Axios Vitals

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March 04, 2019

Good morning. It's now March, which means that it should no longer be snowing, but Mother Nature doesn't listen to me.

1 big thing: Orphan drug approvals skyrocketing

Illustration of medication bottle, pills, dollar, syringe, and price arrow

Illustration: Sarah Grillo/Axios

The past 2 years have seen a surge in the number of prescription drugs approved through a special process that was designed to help foster new treatments for rare diseases.

  • Under President Trump, the Food and Drug Administration has approved 174 drugs to treat rare diseases.
  • The approval pathway has been around for 35 years, but about 23% of all orphan approvals have come in the past 2 years.

Why it matters: Rapid scientific advances have helped fuel this rise. But the sheer volume of rare-disease approvals is also sparking a debate about whether they're too easy to obtain, or whether they give drugmakers a way to game the system.

How it works: Federal law gives extra market protection and tax credits to "orphan" drugs — those that treat conditions affecting fewer than 200,000 people. The financial incentives are there to help offset the fact that not many people will buy these drugs.

  • The rise of personalized medicine, especially through highly complex biologic drugs, has made it easier to target products toward specific rare conditions.
  • But orphan drugs are often very expensive. In 2017, the average annual cost per patient of the top 100 orphan drugs — excluding blockbusters with additional orphan uses — was $147,308, while the average cost for the top 100 non-orphan drugs was $30,708, according to EvaluatePharma.

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2. Amazon bans anti-vax content on Prime Video

Amazon is the latest tech company to crack down on content spreading false information about vaccines from its platform.

  • Buzzfeed News reported on Friday that anti-vax documentaries were available on Amazon Prime Video on Friday morning, but appeared to have been removed by the afternoon.
  • Around noon, Rep. Adam Schiff (D-Calif.) — chairman of the House Intelligence Committee — sent a letter to Amazon CEO Jeff Bezos expressing concern that the company was "surfacing and recommending products and content that discourage parents from vaccinating their children, a direct threat to public health."

The big picture: Amazon's actions follow similar ones taken by other tech companies like Google and Facebook, which have also taken steps to reduce the availability of anti-vaccine content on their platforms in response to increased public pressure.

  • Recent measles outbreaks have been attributed, in part, to reduced vaccination levels in some areas.
  • While tech companies have tried to avoid becoming content arbiters, "the issue is harder to duck when the spread of false information can lead to real-world harm," my colleagues Sara Fischer and Kia Kokalitcheva wrote last month.

3. The future drivers of even higher drug prices

There are hundreds of gene therapies in the development pipeline, raising the question of how these drugs — which could have price tags upwards of $1 million — will be paid for, and whether our system is built to handle that, the Wall Street Journal reports.

  • These gene therapies could treat or cure illnesses that currently cost the system even more over time, via long-term maintenance drugs.
  • Other drugs serve small populations and have huge benefits. Luxturna, a drug that's already approved, treats a hereditary disease that causes blindness, but has a list price of $425,000 per eye.
  • Wall Street expects that these drugs will generate billions in revenue.

But even if these costs are justified, our health care system isn't designed for large, one-time payments.

  • Medicare and Medicaid aren't allowed to pay for drugs in installments.
  • Private insurers usually only insure a patient for a few years at a time. This means they don't reap the long-term savings from these therapies.

4. The poop wars are on

Macaroons in the shape of the poop emoji

Photo: Steve Russell/Toronto Star via Getty Images

There is a new battle brewing among doctors, patient advocates and pharma companies over — believe it or not — human excrement.

  • More specifically, the fight is over fecal microbiota transplants, a remarkably effective treatment for the bacterial infection Clostridioides difficile, the New York Times reports.
  • The battle is over whether the treatment — which involves transplanting healthy fecal matter into the bowels of patients suffering from the infection — should be classified as a drug or as a procedure akin to organ, tissue and blood transplants.

The FDA is expected to make a final decision soon, although in 2013 it made a draft decision to regulate the procedure as a drug.

  • The market for drug-based treatments for C. diff is expected to hit $1.7 billion by 2026, according to GlobalData.
  • Following the success of the procedure, scientists are currently trying to come up with similar treatments for disorders like obesity, autism, ulcerative colitis, and Alzheimer’s and Parkinson’s diseases.
  • Investors are pouring money into such endeavors.

One major concern for critics: Treating the therapy as a drug will give it a 12-year monopoly period upon approval.

  • They say this could slow innovation and cause patients who can't afford the procedure to attempt to replicate it at home.
  • And, of course, there's fear that this would lead to high prices for stool transplants.
  • Drug companies say that regulating the procedure as a drug will help ensure its safety and efficacy.

While you were weekending

  • States are rethinking their policy approaches to lowering prescription drug prices after the Supreme Court refused to hear an appeal of a ruling that struck down an ambitious Maryland law, StatNews reports.
  • Beth Israel Deaconess Medical Center and Lahey Health have officially merged into Beth Israel Lahey Health, Modern Healthcare reports. The deal includes a 7-year price cap.
  • As more women use the VA for health care, the department has responded by improving its women's health services, although some say there's more work to be done, the WSJ reports.

Have a great week. Send any feedback or tips to [email protected].