Spotlight on Market Access

The continuing rise of weight-loss medications and manufacturers entering the direct-to-consumer (DTC) health care arena were among some of the top pharma industry trends in 2024. Oncology remained a leading area of drug development, as did gene therapies, which in turn spurred the use of nontraditional payment models for payers as they grappled with covering the multimillion-dollar therapies. AIS Health spoke with Julia Scanlan, associate consultant in advisory services for MMIT, about those and other noteworthy occurrences within the pharma industry over the past year. (Editor’s note: These comments have been edited for length and clarity. AIS Health and MMIT are both Norstella companies.) 

Since the first chimeric antigen receptor T cell (CAR-T) therapy was approved on Aug. 30, 2017, several similar cellular agents have followed, and 2024 was a particularly noteworthy year. Accomplishments included the first cell therapy for treating a solid tumor, the first new CAR-T since 2022 and two approvals of existing therapies in earlier lines of treatment.

On Feb. 16, 2024, the FDA gave accelerated approval to Iovance Biotherapeutics, Inc.’s Amtagvi (lifileucel) for the treatment of adults with unresectable or metastatic melanoma previously treated with a programmed death receptor-1 (PD-1) inhibitor and, if BRAF V600 positive, a BRAF inhibitor with or without a MEK inhibitor. The tumor infiltrating lymphocyte (TIL) therapy — which is created from patients’ unique T cells from their tumor as opposed to the T cells in a patient’s blood that the CAR-Ts use — is the first and only one-time individualized T cell therapy with FDA approval for a solid tumor cancer.

Amgen Inc. has tapped Optum Health Solution’s new biosimilars-focused private-label subsidiary Nuvaila to be the sole distributor of its Wezlana (ustekinumab-auub), the first available biosimilar of Stelara (ustekinumab) from Johnson & Johnson Innovative Medicine (formerly Janssen Biotech, Inc.). While such deals may be beneficial to manufacturers that can strike them, they are essentially locking out competitors that can’t from the biosimilar market, asserts an industry expert.

Stelara is approved for the treatment of adults and pediatric patients at least 6 years old with moderate-to-severe plaque psoriasis who are candidates for phototherapy or systemic therapy, adults and pediatric patients at least 6 years old with active psoriatic arthritis, adults with moderately to severely active Crohn’s disease and adults with moderately to severely active ulcerative colitis.

The FDA’s accelerated approval pathway has become an increasingly popular way for drugmakers to get novel medications to market. However, the strength of clinical evidence from studies supporting these accelerated approvals is not always high.

Through the accelerated approval pathway, the FDA allows new drugs to come to market that treat serious conditions, based on pivotal trials that use surrogate endpoints or intermediate clinical endpoints that are “reasonably likely” to predict the clinical benefit. Between 2015 and 2022, 159 drug-indication pairs received accelerated approval. Three pairs were excluded due to absence of the product label or FDA review documents in the Drugs@FDA database. Among the remaining 156 approved drug-indication pairs, 77% were supported with evidence from single-arm pre-approval pivotal studies — trials that had no comparators — and 22% from Phase I trials, according to a recent study published by the BMC Medicine journal. The median number of participants in those trials was 92.

While many retail pharmacies are backing off from their provider group subsidiaries, private-equity groups continue to invest in the space, activity that is likely to continue under the new administration. Roll-up mergers have been of great interest to drug wholesalers, which have become almost as vertically integrated as the major PBM/insurer entities. As pressure grows on the buy-and-bill system, this integration will be a big story over the next couple of years, asserted longtime industry expert Adam J. Fein, Ph.D., president of Drug Channels Institute, an HMP Global Company, during a Dec. 13 webinar.

Taking a broad look at the drug channel, Fein observed that private-equity groups have become a “big part of health care markets.” Retail pharmacies had been acquiring primary care organizations that were started by venture capital and private-equity companies but “have now realized that’s not such a great business.” He pointed to Walgreens Boots Alliance, which has considered selling VillageMD and, more recently, is reportedly in talks to sell itself to private-equity group Sycamore Partners, according to The Wall Street Journal.