megan.”

Crowley suffers from Pompe disease, an extremely rare genetic disorder. She was diagnosed with the disease when she was 15 months old, and given only a few years to live. Her father, however, founded a company to find a cure. Eventually his efforts led to the enzyme replacement therapy that still treats Crowley, now a sophomore in college, and her brother, who also has the disease.

The Crowleys’ story is indeed extraordinary — and has been chronicled in a multiple media profiles, a book, and a movie starring Harrison Ford and Brendan Fraser.

Trump, however, used it as ammunition against the FDA, alleging that the department unnecessarily slows down advances like the one that saved Megan Crowley’s life.

“Our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan’s life, from reaching those in need,” Trump said. “If we slash the restraints, not just at the FDA but across our government, then we will be blessed with far more miracles like Megan.”

No one would reasonably argue against advances in medical science, particularly if they could lead to new, miraculous treatments for diseases like the treatment for Pompe disease. Trump’s emotionally-loaded shot at the FDA last night, however, was deeply misleading from beginning to end.

Slashing regulations isn’t the answer to more innovation

One of the reasons Trump’s point is attractive is that it does take a long time for drugs to make it to market. Innovation is slow, and true, ground-breaking advancements are rare.

Most of that innovation, however, happens outside the FDA — in private pharmaceutical labs, universities (sometimes supported by federal National Science Foundation grants), and federal agencies like the National Institutes of Health. Many initially promising hypotheses are rejected during the years-long pipeline. Often, breakthroughs are the result of luck, or painstaking, time-intensive trial and error.

That means funding and support for scientific research is essential if Trump truly wants to support “far more miracles like Megan.”

Trump, however, has indicated that he plans to do exactly the opposite. His proposed 2018 budget would shrink the government’s spending on basic science research by 10.5 percent.

That cut is part of an effort to pay for Trump’s request of a $54 billion increase in defense funding. From the details we have thus far, Trump’s budget would mean siphoning funding from the $31 billion NIH or the $7 billion NSF. Another likely Trump target: research aimed at addressing and understanding climate change.

Those cuts are still speculative — we don’t have the details yet on exactly how the cuts will be distributed—so they could hit some agencies more than others. Trump’s budget would also need to be approved by Congress.

But thus far, Trump has done nothing to indicate that federal funding of research is a priority, though it is often responsible for subsidizing the basic scientific research underlying many advances.

Rare diseases and the pharmaceutical industry

The squeeze on federal funding can be a particular problem for innovations on treatments for rare diseases like Pompe disease. Pharmaceutical companies are primarily driven by profits, not public good, and medical innovation is expensive. That means private companies have an incentive to develop new treatments for diseases that a lot of people have rather than so-called “orphan diseases” like Pompe’s disease.

In 1983, Congress passed the Orphan Drug Act to create financial incentives for companies to address these diseases, resulting in an uptick in innovation. The FDA and NIH concurrently began encouraging development of rare disease treatments, and focused on conducting their own clinical research. Still, however, advocacy groups argue that research into rare diseases is often left behind in the profit-driven pharmaceutical industry.

One of the key things Crowley’s father did in going outside the system was to privately raise millions to support research into the disease. Increasingly, that is the real story of how groundbreaking innovations come to pass. In one recent example, scientists funded by the viral Ice Bucket Challenge were able to discover a breakthrough for ALS, which is devastating but affects a very small percentage of the population.

The recent advance in Cystic Fibrosis treatment is yet another example. Twenty or thirty years ago, the average life expectancy for people with the rare disease was around 20 years — which itself was an improvement. In the 1950s, children with typically didn’t live to attend elementary school.

In the past few years, however, the FDA has approved two treatments for CF that attack the disease at the genetic source, rather than attempting to chase the symptoms. It’s a truly groundbreaking — and lifesaving — development.

But it, too, came about because of unorthodox private funding: In a bid to get scientists to focus on the neglected disease, the Cystic Fibrosis Foundation pioneered a model of “venture philanthropy,” raising money and funneling it to private companies to fund and accelerate research into the disease.

The strategy is not without its critics. Kalydeco and Orkambi, as the treatments for CF are branded, are indeed life-changing. But they’re also extremely expensive — over $250,000 per patient per year, blurring the line between profit and philanthropy.

There, too, we run into a problem with Trump’s promise to slash restraints “across our government.” Drugs in America are the most expensive in the world, and Trump has promised to lower their price — but the way to do so might be more regulation, not less.

One factor keeping the price of drugs down in European countries is the “reference price” system — whereby drugs with the same therapeutic effect are classed together. In the U.S., there are no regulations on pricing for similar drugs, so companies develop different drugs treating the same underlying issue.

The result is two drugs that treat the same problem, both “new,” name-brand, and expensive. It’s a lucrative strategy, and it helps drug companies keep consumer prices high.

The U.S. model also does nothing to encourage true breakthroughs. In the European system, companies who develop novel therapies are rewarded far more than those who are developing variations on the same theme — essentially, incentivizing medical miracles.

The FDA is actually pretty effective

Trump’s attack is not just misleading—it’s also fundamentally wrong. The FDA’s approval process is already reasonably fast.

In 2016, the median time for regular approvals — the main part of the process the FDA is involved in — was 10 months, according to The Washington Post. Priority approvals, on average, made it through the agency in eight months. That’s a drastic improvement over 20 years ago, when the averages were 27 months and 20 months, respectively.

And, according to former FDA director David Kessler, even Trump’s specific example — the Crowleys’ — was misleading.

Aaron Kesselheim, an associate professor of medicine at Brigham and Women’s Hospital, told Vox that contrary to Trump’s claims, the FDA is actually the fastest regulatory agency in the world.

In fact, the kind of deregulation Trump appears to favor could put people in danger without actually speeding up the process of getting new drugs to market.

Peter Thiel, who is leading Trump’s search for a new FDA head (despite making it a priority in his speech, he has yet to appoint a new leader for the agency), likes to claim that under today’s regulations, the life-saving Polio vaccine would never have been developed.

But historians point out that the lack of regulations led to The Cutter Incident, in which live vaccines were accidentally issued to thousands of children, leading to thousands of illnesses, hundreds of cases of paralysis, and 10 deaths.

“It’s exactly the production safeguards and ongoing oversight of vaccines and other products by the FDA today — both before and after approval — that keep these kinds of preventable tragedies from happening again,” Yale medical historian Jason Schwartz told Vox.

And once again, Trump’s own example belies his point. During the approval process for Lumizyme, the treatment for Pompe disease, scientists uncovered an associated risk of severe allergic reaction. In response to the discovery, researchers developed a treatment plan to combat it.

Health care, as Trump recently learned, is complicated. Pharmaceuticals and medical innovations are no exception.

That’s what makes Trump’s political showmanship particularly dangerous: His bid to slash regulations may sound like an attractive, simple answer. In reality, however, it’s unlikely to achieve the result he touts, and would more likely leave vulnerable consumers in danger.