RADAR on Specialty Pharmacy

Medicare Plans to Cover Aduhelm but With Certain Restrictions

To say that the FDA’s approval of Biogen and Eisai, Co., Ltd.’s Alzheimer’s disease treatment Aduhelm (aducanumab-avwa) on June 7, 2021, garnered an immense amount of attention would seem to be an understatement. That said, the drug has somehow gathered even more notice over the past few months due to multiple developments, with CMS most recently issuing a proposed National Coverage Determination (NCD) on Aduhelm and other monoclonal antibodies that target beta amyloid plaque that will allow Medicare coverage for the therapies but only under certain circumstances. While commercial payers often follow CMS’s lead, it remains to be seen whether that decision — plus a dramatic price cut on Aduhelm — will prompt payers that have declined to cover the therapy to change course.

There is a 30-day public comment period on the proposed NCD, which was published Jan. 11. A final decision is expected on April 11.

Notable 2021 Approvals Included Oncolytics, Interchangeables

Between the FDA’s Center for Drug Evaluation and Research (CDER) and its Center for Biologics Evaluation and Research (CBER), the agency approved 60 new products in 2021, including biologics, cell and gene therapies, vaccines and blood products. As the COVID-19 pandemic continued for the second year, much of the pharma industry’s focus, understandably, was on vaccines and therapeutics for the coronavirus. As with prior years, oncology dominated the specialty drug space, but the FDA also approved novel treatments in other areas as manufacturers continued to pursue innovative new products. Moreover, the U.S. saw the first two interchangeable biosimilars gain approval. AIS Health, a division of MMIT, spoke with various industry experts about what they considered the most notable FDA approvals in 2021.

Many Melanoma Therapies Exist, but Treatment Remains Challenging

Since 2011, the FDA has approved multiple 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, filling an unmet need, industry experts note. However, the condition is complex to treat and may be challenging for health plans to manage.

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.

Specialty Pharmacy, Home Infusion Spaces Saw Myriad 2021 Changes

While the COVID-19 pandemic continued to impact the specialty pharmacy and home infusion spaces for the second straight year, other events also played a role. AIS Health spoke to some industry experts about 2021’s impact.

AIS Health: Looking back over the past year, what do you think were the most noteworthy occurrences within the specialty pharmacy industry, and why?

Dea Belazi, Pharm.D., M.P.H., president and CEO of AscellaHealth: Some of the noteworthy trends in SP have included the continued rise of the cost of specialty agents within health care expenditures, the continued emergence of ultra-high-cost specialty agents for rare and orphan diseases and the continued vertical integration of specialty pharmacies into health care organizations.

New FDA Approvals: FDA Approves Additional Indication for FoundationOne CDx | Jan. 13, 2022

Dec. 8: The FDA gave another approval to Foundation Medicine, Inc.’s FoundationOne CDx as a companion diagnostic for current and future BRAF inhibitors used to treat melanoma, including monotherapies targeting BRAF V600E and BRAF/MEK inhibitor combination therapies targeting BRAF V600E or V600K mutations. The agency first approved the test on Nov. 30, 2017.

Dec. 9: The FDA granted premarket approval to Thermo Fisher Scientific’s Oncomine Dx Target Test as a companion diagnostic to help identify people with non-small cell lung cancer whose tumors have epidermal growth factor receptor (EGFR) exon 20-insertion mutations who may be candidates for treatment with Rybrevant (amivantamab-vmjw) from Janssen Biotech, Inc., a Johnson & Johnson company. The agency initially approved the test on June 22, 2017.

News Briefs: CMS Rescinds Most Favored Nation Model | Jan. 13, 2022

CMS issued a final rule on Dec. 29 that rescinded the Most Favored Nation model. The mandatory model would have priced Medicare Part B drugs on the U.S. market based on their prices in certain countries. An interim final rule that was published in November 2020 had been blocked from being implemented on Jan. 1, 2021.

CMS published a proposed rule on Jan. 12 that would rein in direct and indirect remuneration (DIR) fees, which pharmacies have long complained about. The proposal would save consumers about $21.3 billion but cost the federal government $40.0 billion from 2023 through 2032.

Study: Opportunities to Improve Management of PNH Exist

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, 2019. 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.

Biomarker Testing Is Not Keeping Up With Drug Innovations

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.

Scemblix Is First-in-Class STAMP Inhibitor for CML

The FDA recently approved a first-in-class agent for the treatment of chronic myeloid leukemia (CML). The drug’s novel mechanism of action may offer an improvement over other therapies within the class, industry experts say.

On Oct. 29, the FDA approved Novartis Pharmaceuticals Corp.’s Scemblix (asciminib) for the treatment of CML in two indications: (1) adults with Philadelphia chromosome-positive CML in chronic phase previously treated with at least two tyrosine kinase inhibitors (TKIs), which was given accelerated approval, and (2) adults with PH+ CML-CP with the T315I mutation, which was granted full approval. It is the first FDA approval of a CML therapy that is a Specifically Targeting the ABL Myristoyl Pocket (STAMP) inhibitor.

Two More Accelerated Approval Indications Are Being Pulled

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.