Specialty Drugs

All Eyes Are on Novel New Therapies, Impact of IRA, AI

The FDA approved the highest number of novel therapies in years in 2023, and multiple potential blockbusters are slated for decisions in 2024. In addition, the Inflation Reduction Act looms large over the pharma industry as HHS negotiates the prices of the initial 10 Medicare Part D agents and lawsuits over the legality of the IRA play out. And while life sciences dealmaking was down a bit in 2023, the new year already looks to be more promising. AIS Health, a division of MMIT, spoke with some industry experts about their outlook for 2024.

AIS Health: What are some pharma issues to keep an eye on in 2024, and why?

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FDA Approves Game-Changing Regimen for Urothelial Cancer, but Will Cost Pose Issue?

In late 2023, the FDA approved a new treatment regimen for urothelial cancer that represents a “paradigm change,” according to one industry expert. The cost of the therapy, however, could pose a barrier to its uptake.

On Dec. 15, 2023, the FDA approved Pfizer Inc. and Astellas Pharma Inc.’s Padcev (enfortumab vedotin-ejfv) in combination with Merck & Co., Inc.’s Keytruda (pembrolizumab) for the treatment of people with locally advanced or metastatic urothelial cancer (la/mUC). The agency gave accelerated approval to this combination for people with la/mUC who are not eligible for cisplatin-containing chemotherapy on April 3, 2023. The newest approval converts that accelerated approval to full and expands the labeled indication to include the treatment of people eligible for cisplatin chemotherapy. The application had priority review and breakthrough therapy designation.

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Copay Accumulator Lawsuit Comes to an End; Will Ruling Be Enforced?

An ongoing lawsuit over the use of copay accumulators is drawing to a close following the defendants’ and plaintiffs’ motions to dismiss their appeals. The ball is now in the federal government’s and state insurance commissioners’ courts to enforce a district court judge’s ruling, which states that manufacturer assistance must be counted toward patients’ out-of-pocket responsibility unless a brand-name drug has a medically appropriate generic equivalent.

Health plans and PBMs several years ago began implementing copay accumulators — and then a new iteration known as copay maximizers that declare certain drugs non-essential health benefits to avoid covering them per the Affordable Care Act (ACA) — to counter manufacturer copay assistance programs. Before these tools, that assistance would count toward beneficiaries’ annual out-of-pocket expenses. When those out-of-pocket maximums were reached, health plans would cover the remainder of members’ costs for the year. With accumulators and maximizers, patients can still use that assistance, but it does not help reduce their out-of-pocket costs.

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Incorporating Pharmacy Spending in Value-Based Payment Models Remains Challenging

Payers face challenges incorporating drug spending into value-based payment models, making it difficult to reduce overall health care costs, according to panelists who participated in a session at the virtual Value-Based Payment Summit on Jan. 31. The speakers were encouraged with the increased attention being paid to expensive medications and difficulties in ensuring people take their prescriptions. However, they stressed that more needs to be done to educate providers and ensure pharmacy spending is in check.

Frank W. McStay, II, assistant research director at the Duke University Margolis Institute for Health Policy and the session’s moderator, noted the federal government and states have recently proposed and implemented policies to reduce drug costs and increase access to medications. For instance, the Biden administration on Jan. 30 announced that it planned to increase access to sickle cell treatments via the Cell and Gene Therapy (CGT) Access Model that CMS introduced last year.

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Is Sale of Specialty Pharmacy Accelerator Shields in Walgreens’ Best Interests?

Less than two years after Walgreens Boots Alliance Inc.’s purchase of Shields Health Solutions, the pharmacy giant is rumored to be seeking a sale of the health system-owned specialty pharmacy integrator, per a Jan. 23 Bloomberg article, which valued the company at around $4 billion. While returns from a sale could help the company shore up certain units, divesting itself of Shields at a time when specialty pharmacy continues to be a hot commodity may be a questionable approach, say industry experts.

A Walgreens spokesperson told AIS Health, a division of MMIT, that the company has no comment.

