First Opinion readers respond to essays on right to try lies, pharma execs, and more

First Opinion is STAT’s platform for interesting, illuminating, and maybe even provocative articles about the life sciences writ large, written by biotech insiders, health care workers, researchers, and others.

To encourage robust, good-faith discussion about issues raised in First Opinion essays, STAT publishes selected Letters to the Editor received in response to them. You can submit a Letter to the Editor here, or find the submission form at the end of any First Opinion essay.

“Give pharmaceutical execs the benefit of doubt — but they need to work for it,” by Fred D. Ledley

The author believes “the pharmaceutical industry can develop products that are affordable, universally available…without compromising their profits” and yet also contends that industry executives are not the monocled barons the media makes them out to be. How can both be true?

If the utopian vision Fred Ledley describes is so easily attainable, surely it is the malaise and greed of industry that prevents it. But, as he is quick to point out, that simply is not his experience — or a reality experienced by thousands of scientists, entrepreneurs, investors and yes, corporate executives, dedicated to bringing medicines to patients.

Instead of trying to square this circle, the commentary adds to a long line of other “observers” who insist there is a path to having it all. For example, the author points to his research, which concludes that “drug price negotiation provisions of the Inflation Reduction Act (IRA) could have little or no impact on the number of drug approvals.”

Several other studies point to an opposite conclusion, including newly released data showing dozens of programs paused or stopped because of the IRA. That has certainly been the experience of those with ovarian cancer or those suffering from Stargardt’s disease.

A different report by Ledley and a colleague mistakenly concludes that cutting current drug prices won’t impact the funding for the early-stage companies, to which he attributes the bulk of new medicine development. This is a faulty argument that’s been seen time and again and it is a particularly tough sell now, when on the same day STAT published Ledley’s commentary it covered how sluggish the biotech VC scene is this year. A headline in Thursday’s Wall Street Journal similarly captured the trickle-down effects: “Big Pharma Cuts R&D, Sending Shudders Through Industry.”

As much as people want to demonize large pharma and champion small biopharma companies, that’s not how the ecosystem works. And this symbiotic relationship isn’t just inseverable, it is invaluable. It reflects decades, if not a century, of specialization of labor and efficient allocation of capital.

But Ledley writes that breaking these ties, and navigating hundreds of billions of dollars in lost revenue can be done if industry is “held accountable …” and by “executing effective strategic management and investment practices.”

If only it was so easy. The reality is that economics, like life, is governed by tradeoffs. We cannot have our cake and eat it too. Is it really that hard for all of us to admit that?

— John Stanford, Incubate Coalition

“Trump gave patients a ‘right to try’. It hasn’t helped them,” by Alison Bateman-House and Holly Fernandez Lynch

At the Republican National Convention, Donald Trump claimed that “Right to Try” had saved hundreds of thousands of lives. People living with terminal diseases that cannot yet be treated should not be lied to. It is sad that they are being used as pawns by unscrupulous politicians.

— Kim Meyers

“Mark Cuban’s company won’t fix drug costs, but it can still help rectify America’s drug shortages,” by Ezekiel J. Emanuel and John Connolly

I’m not sure if there’s a medical procedure code for “tall poppy syndrome” — the act of cutting down someone who has become successful or noteworthy — or not but this article is the embodiment of it. The authors create a ridiculously high standard that they argue Mark Cuban needs to achieve in order to “fix costs” while ignoring the fact that he is changing the conversation about the pricing of generic medications to payers, be they individuals, companies, or the government.

Roughly 90% of people have insurance and most of the time it works well to cover medications, especially common generics. However, if you find yourself among the 27 million uninsured Americans or the tens of millions in a high deductible plan, you may encounter a situation where you must pay cash for a generic medication. Many surveys have shown that this unexpected type of expense can be difficult. But what makes Cost Plus so interesting (and often revered) is that consumers know they’re not getting ripped off by a bunch of excessive markups. Who would buy milk at a store if marked up randomly by $40 when the store across the street priced it as usual?

If the country’s entire pharmacy-based reimbursement system went to Cost Plus for the roughly 6 billion generic prescriptions a year, it wouldn’t save that much if set at a price point of 10% to 20% over the true, net wholesale price (to the pharmacy). But the pharmacy pricing roulette game that many people experience is really frustrating and has no doubt led to early deaths and complications from lack of adherence.

I applaud Cuban for his leadership and risk-taking here. My advice is to ignore the critics who appear to be envious of your ability to capture so much attention and nudge the markets in an exciting new direction.

— Stephen Buck, CancerSurvivalRates.com

“PBMs aren’t opening access to lower-cost biosimilars. Reform is needed now,” by Juliana M. Reed

As Congress, the Federal Trade Commission, and advocates from across the health care system have long known, pharmacy benefit managers are not doing nearly enough to ensure that biosimilars are accessible and affordable for patients.

This is a serious, and sometimes life-threatening, problem for the millions of Americans living with gastrointestinal diseases, such as Crohn’s disease or ulcerative colitis. While biosimilar medications like infliximab are safe, effective, and clinically appropriate for many people, the pharmaceutical company rebates secured by PBMs for these drugs do not translate to reduced costs for the people who need them. Instead, these rebates are lining the pockets of PBMs, while patients pay exorbitant prices out-of-pocket for necessary medications.

These rebates don’t factor into the cost of biosimilars purchased by independent practices, meaning physicians lose money for each dose they provide to patients. Just to stay afloat, at a time when costs are rising across the board, practices are often forced to switch patients to other drugs (thereby starting a fresh round of prior authorization approvals from the patients’ insurance company) or refer them to hospitals or standalone infusion centers (often owned by private payers) — both settings where costs are higher and the continuity of care is disrupted.

As policymakers in Washington rightly consider putting up guardrails around PBMs, it is critical for them to regulate dangerous white bagging mandates, delink the list price of a biosimilar drug from PBMs’ compensation, and ensure that patient access to necessary infusions is not disrupted every time an insurer updates its formulary. Patient access and health depend on it.

— Dr. Maria T. Abreu, president of the American Gastroenterological Association, director of the Crohn’s & Colitis Center at the University of Miami Health System, and professor of medicine, microbiology and immunology at the University of Miami Miller School of Medicine.