Radar on Specialty Pharmacy

The FDA is conducting an industrywide evaluation of drugs with indications given accelerated approval in an effort to determine whether confirmatory clinical trials verified the therapies’ clinical benefits. Manufacturers already have withdrawn indications approved in the U.S. for four drugs, and an upcoming meeting will scrutinize six additional indications. It’s important that the agency speed approvals of cancer drug indications if they look promising, contend experts, but it’s equally important that the FDA confirms their efficacy.

“The FDA has a large toolbox of options for getting important products to the market,” says Nancy Dreyer, Ph.D., chief scientific officer at IQVIA Real World Solutions. These include priority review, accelerated approval, fast track and breakthrough therapy designations, as well as emergency use authorizations (EUAs), which are used in emergency situations, such as the COVID-19 pandemic. “It appears that we are well covered in terms of having EUA for emergencies and other fast-track options that assure that the risks are not likely to outweigh the benefits of a new treatment and that the new treatment offers meaningful therapeutic benefit.”

✦ AstraZeneca said Feb. 22 that it would voluntarily withdraw the Imfinzi (durvalumab) indication in the U.S. for previously treated adults with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or who have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. The FDA gave the indication accelerated approval on May 1, 2017 (RSP 5/17, p. 10). A confirmatory trial did not meet post-marketing requirements. View the statement at https://bit.ly/3kF92RO.

✦ Merck & Co., Inc. said March 1 that it was voluntarily withdrawing the U.S. indication for Keytruda (pembrolizumab) for the treatment of patients with metastatic small cell lung cancer with disease progression on or after platinum-based chemotherapy and at least one other line of therapy. The FDA gave the drug accelerated approval on June 18, 2019, for the indication (RSP 7/19, p. 8). The company was required to conduct a post-marketing study to establish superiority of the drug as determined by overall survival. The confirmatory Phase III trial had two primary endpoints; the drug met the progression-free survival endpoint but did not reach statistical significance for the overall survival endpoint. The move comes just a few months after Bristol Myers Squibb said on Dec. 29, 2020, that it was withdrawing the exact same indication for Opdivo (nivolumab) following accelerated approval on Aug. 17, 2018 (RSP 1/21, p. 12). View Merck’s statement at https://bit.ly/2NKEe6t.

✦ Feb. 3: The FDA gave accelerated approval to Merck KGaA unit EMD Serono, Inc.’s Tepmetko (tepotinib) for the treatment of adults with metastatic non-small cell lung cancer (NSCLC) harboring mesenchymal-epithelial transition (MET) exon 14 skipping alterations. The agency gave the drug priority review and breakthrough therapy and orphan drug designations. The therapy was approved under the FDA’s Real-Time Oncology Review pilot program. Dosing for the tablet is 450 mg via two 225 mg tablets once daily. The list price of a 30-day supply is $20,898. Visit www.tepmetko.com.

✦ Feb. 5: The FDA approved Bristol Myers Squibb’s Breyanzi (lisocabtagene maraleucel) for the treatment of adults with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma not otherwise specified, high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma and follicular lymphoma grade 3B. The agency gave the CD19-directed chimeric antigen receptor T cell (CAR-T) therapy orphan drug, regenerative medicine advanced therapy and breakthrough therapy designations. The list price for the one-time treatment is $410,300. Visit www.breyanzi.com.

Therapeutic interventions have evolved from people taking a pill every day to treat a condition to the one-time cell and gene therapies aimed at halting disease progression or even curing a disease. The rise of these innovative new products means that payers need more information than ever in order to properly assess treatments, as well as updated reimbursement models.

“The science is moving at lightning speed, and what we thought was previously impossible has now become a reality with some of the newer gene therapies and other novel drug approvals,” observes Reta Mourad, Pharm.D., a senior director on the access experience team at PRECISIONvalue. “As we learn more about diseases and develop a deeper understanding down to the genetic and molecular level, we are able to see advances in therapies that were unheard of,” for example with chimeric antigen receptor T cell (CAR-T) therapies, the first of which were approved in 2017.

The FDA recently approved the first long-acting regimen for the treatment of HIV in adults. The dosing schedule could help with adherence in a condition where that is particularly crucial. However, some potential challenges exist with the medication, including whether health plans actually will cover it.

On Jan. 21, the FDA approved ViiV Healthcare’s Cabenuva (cabotegravir and rilpivirine) for the treatment of HIV-1 infection in adults to replace a current antiretroviral regimen in people who are virologically suppressed on it with no history of treatment failure and no known or suspected resistance to either cabotegravir or rilpivirine.