Goldwater Institute’s Brian Norman: ‘We need to provide a pathway’ for patients to access individualized treatments

Brian Norman, director of state affairs at the Goldwater Institute, said expanding “Right to Try” laws could help patients with rare and life-threatening conditions access cutting-edge treatments that are currently out of reach due to federal regulatory barriers.

“We think the science is right for this moment, and we think we need to provide a pathway for them to access the most promising treatments,” Norman said on the Health Policy Podcast.

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Norman discussed “Right to Try 2.0,” an effort to build on the original Right to Try law, which was signed into federal law in 2018 and allows terminally ill patients to access investigational drugs that have passed early safety trials. The updated approach focuses on individualized or “n-of-one” treatments, including gene therapies and other personalized interventions tailored to a patient’s genetic profile.

“These individualized treatments… often are designed using an analysis of the patient’s own genetic information,” Norman said, adding that they represent some of the most innovative options available for patients with rare or ultra-rare diseases.

However, he said the current regulatory framework was designed for mass-market drugs and does not align with the realities of highly specialized treatments. The traditional approval process can take up to a decade and cost more than $1 billion, making it impractical for therapies targeting small patient populations.

Norman pointed to a case involving an Arizona family whose daughter was able to receive a gene therapy treatment in Italy but not in the United States. “They should have been able to access that here at home in Arizona,” he said, describing the situation as a key example of why reform is needed.

The proposed Right to Try 2.0 framework would allow eligible patients with rare or life-threatening conditions to work with their physicians to seek access to individualized treatments, provided certain safety and facility requirements are met. Norman said the goal is to create a more efficient pathway without eliminating safeguards.

Opposition to similar reforms has historically come from medical ethicists concerned about patient safety and the potential for unproven treatments to create false hope. Norman rejected that argument, saying decisions about care should ultimately rest with patients and their doctors.

“If a doctor thinks this might help, then I should be able to pursue that,” he said.

Norman said 16 states have already adopted versions of Right to Try 2.0, and advocates are now working to establish a federal pathway to expand access nationwide. He emphasized that broader reform of the drug approval process is still needed, but said targeted efforts like Right to Try can help patients who do not have time to wait.

“Time is of the essence for them,” Norman said. “We really should not be dragging our feet… when it comes to their healthcare decisions.”