Experts Express Concern About Right to Try, Offer Alternatives

With a Right to Try Act passed by the Senate and lingering in the House of Representatives, a panel of experts gathered at the Patient-Centered Oncology Care 2017 meeting in Philadelphia to discuss the merit of programs and laws that allow patients access to unproven medications. Across the board, they were dissatisfied with the legislation. They supported the current model of FDA access and offered potential improvements.

National Center for Health Research president Diana Zuckerman, PhD, spoke candidly.

“It’s so frustrating when members of Congress who know nothing about medicine try to pass a law like this. They’re responding to political pressure,” she said. The version that Congress is considering would make extended access to experimental drugs “very independent of the FDA,” she said, and she found that concerning.

Currently, patients can seek the drugs through the FDA’s expanded access program. Bypassing that system, Zuckerman worries, robs the FDA of access to the data about the drugs while opening the potential for companies to financially exploit desperate patients.

Marjorie A. Speers, Executive Director of the WCG Foundation, also had problems with Right to Try laws. Under the existing system, the process encourages deliberate decisions: first, the patient and their physician decide they want to pursue a drug undergoing clinical trial; then, they request that access from the drug’s maker and the company can decide if they believe the drug will be useful; finally, the FDA reviews and approves the request.

Right to Try, Speers argued, bypasses all of that, which could be detrimental. “These uses can only occur for desperately ill people, there’s no other treatment option for them,” she said. “It’s not necessarily the best time to be making a decision.”

Besides, the FDA has been working on the expanded access system. The application forms have been streamlined, and the agency’s website provides a thorough, transparent explanation of use cases and criteria. Zuckerman added that FDA already approves expanded access requests nearly 99% of the time.

The third panel member, Wm. Kevin Kelly, DO, brought firsthand experience to the conversation. He advised the FDA for 7 years and works as an oncologist at the Sidney Kimmel Medical College at Thomas Jefferson University, where he is involved in enrolling patients in clinical trials.

“Being on the frontline, it’s a lot different,” he said. Patients lack education about their options, and physicians are often overwhelmed by requests and requirements.

When a patient receives access to a drug through expanded access, their physician is supposed to report back to the FDA about how the treatment is working. The panel agreed that often, that does not happen. Kelly said the paperwork is extensive, and that could be a limiting factor: a streamlined reporting system would encourage doctors to hold up their end of the expanded access bargain.

“There’s a lot of hype in the literature… ‘They say there’s a cure for my cancer, can I have it doctor?’” he added. All too often, he said, a patient will dump study printouts from clinicaltrials.gov on his desk and ask if they can take part in any of them.

Rather than passing things like Right to Try laws, Kelly said, he’d like to see the existing infrastructure and clinical trial process improved. Less than 10% of patients in the United States participate in a clinical trial. With more education and a patient-centric navigation source that helps connect people with trials, more patients could get access to experimental drugs while providing the FDA and pharma with more data.

“If we had a system that was patient-centric and could help patients find the right place to go, a lot of these problems would be solved,” he said.