Insurer Unconvinced by Divided FDA’s Approval of Muscular Dystrophy Drug

Published December 26, 2016

Health insurer Anthem has announced its refusal to cover a $300,000 drug used to treat patients suffering from Duchenne muscular dystrophy (DMD) after the U.S. Food and Drug Administration (FDA) announced a controversial accelerated approval of the drug.

FDA Commissioner Robert Califf approved Exondys 51, the first drug FDA has approved to treat DMD, in September 2016, backing the director of the agency’s Center for Drug Evaluation and Research, Dr. Janet Woodcock, in her recommendation to overrule an FDA advisory committee.

Seven of the 13 committee members voted the drug failed to produce a crucial protein called dystrophin “at a level likely to result in clinical benefit,” according to a Journal of the American Medical Association (JAMA) article published on October 24.

Exondys 51, or eteplirsen, treats a rare form of DMD afflicting about one in 3,600 male infants, usually causing death between age 20 and 30, according to an FDA press release announcing the drug’s approval through the year 2021. FDA gave the drug’s manufacturer, Sarepta Therapeutics, until then to confirm the drug’s effectiveness in clinical trials or risk retraction of its approval.

On October 6, Anthem signaled its refusal to pay for the drug by classifying Exondys 51 as “investigational and not medically necessary” in a medical policy analysis of FDA’s divided, tentative approval.

Humana, another insurer, will consider the drug medically necessary and pay for its use on the condition patients remain “able to walk with assistance, not wheelchair dependent,” according to a pharmacy coverage policy that took effect on October 26.

Accelerated? Not So Fast

Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy, says FDA’s seven-year accelerated approval process for Exondys 51 was too slow.

“We are grateful for the outcome of the review, and we appreciate that [this] rare disease is complex, but we do not consider this review to have been a shortened process,” Furlong said. “Quite the contrary.”

FDA’s fast track failed DMD patients who might have benefitted from Exondys 51 sooner, Furlong says.

“In the context of the average life expectancy of DMD patients, who are eligible for Exondys 51 but had not been included in the original trial, [they] have waited more than 25 percent of their lifespans for this product’s trial to be conducted and reviewed,” Furlong said.

Dr. Richard Dolinar, a practicing endocrinologist and pharmaceutical industry consultant, says FDA should accelerate its approval process across the board.

“I am in favor of shortening the review process for all drugs,” Dolinar said. “Some people awaiting these drug approvals are suffering greatly. Others are actually dying. We should thus speed them through as quickly as possible.”

Must Hear Out Patients

FDA’s approval was preceded by a public opinion campaign highlighting support for Exondys 51 by families affected by DMD, illness experts, and Sen. Marco Rubio (R-FL), the business news site Quartz reported on November 1.

Supporters spoke directly to FDA in April. Their testimony was “frequently emotional,” according to the JAMA article, which describes Exondys 51 as a “worrisome model for the next generation of molecularly targeted therapies.”

Furlong says the Patient-Focused Drug Development (PFDD) initiative, part of the Prescription Drug User Fee Act, requires FDA to consider the perspectives of patients undergoing treatment.

“The FDA needs to be sensitive to the opinions of patients and families as part of the PFDD concept that is currently law,” Furlong said. “The FDA needs to consider the totality of data when making a decision about whether to approve or not to approve an indication.”

Costs of Maintaining Prevention

Dolinar says preventing patients from trying drugs not proven harmful is an overreach by government and endangers patients.

“The role of the FDA is to protect us from medicines that could harm us,” Dolinar said. “But by not allowing us to try a medicine that has not been FDA-approved, they instead leave us at risk to be harmed by the very disease itself that the medicine was intended to address.”

Patients should be the final arbiters weighing a new drug’s risks and rewards, Dolinar says.

“The decision to actually take the drug [should be] ultimately up to the patient,” Dolinar said. “That is a value judgment by the patient. Value judgements are subjective and as such cannot be measured nor made by anyone other than the patient.”

Tony Corvo ([email protected]) writes from Beavercreek, Ohio.

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