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FDA Warns on CAR-Ts, but Benefits Outweigh Risks for Now

A couple of months after the FDA revealed that it was investigating the risks of chimeric antigen receptor T cell (CAR-T) immunotherapies, the agency is seeking updated language in their labels warning about those risks. However, the fact that the FDA is allowing the therapies to remain on the market suggests that their overall benefits outweigh their potential risks, says one industry expert.

In late November, the FDA revealed that it was investigating the risks of CAR-T therapies following reports of T cell malignancies in people who had undergone treatment with the agents. Less than two months later, the agency issued safety labeling change notification letters to the six available B cell maturation antigen (BCMA)-directed or CD-19 directed autologous CAR-Ts, which cover a range of hematologic cancers:

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News Briefs: CMS’s Cell and Gene Therapy Model Will First Focus on Sickle Cell Disease

CMS’s Cell and Gene Therapy Access Model’s first focus will be on therapies for sickle cell disease, the agency said Jan. 30. The administration unveiled the model almost one year ago as one of three new models for testing by CMS’s Innovation Center to lower the cost of drugs and increase access to new treatments. The model will implement outcomes-based agreements (OBAs) for cell and gene therapies to treat sickle cell disease beginning in 2025 “and may be expanded to other types of CGTs in the future.” The administration estimates that about half of people with sickle cell disease are enrolled in Medicaid, with health care services costing about $3 billion annually. Through the model, CMS will negotiate OBAs, and states can decide whether to enter into the arrangements. “CMS will negotiate financial and clinical outcome measures with drug manufacturers and then reconcile data, monitor results, and evaluate outcomes. The CGT Access Model will begin in January 2025, and states may choose to begin participation at a time of their choosing between January 2025 and January 2026.”

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New FDA Approvals: FDA Broadens Keytruda Use in Cervical Cancer

Jan. 12: The FDA broadened the label of Merck & Co., Inc.’s Keytruda (pembrolizumab) to include, in combination with chemoradiotherapy, the treatment of people with FIGO (International Federation of Gynecology and Obstetrics) 2014 Stage III-IVA cervical cancer. The agency initially approved the programmed death receptor-1 (PD-1) inhibitor on Sept. 4, 2014. The indication is the drug’s third in cervical cancer and 39th overall. The application was granted priority review. That review was conducted under Project Orbis in collaboration with the Australian Therapeutic Goods Administration and the Brazilian Health Regulatory Agency, and it used the Assessment Aid. Dosing is 200 mg every three weeks or 400 mg every six weeks via a 30-minute intravenous infusion. The agent’s price for every-three-weeks dosing is $11,115; for every-six-weeks dosing, it’s $22,230.

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Cell and Gene Therapies, Biosimilars, Medical Benefit Drugs Are Areas to Watch Moving Forward

While the FDA approved a large number of specialty drugs in 2023, payers shouldn’t let their attention wander in 2024. Multiple potential blockbusters are slated for decisions by the agency this year, as are several biosimilars and generic versions of specialty medications. Gene therapies will continue to garner headlines — and payer dollars. All of these trends and more point to an active year ahead within the specialty pharmacy space. AIS Health, a division of MMIT, spoke with some industry experts about what’s on board.

AIS Health: What are some specialty pharmacy issues to keep an eye on in 2024, and why?

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Copay Accumulator Lawsuit Comes to an End; Will Ruling Be Enforced?

An ongoing lawsuit over the use of copay accumulators is drawing to a close following the defendants’ and plaintiffs’ motions to dismiss their appeals. The ball is now in the federal government’s and state insurance commissioners’ courts to enforce a district court judge’s ruling, which states that manufacturer assistance must be counted toward patients’ out-of-pocket responsibility unless a brand-name drug has a medically appropriate generic equivalent.

Health plans and PBMs several years ago began implementing copay accumulators — and then a new iteration known as copay maximizers that declare certain drugs non-essential health benefits to avoid covering them per the Affordable Care Act (ACA) — to counter manufacturer copay assistance programs. Before these tools, that assistance would count toward beneficiaries’ annual out-of-pocket expenses. When those out-of-pocket maximums were reached, health plans would cover the remainder of members’ costs for the year. With accumulators and maximizers, patients can still use that assistance, but it does not help reduce those out-of-pocket costs.

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