Radar on Specialty Pharmacy

Almost two months after a U.S. district court judge struck down a federal rule allowing health plans to not count copayment assistance against members’ out-of-pocket costs, ruling in favor of patient advocacy groups in a lawsuit against HHS, the agency has signaled that it will not — at least for the time being — take action against plans based on how they treat that assistance. The agency in a recent court filing also said it plans to issue rulemaking in response to the September ruling and requested feedback from the judge on his decision. Shortly thereafter, the plaintiffs appealed the government’s move.

To help patients pay for pricy therapies — usually specialty drugs — pharmaceutical manufacturers offer assistance that can help cover their out-of-pocket costs. Companies claim that the assistance helps improve patient adherence to medications that often treat rare and deadly conditions. But critics of them say such programs incentivize drugmakers to raise prices of these agents.

The US Food and Drug Administration’s simultaneous approval of two gene therapies for sickle cell disease from Vertex Pharmaceuticals Incorporated/CRISPR Therapeutics AG and bluebird bio on 8 December provides the competitors an equal start out of the gate, and offers another test for the Rx policy concept that intra-class competition can drive down prices.

Based on the initial list prices, though, it seems like perhaps competition cannot do that, at least not in this case, or at least not yet. Bluebird bio’s Lyfgenia has a wholesale acquisition cost of $3.1m, while the WAC for Vertex and CRISPR’s Casgevy is $2.2m, which might be a significant handicap for bluebird in securing reimbursement.

The ulcerative colitis (UC) space has seen multiple new approvals recently that could focus payer management even more on the condition. Payers already take an aggressive approach toward managing branded agents within the class, according to a Zitter Insights survey. That stance, say industry experts, will only grow with the new drugs and administration routes.

While no cure exists for the inflammatory bowel disease, the FDA has approved numerous agents to treat signs and symptoms of the condition. In addition to the nine biosimilars of AbbVie Inc.’s Humira (adalimumab) that have launched in the U.S. in 2023, a handful of other approvals happened from late September through late October. All of the agents offer maintenance dosing either through an oral or subcutaneous route of administration.

Bayer recently revealed that it will work with the FDA to voluntarily withdraw the New Drug Application (NDA) for its cancer drug Aliqopa (copanlisib). The therapy is the latest phosphoinositide 3-kinase — also called phosphatidylinositol 3-kinase — (PI3K) inhibitor/indication with accelerated approval to treat a hematologic malignancy to be pulled from the U.S. market, potentially spurring payers to take a closer look at these agents.

The agency gave Aliqopa accelerated approval on Sept. 14, 2017, for the treatment of adults with relapsed follicular lymphoma (FL) who have received at least two systemic therapies. Approval was based on the CHRONOS-1 Phase II clinical trial. In the confirmatory study, CHRONOS-4, adding Aliqopa to standard immunochemotherapy regimens did not meet the primary endpoint of progression-free survival (PFS) vs. the standard immunochemotherapy control arm. Bayer says it will publish the trial results “in a timely manner.”

Scrutiny of the FDA’s accelerated approval process has shown no signs of slowing, with an FDA committee holding a recent meeting on the program’s use for oncology indications, as well as on two cancer drugs lacking confirmatory trial data long after their accelerated approvals. And an FDA leader recently asserted his stance on granting the designation only when companies already have started postmarketing trials. Still, these developments should not dampen interest by either manufacturers or payers in drugs hitting the market via the accelerated pathway, says one industry expert.

The FDA established the accelerated approval pathway in 1992 to bring HIV/AIDS medicines onto the market sooner during the ongoing epidemic. Between that time and Dec. 31, 2020, the agency has granted more than 253 accelerated approvals, according to a 2021 report from the Institute for Clinical and Economic Review (ICER) examining the designation. Of those, 125 — 49.4% — later received full approval, with a median time of 3.2 years from accelerated approval to full. Sixteen — 6.3% — of the indications have been withdrawn, and the remaining 112 drugs have been on the market for a median of 1.9 years.