Montana opened a market for experimental medicines. Now it should watch who shows up

Montana has created something no other state has: Licensed experimental-treatment centers where patients can obtain treatments that have completed Phase 1 clinical trials but have not been approved by the Food and Drug Administration. For Montanans facing serious illnesses, the appeal is strong. Geography should not determine access to promising medicines.

But Montana’s experiment raises a question: Which drug developers will choose to participate?

For more than 15 years, I have worked in oncology research and pharmaceutical drug development, including a decade in senior leadership. From that perspective, the crucial feature of Montana’s market is that manufacturers decide whether to make treatments available.

Montana cannot compel a company to provide its investigational drug. A treatment center still needs access to the product, and the developer controls that supply. Their incentives are not equal.

Consider a well-financed company with encouraging early data and a clear path into Phase 2 and, if warranted, Phase 3 trials. The value of its drug does not come from the vial alone. It comes from evidence that may establish whether the drug works, for which patients, at what dose and with what balance of benefit and risk.

For such a company, tightly controlled development is not a bureaucratic inconvenience. It is how value is created. The sponsor controls manufacturing, patient eligibility, dosing, safety monitoring, data collection and interpretation. Even drug supply can be strategic.

Montana offers something different: The possibility of treating paying patients before efficacy has been established.

That may be unattractive even to a developer short of cash. Reputational risk, limited supplies needed for clinical trials and uncertainty about federal regulation can deter participation. But those deterrents will not be equal. A developer struggling to finance its next trial or seeking an unconventional route to generate revenue may value early commercial access differently from a company with a well-financed conventional path.

That does not mean such a company has a bad drug. Nor does it mean a large pharmaceutical company would never participate. It means participation will not be random.

That is the overlooked risk in Montana’s experiment. A market for experimental medicines will not necessarily attract the most promising medicines. It may disproportionately attract developers with stronger incentives to monetize before efficacy has been established, while companies with conventional regulatory paths may have less reason to participate.

This is not primarily an argument about whether patients should accept the uncertainties of experimental treatment. A therapy that has completed Phase 1 has undergone initial testing of safety, tolerability, dosing and pharmacology and may provide early signs of efficacy. But Phase 1 generally does not establish that a treatment is effective or that its benefits outweigh its risks for routine use.

Commercial questions follow. What is a drug worth before its clinical benefit is known? Who sets that price? Willingness to pay is not evidence of therapeutic value, particularly when a patient is seriously ill and searching for options.

What happens in Montana could matter beyond Montana. If other states adopt similar systems, small markets could become a parallel commercial pathway in which companies monetize products before completing trials designed to determine whether they work. Policymakers should ask what that would do to trial enrollment, development incentives and investigational drug supplies.

Montana is right that geography should not determine access to medical innovation. But creating a market is not the same as creating a reliable signal of value.

Clinical trials ask whether a medicine works. Markets ask whether someone will buy it. Montana’s experiment now requires a third question: Which companies will choose to sell before the first question has been answered, and why?

Professor David Adler is an oncology drug-development and translational medicine leader with more than 15 years of industry and academic experience. He spent a decade in senior leadership within Bayer AG’s Global Oncology Clinical Development organization and currently serves as Chief Scientific & Medical Officer of the PATHORA Institute of Pathology & Tissue Medicine. He also holds academic appointments at the Hebrew University of Jerusalem, Ben-Gurion University of the Negev and the University of Bonn.