Could States Create Their Own Pathways to Access Experimental Treatments? Montana May Be Testing the Limits of FDA’s Traditional Role

Historically, the U.S. Food and Drug Administration (FDA) has served as the primary gatekeeper for the development and approval of medical products, including new drugs and biologics. While states have traditionally regulated the practice of medicine, regulation of the development and approval of pharmaceuticals has remained within the federal government’s domain.

Montana may be testing whether that balance can change.

Earlier this summer, Montana finalized regulations implementing an expanded “Right to Try” framework that could allow certain investigational therapies to be offered through licensed “experimental treatment centers” after review by an independent state-authorized review board, rather than through FDA approval or an expanded access process. Under the Montana framework, companies whose products have completed early clinical testing may seek approval to offer those therapies to consenting patients who choose to pursue treatment.

Although the program is still in its infancy, it raises a provocative question. Are we witnessing the emergence of a parallel regulatory pathway for innovative medicines that could operate alongside, rather than entirely within, the FDA’s traditional frameworks?

More Than an Expansion of Right to Try

Most Right to Try statutes have been narrowly focused on patients with terminal illnesses who have exhausted conventional treatment options.

Montana’s approach appears designed to accommodate a significantly broader scope.

Rather than limiting to compassionate use, Montana has established an administrative structure through which investigational therapies may be reviewed by an independent board and offered by licensed treatment centers under state law. If successful, this model could create a new avenue for patients seeking access to experimental therapies, particularly in areas such as rare diseases, neurodegenerative disorders, oncology, regenerative medicine, and even preventive or longevity-focused treatments.

Whether other states ultimately adopt similar frameworks remains to be seen, but Montana’s law clearly seeks to move beyond the traditional conception of Right to Try.

Potential Implications for Biotech Companies

For certain biotechnology companies, the potential implications of such a pathway could be significant.

Drug development continues to require enormous investments of time and capital. Many companies spend hundreds of millions of dollars and nearly a decade (or more) navigating clinical development before seeking approval and generating meaningful commercial revenue. During that period, promising programs may stall because of financing constraints rather than scientific failure.

If carefully structured state pathways become available, some companies may begin evaluating whether limited commercialization in a state such as Montana could complement, rather than replace, the traditional FDA approval process.

Such programs could potentially provide earlier patient access, generate real-world clinical experience, strengthen physician familiarity with emerging therapies, and create modest commercial revenues during continued clinical development.

At the same time, participation could introduce meaningful regulatory uncertainty. Companies would need to carefully consider potential impacts on ongoing FDA interactions, investor expectations, reimbursement strategies, product liability, insurance coverage, and future partnering discussions. In addition, unexpected patient safety outcomes could draw unwelcome publicity and have reputational implications.

Questions Investors May Begin Asking

The Montana framework is also likely to attract attention from venture capital investors, private equity firms, and strategic acquirers. Some potential questions include: 

• Could early state-based use reduce financing needs?
• Might real-world treatment data enhance company valuations?  Would the data generated through state-level experimental use be of sufficient quality to support development programs that are intended to be submitted to regulators for review?
• Would investors view participation as evidence of commercial traction or as an unnecessary regulatory risk?
• Could this become particularly attractive for companies developing therapies for rare diseases where patient populations are small and highly motivated?  Alternatively, could this pathway undermine the ability to recruit patients for traditional clinical trials?
• Will companies consider pricing their products differently?

These questions do not yet have clear answers, but they are precisely the types of strategic issues sophisticated investors are likely to begin evaluating.

Important Legal Questions Remain

The Montana law also raises significant legal and regulatory issues that have yet to be resolved.

Among them are:

• Could situations arise where the FDA may seek to exercise its enforcement authority over uses authorized under Montana’s law? To what extent could federal law preempt aspects of Montana’s regulatory framework?  
• Could participation complicate later FDA interactions, including seeking approval?
• How will product liability exposure differ from traditional clinical trials or expanded access programs?
• What standards of informed consent, physician oversight, and adverse event reporting will ultimately emerge?
• Will insurers provide coverage for investigational therapies offered under state law?
• How will these programs interplay with coverage from existing federal healthcare programs such as the Medicaid Drug Rebate Program, the 340B Drug Pricing Program, Medicare, and the Department of Veterans Affairs?

The answers to these and other questions will determine whether Montana remains an isolated experiment or becomes another step toward broader state involvement in pharmaceutical regulation.

Looking Ahead

Montana’s new framework is unlikely to replace the FDA. Nor should it.

FDA review remains the foundation for nationwide development, approval, and commercialization.

Nevertheless, Montana may have initiated an important conversation about whether states can play a larger role in facilitating earlier access to innovative therapies while maintaining appropriate safeguards for patient safety.

If other states begin adopting similar legislation, and if medical product manufacturers and healthcare providers opt to participate in these state frameworks, biotechnology companies may find themselves navigating not only federal regulatory strategy, but also an evolving landscape of state-level opportunities and risks.

For companies, investors, healthcare providers, and policymakers alike, Montana’s experiment deserves closer attention. It may ultimately prove to be a limited policy experiment — or it may represent the beginning of a broader rethinking of how innovative medicines can reach patients in the United States.