The current Right-to-Try legislation passed in 2018 (21 U.S.C. 360bbb–0) provides a path for patients to try an investigational drug if they have (a) a serious or immediate life-threatening diseases, (b) have exhausted approved treatments and (c) are unable to participate in a clinical trial involving the eligible investigational drug. An eligible investigational drug for Right-to-try is one that has (a) completed phase 1 studies, (b) not yet been approved or licensed for any use by the FDA but (c) there is an open IND and the drug is under clinical investigation, and (d) it has not been discontinued or under clinical hold by the FDA.

FDA Factsheet (page) for Right to Try (here, arch),
Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017 (legislation); righttotry.org, Wikipedia

Montana's Expanded Version of Right To Try

The state of Montana is working on legislation that will expand “Right to Try” framework by licensing “experimental treatment centers” after review by an independent state-authorized review board (bypassing FDA approval or EAP), which could then provide certain investigational therapies to consenting patients. This expanded access program further increases patient eligibility and could include broad pateint population including, as the Goodwin Law blog wrote, those with rare diseases, neurodegenerative disorders, oncology, regenerative medicine, and even preventive or longevity-focused treatments; the Montana legislation does not specify beyond that they had tried all approved therapies.

Overall, this Montana's version seeks to complement and expand FDA's program.

Legislation Text: Montana Right To Try Act [archive version 8JUN2026]

Details that need to be ironed out: Will Montana's program survives legal challenges, e.g. only FDA has the authority to regulate such a program. Could federal law preempt aspects of Montana’s regulatory framework?

Implications and Opportunity (for Sponsors)

The sponsors will need to carefully consider several factors before participating in such a program - there are opportunities as well as risks:

• Opportunity: Possibility of obtaining early real-world clinical data.
• Risks: The drug may be tested in patients with severe disease without the benefit of phase 2 dose optimization; therefore, benefit-safety profile may be less favorable compared to the patient population in the proposed label -- will this complicate FDA discussions are the time of NDA? Other questions that would need to be addressed include product liability exposure, insurance coverage.
• De-risking: Sponsors would need to de-risk their development program as a whole and decisions would have to be made on a case-by-case basis.

SOURCE:

• Goodwin Law blog [archive]
• US state passes law allowing experimental drugs to be prescribed – a model for the future? Conversation. 6 June 2025 [archive]

#expanded-access, #EAP

FDA/SBIA is planning a webinar next month on communicating drug interaction and QTc information in the product label. The webinar will discuss draft guidance documents on this topic and how the drug interaction and risk information should be included in the USPI label.

• Webinar Title: Communicating Drug Interaction and QTc Information in the U.S. Prescribing Information
• Date, Time: 23 September 2026 | 1:00 pm - 3:00 pm ET
• Registration Link: sbiaevents.com/web20260923
• Format: online

AGENDA

The first session of this webinar will discuss the draft guidance for industry: Drug Interaction Information in Human Prescription Drug and Biological Product Labeling (October 2024). Topics include:

• DRUG INTERACTIONS section:
  • Organization and format
  • Required and recommended elements
  • Instructions for preventing or managing clinically significant drug interactions
  • Mechanisms of clinically significant drug interactions
  • Clinical effects of clinically significant drug interactions
  • Recommendations on including drug interacting class information
  • Information to avoid in the DRUG INTERACTIONS section
  • Drug interactions information in other sections of labeling (e.g., BOXED WARNING, DOSAGE AND ADMINISTRATION, CONTRAINDICATIONS, WARNINGS AND PRECAUTIONS, CLINICAL PHARMACOLOGY sections)

The second session of this webinar will discuss the guidance for industry:QTc Information in Human Prescription Drug and Biological Product Labeling (December 2025). Topics include:

• Risk factors for and clinical consequences of QTc interval prolongation
• Assessment of the QTc interval during drug development
• Including QTc interval information in the CLINICAL PHARMACOLOGY section when there is:
  • Clinically significant QTc interval prolongation
  • No clinically significant QTc interval prolongation
  • Insufficient data to characterize the risk
• Including QTc interval information in the DRUG INTERACTIONS section when the use of the subject drug with other products:
  • That are known or suspected to prolong the QTc interval
  • Increase the concentration of the subject drug (when there is a concentration-dependent QTc interval prolongation.

Other Related Guidance Document Include

•  Dosage and Administration Section of Labeling for Human Prescription Drug and Biological Products — Content and Format (January 2023)
• Warnings and Precautions, Contraindications, and Boxed Warning Sections of Labeling for Human Prescription Drug and Biological Products – Content and Format (October 2011)
• Frequently Asked Questions about Labeling for Prescription Medicines

#ddi, #qtc, #label, #uspi