Matt Kaeberlein, chair of Montana’s experimental treatment board, responds to criticism of a law allowing unapproved drugs to be sold after Phase 1 trials: an independent board reviews every treatment protocol

Matt Kaeberlein, chair of Montana’s experimental treatment board, responds to criticism of a law allowing unapproved drugs to be sold after Phase 1 trials: an independent board reviews every treatment protocol

On October 31, Kaeberlein responded to an essay by Thomas Seoh (Thomas Seoh), executive vice president of the Kitalys think tank. Seoh argues that the law weakens incentives to test whether drugs work.

Montana’s SB 535 allows unapproved drugs to be sold after Phase 1 trials, the initial safety testing typically conducted in 20–100 people. The law requires no diagnosis: patients confirm that approved treatments are unlikely to produce the desired result or are unavailable, obtain a recommendation from their treating healthcare professional, and sign a consent form. A private board, launched on July 25, reviews the treatment protocol.

Seoh argues that Phase 1 provides limited information: if no noticeable adverse effect is found among 30 participants, all that can be said is that it occurs in fewer than roughly one in ten people. Allowing sales immediately afterward changes the incentives: patients who can buy the drug have less reason to enter a placebo-controlled trial, while investors may prefer revenue to a trial that could show the product does not work. Seoh trusts the current board but asks who will serve on boards in other states, given that the law requires only Phase 1 testing.

“A law that sets a minimum and relies on officials to exceed it describes today’s officials rather than a system.”

Kaeberlein counters that Montana’s law is “not a longevity law” and that Phase 1 is “a starting point for review, not a license to sell.” The law does not mention longevity, although its sponsor, Senator Ken Bogner, told NBC Montana that it was important to “give people access to medicines that keep them healthy longer.”

According to Kaeberlein, an independent board reviews every protocol. Under state rules, the board assesses safety, the clarity of the consent form, the balance of risks and benefits, and other treatment options. At a conference in Hong Kong, Kaeberlein said that the review “primarily concerns safety rather than efficacy,” according to Seoh’s account.

Kaeberlein shares Seoh’s concern about whether future boards will maintain rigorous standards. He also supports THRIVE, a Kitalys legislative proposal that would allow early market access with an obligation to demonstrate efficacy within a specified period. Reforms such as Montana’s arose, he says, because the FDA has become “prohibitively slow and expensive,” while weak oversight has allowed a black market in peptides to grow. At the same conference, David Fox, a former FDA lawyer, countered that companies are the obstacle to individual access to unapproved medicines, rather than the FDA. According to FactCheck.org, the FDA approved 99% of such requests for seriously ill patients during fiscal years 2019–2023.

The board issued its first decision on August 26, voting 5 to 0 to approve a Parley Neurotech therapy with conditions. The therapy combines the antihistamine clemastine with specially designed audio delivered through headphones. It is intended to treat an age-related impairment in the brain’s ability to understand speech in noise. The conditions require removing a statement from the consent form that the drug’s safety in humans has already been established and adding a recommendation that the physician assess kidney and liver function, blood tests, heart health, and fall risk. Clemastine completed Phase 1 during its original development, while the combination is still being tested against placebo. The board found “reasonable grounds” to consider the law’s Phase 1 requirement met for the combination, based on the company’s assurance and a legal memorandum. The decision states: “Another reasonable interpretation does not, in itself, preclude approval.”