RARE Daily

The Lost Art of Letter Writing

February 21, 2020

A few weeks ago, I wrote about an effort by a group of more than 200 biopharmaceutical executives and investors to address the industry’s tanking public image.

In a letter published in Stat, the group made a public affirmation of a “new commitment to patients” and laid out a set of core principals and actions. The organizers of the effort included several prominent CEOs of rare disease drug developers. There was a notable absence of executives from large pharmaceutical companies.

While I viewed this as a sincere effort, I expressed reservations about how meaningful it would prove. The executives signed the letters as individuals and not as representatives of the companies that they lead.

I noted that while most of the points in the letter were noncontroversial, the one that stood out to me was a commitment to speak out and not tolerate companies and others who abuse their commitment to patients and abuse policies aimed at fairly rewarding innovation.

Now, they are making good on their word. STAT reported on a new letter signed by a handful of the prominent executives behind the earlier letter, which takes to task Belcher Pharmaceutical for its pricing of dehydrated alcohol.

Though the injectable substance has long been available, Belcher won U.S. Food and Drug Administration Orphan Drug designation and approval for it in 2018 to treat hypertrophic obstructive cardiomyopathy, a rare condition in which a thickening of the heart muscle impedes the flow of blood. The Orphan Drug designation conferred seven years of exclusivity to the company. With that monopoly in hand, the company raised the price for a 10-vial pack to $10,000 from $1,300.

Belcher defended its pricing by saying it invested millions of dollars in development.  Fabio Lanzieri, president of the Belcher subsidiary BPI labs told STAT that its treatment “is priced very efficiently, and will actually cost the healthcare institutions less, as they have an approved product for a less costly procedure.”

Doug Williams, Ron Cohen, John Crowley, Paul Hastings, Rachel King, Jeremy Levin, Ted Love, and John Maraganore signed the letter criticizing Belcher’s pricing decision as individuals.

“Executives at Belcher have justified this price escalation by arguing that it invested ‘multiple millions of dollars,’” the group wrote. “That said, in our view, seven years of exclusivity based on existing published research and knowledge is a misuse of the ODA, and serves as new fodder for the arguments around fair and equitable drug pricing by biopharma companies. It also potentially could compromise the ODA itself, harming the Act’s ability to delivery enormous benefits to patients with rare diseases.”

Though Belcher may seem like a safe target of their ire, industry executives have been reticent to call out bad actors. It’s refreshing to seem prominent people do this.

Chalk it up to righteous indignation or enlightened self-interest. I don’t think it matters. I applaud them for speaking out.

What it does reflect is an awareness that the best way to get rid of cherished incentives is to abuse them. The Orphan Drug Act continues to fall in the crosshairs of critics. The question now is whether these executives will be willing to punch up in defense of patients and the Orphan Drug Act.



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