FDA approved a record high of 43 drugs through fast-track programs in 2018, representing 73% of new drugs approved by the agency last year and establishing a "new normal" that "is transforming medical decision-making for the seriously ill."

What executives need to know about pharmacy

'A new normal'

Under FDA's accelerated approvals process, drugmakers can receive FDA approval to sell their products in the United States if preliminary studies suggest they improve so-called "surrogate measures" of a disease, such as by shrinking the size of a tumor. In exchange for early entry into the marketplace, FDA requires drugmakers to conduct follow-up clinical trials to determine whether the drug presents a clinical benefit to patients, such as improving overall survival or quality of life.

According to the Wall Street Journal, FDA generally fast tracks drugs intended to treat conditions that are debilitating or deadly and have few or no other treatments. The agency typically fast tracks drugs in response to pressure from drugmakers, patients, physicians, and policymakers, and approves such drugs based on early research that indicates the drugs could be effective, rather than longer-term studies the agency usually requires for approval, the Journal reports.

According to the Journal, FDA in recent years has been approving increasing shares of drugs via fast-tracked programs. FDA granted fast-tracked approval to at least 60% of new drugs the agency approved in each of the past five years, the Journal reports. In comparison, FDA 10 years ago approved just 10 drugs through a fast-tracked process, representing 38% of new drugs the agency approved that year, according to the Journal.

Increase in fast-tracked drugs raises quality, cost concerns

But FDA's increasing likelihood to approve drugs via fast-track programs is shifting "[f]amilies and doctors … into a new world of trade-offs, raising complex questions about the medical and financial value of drugs with limited track records," the Journal reports.

Janet Woodcock, director of FDA's Center for Drug Evaluation and Research, has said the agency's fast-track programs are beneficial even without having long-term evidence on drugs' effectiveness, because they allow patients to more quickly access drugs that later prove to be effective.

However, Joseph Ross, an associate professor at Yale School of Medicine who researches drug development, said FDA's increasing fast-tracked approvals also means that government health programs and private insurers sometimes end up paying for costly treatments that never prove effective, and that patients in some instances face unnecessary out-of-pocket costs and side effects.

For instance, Eli Lilly in April announced that it was pulling its cancer drug Lartruvo, which FDA had approved in 2016 via a fast-track program, from the market because a new, large study showed the drug did not extend the lives of patients with advanced-sarcoma patients, the Journal reports.

According to the Journal, FDA granted accelerated approval to the drug based on one small study that showed patients who took Lartruvo lived longer than patients who did not take the drug. Patricia Keegan, director of FDA's Division of Oncology Products 2, said she and her colleagues granted Lartruvo accelerated approval even though the initial study offered confounding information. She said regulators were unsure how to "weight" confounding data from the study against the drug's apparent benefits. "We were aware it was imperfect," she said.

FDA stipulated that it would not give Lartruvo the agency's full approval until a follow-up study confirmed the drug's clinical benefit. Eli Lilly released the results of that study in January, and found that median overall survival for patients treated with Lartruvo and chemotherapy was 20.4 months, compared with 19.7 months for patient treated with only chemo. Eli Lilly said the findings did not meet the standards required to show Lartruvo significantly extended patients' lives when compared with other treatments.

But other fast-tracked drugs have proved to be effective and have helped to treat debilitating diseases such as hepatitis, HIV/AIDS, and leukemia, the Journal reports. For example, FDA in 2001 approved Novartis' Gleevec under a fast-tracked program to treat leukemia based on evidence showing the drug's effect on cancer cells. Gleevec since has proved to significantly affect patients' long-term survival, according to the Journal.

Overall, an analysis of FDA data on fast-tracked cancer drugs by the Journal found that 19% of the 42 cancer drugs the agency approved via fast-track programs from 2015 through 2018 now have evidence showing they significantly extend patients' lives, as well as 26% of the 34 expedited cancer drugs FDA approved from 2011 through 2014.

Some say fast-tracked drugs should come with reimbursement requirements

According to the Journal, some health care officials say FDA should require drugmakers to sell products approved via the agency's fast-track programs at a discounted price until they submit research proving the drugs are effects, or they should be required to reimburse patients and health insurers for the treatments if it turns out the drugs are ineffective.

Michael Sherman, CMO at the insurer Harvard Pilgrim Health Care, has proposed having a third party set prices for fast-tracked drugs, and stipulating that drugmakers are paid for the treatments only if they prove effective (Loftus, Wall Street Journal, 7/5; Owens, "Vitals," Axios, 7/8).