FDA Leader Defends Agency's Drug Approval Process

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By Bronwyn Mixter

Oct. 17 — The head of the FDA defended the agency's drug approval process and emphasized the benefits of career employees, not political appointees, making the key decisions.

Commissioner Robert Califf said Oct. 17 his agency has “enormous latitude” through its accelerated approval pathway to consider the evidence and make a decision on a new product. The Food and Drug Administration's accelerated approval program allows a drug to be approved based on substantial evidence that the drug has an effect on a surrogate endpoint reasonably likely to predict a clinical benefit to patients. Califf said “there is no guidance on what reasonably likely means” and “this approach invests a lot of trust in FDA.”

The FDA leader said political appointees, like himself, shouldn't make accelerated approval decisions because they may be influenced in some way and these decisions should be make by “career people” at the FDA.

For instance, Califf said Janet Woodcock, the director of the FDA's Center for Drug Evaluation and Research, “made a reasonable decision” to grant accelerated approval to Sarepta Therapeutics Inc.'s Exondys 51 (eteplirsen) for treating Duchenne muscular dystrophy. The approval of the drug has been criticized because the FDA's advisory panel voted that insufficient evidence existed to show the drug is effective against the disease.

Califf was asked if he saw a need for guidance on what constitutes a “reasonably likely” benefit. He replied, while there should be more discussion on accelerated approval, he thinks “it would be a big mistake to get too prescriptive” and a guidance “shouldn't set a threshold” for what determines a reasonably likely benefit.

Califf spoke at a conference sponsored by the National Organization for Rare Disorders (NORD).

Access to New Products

Califf said the pipelines for new products “are bursting” and “the question is what we should invest in and what's likely to succeed.”

“The latest data indicates that the vast majority of drugs won't make it to the market” because they either are not effective or they have safety issues, the FDA leader said. “While speed to access is important, we must continue to develop methods that separate the wheat from the chaff.”

Califf said the FDA grants access to investigational treatments over 99 percent of the time through its expanded access program.

Expanded access is the use outside of a clinical trial of an investigational product.

However, Califf said even if the FDA grants expanded access to a treatment, “it's up to the company to decide whether the drug can be made available.”

Grants for Development

Califf also said the FDA is awarding additional clinical trial research grants for rare disease products.

In an Oct. 17 press release, the FDA said it is awarding 21 clinical trial research grants totaling more than $23 million over the next four years to boost the development of products that treat rare diseases. The FDA awards the grants through its orphan products clinical trials grants program to encourage clinical development of drugs, biologics, medical devices or medical foods for use in rare diseases.

The FDA said 24 percent of the grants will fund studies enrolling cancer patients and 43 percent will fund studies that enroll pediatric patients as young as newborns. Also, one funded project is a medical device trial to develop a fully implantable neuroprosthesis for grasp, reach and trunk function in individuals with spinal cord injury, the agency said.

Califf said that since the orphan products clinical trials grants program was created in 1983, the FDA has provided $370 million to fund more than 590 new clinical studies that supported the marketing approval of more than 55 products.

Patient Involvement

Califf said patients and their advocates should be involved in every stage of drug development.

“The commitment to patient involvement is deep and fundamental across the organization,” he said.

Califf said the FDA's patient-focused drug development program is helping the agency learn about the priorities of patients, especially how they think about risks and benefits.

To contact the reporter on this story: Bronwyn Mixter in Washington at bmixter@bna.com

To contact the editor responsible for this story: Brian Broderick at bbroderick@bna.com

For More Information

More information about the conference is available at https://rarediseases.org/summit-event-details/.

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