Spotlight on Market Access

On Jan. 10, the U.S. reported 1.35 million new coronavirus cases as the highly contagious Omicron variant swept across the nation, breaking the previous daily record of 1.03 million. While a COVID-19 vaccine booster could increase immune response, the Centers for Disease Control and Prevention’s data show that only 36.7% of fully vaccinated people have received a booster dose nationwide, as of Jan. 11, 2022. Doubling the pace of booster vaccinations per day could prevent more than 41,000 deaths and 400,000 hospitalizations by the end of April, according to a recent analysis by The Commonwealth Fund.

As 2021 began, the pharmaceutical topic arguably drawing the most attention was COVID-19 and the rollout of vaccines against it. Moving into 2022, the COVID pandemic unfortunately continues to dominate headlines, as the U.S. marked 1 million new cases of COVID on Jan. 3, a single-day record for any country. However, it’s not all bad news, as the pandemic is continuing its impact across various services. For example, drugmakers are applying knowledge gained in their innovative efforts to develop vaccines and treatments to other areas of drug development, and a shift to virtual operations and health care is expected to continue.

AIS Health spoke with a variety of industry experts about their 2022 projections for pharma.

Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare, potentially fatal disease. The FDA approved the first therapy, Alexion Pharmaceuticals, Inc.’s Soliris (eculizumab), a complement C5 inhibitor (C5I), to treat adults with the condition on March 16, 2007. Then the agency approved another C5I to treat adults with PNH from the same company, Ultomiris (ravulizumab-cwvz), on Dec. 21, 2018. A recent study of those two therapies from Prime Therapeutics LLC shows that there may be opportunities for payers in PNH management that will result in better health care outcomes for their members.

Prime presented findings from the study at the Academy of Managed Care Pharmacy (AMCP) Nexus meeting, which was held Oct. 18 through 21 in Denver. Funding for the study was provided by Apellis Pharmaceuticals, Inc., manufacturer of Empaveli (pegcetacoplan), a C3 inhibitor approved May 14, 2021, for the treatment of PNH in treatment-naïve people, as well as ones switching from any C5I. Researchers were from Prime, Apellis and the University of Minnesota College of Pharmacy.

Within the span of one week, Secura Bio, Inc. has revealed that it will withdraw one oncology drug from the U.S. market, as well as an indication for another oncolytic. The FDA had given both accelerated approval. The moves come amid growing scrutiny of that approval pathway, and they mark the ninth and 10th oncology indications and/or drugs taken off the U.S. market since December 2020.

Secura Bio said on Nov. 30 that it will withdraw the new drug application for Farydak (panobinostat). The FDA granted the capsule accelerated approval on Feb. 23, 2015, for the drug in combination with Takeda Pharmaceuticals U.S.A., Inc.’s Velcade (bortezomib) and dexamethasone for the treatment of people with multiple myeloma who have received at least two regimens, including Velcade and an immunomodulatory agent. That indication is the only one the drug has in the U.S. The company said it will continue to market the drug in other areas in which it is approved.

Founded as a consumer-oriented, technology-focused startup in 2015, Bright Health has quickly made its presence known in the health care industry, despite its newcomer status. The company launched its first product offerings in Colorado's individual market in 2016 and has since expanded to offering individual, Medicare Advantage (MA) and small group plans in 17 states. Bright Health's value-based care model revolves around partnerships with select health and hospital networks, including Tenet Health, Novant Health and HCA Healthcare, emphasizing the importance of the patient-provider relationship. CVS Caremark serves as Bright Health's pharmacy benefits manager.

Almost 12.8 million adults delayed or did not get prescription drugs in 2018-19 due to costs, including about 3.8 million privately insured nonelderly adults and 2.3 million elderly Medicare beneficiaries, according to a study by the Robert Wood Johnson Foundation and Urban Institute based on 2018–19 data from the Medical Expenditure Panel Survey. About 9.5% of adults who were uninsured all year reported unmet prescription drug needs, compared with 4.9% of Medicare beneficiaries and 5.6% of nonelderly Medicaid enrollees. More than one-quarter of adults with Medicare and 5.3% of privately insured people spent more than 1% of their family incomes on out-of-pocket prescription drug costs. In November, the House of Representatives passed the Build Back Better Act, which includes provisions seeking to bring down drug costs, but the bill’s fate in the Senate is in peril.

While the COVID-19 pandemic still drove a lot of the conversation around the pharmaceutical industry over the past year, much of it was focused on the development of vaccines and their rollout starting in late 2020, as well as treatments for the virus. But other innovative agents also came to market in 2021, continuing the industry’s trend of producing pioneering products. AIS Health, a division of MMIT, spoke to industry experts about the impact of COVID-19 on the industry and other 2021 pharma trends.

Despite the tremendous progress made over the past decade in developing targeted oncolytic therapies, testing for specific mutations —or biomarkers — to determine the proper candidates for those treatments lags behind. A few states have passed or are considering legislation requiring insurers to cover biomarker testing, with California becoming the most recent state to pass such a law.

Non-small cell lung cancer (NSCLC) is one area that has undergone the most development. There are now more than two dozen drugs that target nine different changes in NSCLC cells that help them grow. But an article published July 7 in Cancer Therapy Advisor — which summarized a presentation at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting — revealed that less than half of people with metastatic NSCLC received a test for five analyzed biomarkers before starting first-line treatment.

Since 2011, the FDA has approved 10 therapies for advanced or late-stage melanoma. Recently the agency granted an additional approval to one of those drugs for the earlier stage melanoma setting.

On Dec. 3, the FDA approved Merck & Co., Inc.’s programmed death receptor-1 (PD-1) inhibitor Keytruda (pembrolizumab) for the adjuvant treatment of people at least 12 years old with stage IIB or IIC melanoma following complete resection. The agency also expanded the indication for the agent’s use as an adjuvant treatment of stage III melanoma following complete resection to include pediatric patients at least 12 years old.

A growing share of patients have been prescribed oral targeted cancer drugs without documented overall survival (OS) benefit — from 12.7% in 2011 to 58.8% in 2018, according to a JAMA study, which analyzed dispensing claims for oral cancer drugs first approved by the FDA between Jan. 1, 2011, and Dec. 31, 2018. Cumulative spending on all 44 sample drugs reached $3.5 billion by the end of 2018, and 96.8% of that spending was on drugs approved based on a pivotal randomized clinical trial (RCT). Meanwhile, cumulative spending on drugs without documented OS benefit surpassed that on drugs with a documented benefit by the end of 2018. Among the top 20 drugs by spending amounts in 2018, one drug lacked a pivotal RCT and 13 drugs had no documented OS evidence at the time.