A FDA panel voted against approving MDMA (ecstasy) for post-traumatic stress disorder. Putting aside the specifics of the case, I was vexed by this statement on innovation from one of the experts voting no:

“I absolutely agree that we need new and better treatments for PTSD,” said Paul Holtzheimer, deputy director for research at the National Center for PTSD, a panelist who voted no on the question of whether the benefits of MDMA-therapy outweighed the risks.

“However, I also note that premature introduction of a treatment can actually stifle development, stifle implementation and lead to premature adoption of treatments that are either not completely known to be safe, not fully effective or not being used at their optimal efficacy,” he added.

A textbook example of making the perfect the enemy of the good. But the problem is even worse. Holtzheimer seems to think that treatments spring from the lab perfectly formed like Athena springing from the brow of Zeus. Indeed, Holtzheimer suggests that treatments should be kept in the lab until they are perfect. News flash: there are no perfect treatments–no drug or device in use today is completely known to be safe, fully effective, and used at its optimal efficacy. Not one. If we follow Holtzheimer’s counsel, we will never approve a new drug.

Innovation is a dynamic process; success rarely comes on the first attempt. The key to innovation is continuous refinement and improvement. A firm with sales gains greater resources to invest in further research and development. Additionally, they benefit from customer feedback, which provides valuable insights for enhancing their products and processes. Learning by doing requires doing. But if imperfect treatments are never approved, scientists often don’t return to the lab to refine and improve them. Instead, the project dies. Thus, when considering innovation today, it’s essential to think about not only the current state of technology but also about the entire trajectory of development. A treatment that’s marginally better today may be much better tomorrow.

Small steps toward a much better world.

The excellent Tim Taylor on a new paper on plasma donation:

John M Dooley and Emily A Gallagher take a different approach in “Blood Money: Selling Plasma to Avoid High-Interest Loans” (Review of Economic Studies, forthcoming, published online May2, 2024; SSRN working paper version here). They are investigating how the opening of a blood plasma center in an area affects the finances of low-income individuals. As background, they write:

“Plasma, a component of blood, is a key ingredient in medications that treat millions of people for immune disorders and other illnesses. At over $26 billion in annual value in 2021, plasma represents the largest market for human materials. The U.S. provides 70% of the global plasma supply, putting blood products consistently in the country’s top ten export categories. The U.S. produces this level of plasma because, unlike most other countries, the U.S. allows pharmaceutical corporations to compensate donors – typically about $50 per donation for new donors, with rates reaching $200 per donation during severe shortages. The U.S. also permits comparatively high donation frequencies: up to twice per week (or 104 times per year)…”

Not too surprisingly, plasma donors tend to be young and poor and they use plasma donation to substitute away from non-bank credit like payday loans.

I am struck by Tim’s thoughts on how this connects with labor markets and regulation:

…I find myself thinking about the financial stresses that many Americans face. Being paid a few hundred dollars for a series of plasma donations isn’t an ideal answer. Neither is taking out a high-interest short-term loan; indeed, taking out a loan at all may be a poor idea if you aren’t expecting to have the income to pay it back. In the modern US economy, hiring someone for even a short-term job involves human resources departments, paperwork, personal identification, bookkeeping, and tax records. These rules have their reasons, but the result is that finding a short-term job that pays for a few days work isn’t simple, even if though most urban areas have a semi-underground network of such jobs.

Roger Miller’s classic 1964 song, “King of the Road,” tells us that “two hours of pushin’ broom/ Buys an eight by twelve four-bit room.” Even after allowing for a certain romancing of the life of a hobo in that song, the notion that a low-income person can walk out the door and find an two-hour job that pays enough to solve immediate cash-flow problems–other than donating plasma–seems nearly impossible in the modern economy.

Robert Kuttner discovered an excellent treatment for colds while vacationing in France and is rightly outraged that it’s not available in the United States:

Toward the end of our stay, my wife and I both got bad coughs (happily, not COVID). We went to our wonderful local pharmacist in search of something like Mucinex or Robitussin, which are not great but better than nothing.

“We have something much better,” said he. And he did. It’s called ambroxol. It works on an entirely different chemical principle, to thin sputum, facilitate productive coughing, and also operates as a pain reliever and gentle decongestant with no rebound effect.

We experienced it as a kind of miracle drug for coughs and colds. A box cost eight euros.

Ambroxol is available nearly everywhere in the world as a generic. It has been in wide use since 1979.

But not in the U.S.

He continues the story:

…You can’t get ambroxol in the U.S. because of the failure of the Food and Drug Administration to grant reciprocal recognition to generic medications approved by its European counterpart, the European Medicines Agency, when they have long been proven safe and effective. To get FDA approval for the sale of ambroxol in the U.S., a drug company would need to sponsor extensive and costly clinical trials. Since it is a generic, as cheap as aspirin, no drug company would bother.

…I’ve petitioned the FDA, asking them to create a fast-track procedure, whereby generic drugs approved in Europe, and well established as safe and effective, could get reciprocal approval in the U.S.

This would produce approval of ambroxol as over-the-counter medication for coughs and colds without unnecessary new clinical trials. And should ambroxol turn out to have real benefits for Parkinson’s as well, it would already be well established in the U.S. as an inexpensive generic.

Influenced by my work on FDA reciprocity aka peer approval, Ted Cruz introduced a bill, the Result Act to fast-track approval in the United States for drugs and devices already approved in other developed countries. Similarly, AOC has noted that the FDA is far behind the world in approving advanced sunscreens. Perhaps there is an opportunity here for bipartisan support.

Hat tip: the excellent Scott Lincicome.