Approval Is Just One Piece of the Specialty Drug Pie

The FDA could approve a record number of new therapies this year, but a Wednesday session at the Academy of Managed Care Pharmacy Nexus 2026 meeting argued that that number tells payers little about what comes next. “The number of approvals is really only 1 piece of the pie,” said Nicole A. Caffiero, PharmD, MBA, BCACP, senior clinical pharmacist, Evernorth Health Services, who moderated the session, “Ahead of the Curve: Emerging Specialty Therapies and Managed Care Readiness.”

Fifty-eight new therapies were approved by the FDA in 2025, and nearly three-quarters of them were specialty medications, Caffiero said. Through this year’s session date, there had been 53 novel approvals, and 36 of them were specialty medications. The agency is on track for an estimated 79 approvals in 2026, which would top the 72 drugs approved in 2023.¹

Evernorth’s 2025 figures show specialty drugs made up about half of the per-member-per-year pharmacy spend, even though only about 4% of patients use them. Specialty utilization rose 11.7%. The Pharmaceutical Strategies Group projects that specialty spending will rise another 32% by 2028, and Evernorth forecasts that specialty trend holding near 11%.² The near-term pipeline through the first half of 2027 holds 68 specialty products: 43 novel therapies and 25 new indications. Just over 60% are entering already crowded classes, and roughly a third carry orphan or rare disease positioning.

Instead of walking through all 68, Caffiero organized the session around the 4 forces she believes are shaping the specialty pipeline: gene and cell therapies, rare and orphan diseases, crowded therapy classes, and care delivery innovation. She was joined by Katelyn B. Meyer, PharmD, clinical pharmacy manager, MassHealth, and Casey Koch, PharmD, MBA, clinical pharmacist, SelectHealth.

Gene and Cell Therapies: Is Uncertainty the Baseline?

Gene and cell therapies often reach the market through expedited pathways, with surrogate end points and little long-term data. Caffiero pointed to rebisufligene etisparvovec (Fayuvi; Ultragenyx), which is approved to treat mucopolysaccharidosis IIIA.³ The full approval was based on outcomes vs an external natural-history control. By contrast, the investigational doruxapapogene ralaplasmid (Inovio) for respiratory papillomatosis is seeking accelerated approval, although the FDA has questioned whether surgery reduction supports that pathway, Caffiero explained.

Meyer said readiness does not require every answer. “Organization readiness doesn’t mean that we have every answer. It means that we understand the uncertainty well enough that we can make a defensible coverage decision,” she said. In Massachusetts, she turns to local treatment centers for clinical perspective and estimates how many patients the plan will realistically see.

Medicaid faces added constraints, she added. Because MassHealth covers about half of the state’s children and many of these drugs treat life-limiting childhood diseases, Medicaid sees a disproportionate share of these patients while working with a limited state budget. They are priced as if they’re curative,” Meyer said, so shared risk becomes a real question when they are not.

Unlike other plans, which may be able to exclude a drug while waiting for better data, Medicaid programs generally cannot when a manufacturer participates in the Medicaid Drug Rebate Program. “It’s coverage in exchange for rebates,” Myers said, although states can still set clinical criteria and use utilization management. She invited manufacturers to discuss value-based outcomes agreements.

The “Orphan” Designation Doesn’t Tell Payers Enough

This categorization, Caffiero said, “identifies the regulatory category; it doesn’t necessarily define the management model.” Session attendees rated its usefulness an average of 4.33 on a 10-point scale. However, challenges vary by drug. Some conditions lack a diagnostic code, Koch noted, forcing plans to build algorithms to estimate patient counts. Others require subtyping. Small trials in ultra-rare disease are not automatically a red flag, Caffiero said; what matters more is whether the totality of the evidence is strong enough to support the drug’s claimed benefit.

How should payers prioritize then? Koch starts with budget impact: the eligible population multiplied by cost. Meyer ranks unmet need alongside cost and notes that a pharmacy-dispensed drug reaches patients much faster than a gene therapy that may take months to set up at a qualified treatment center.

Koch added that “just getting FDA approval is no longer a baseline guarantee [of] coverage.” Reviews do not stop at launch, the panelists concurred, especially for accelerated approvals that must confirm benefit later.

“Evidence evaluation is not a one-time thing,” Caffiero said.

Crowded Classes, Delivery Models, and Timing

In IgA nephropathy, myasthenia gravis, multiple sclerosis, and inflammatory conditions, Caffiero said, approval “earns entry, not adoption.” Audience members averaged 1.79 on a 5-point scale when asked how likely the next approval in a crowded class is to change practice meaningfully. Meyer sorted new entrants into 3 buckets—true clinical advances, line extensions, and just another option—which she sees as competition drivers.

Biosimilars are reshaping contracting. Meyer said a biosimilar expected within 6 months may keep a plan from signing a brand contract. She questioned the “biosimilar void,” noting blockbuster drugs have many biosimilars in development, while asking who will make them for rare disease therapeutics. Without head-to-head trials, the panel agreed, payers rely on indirect comparisons, provider input, and close study of trial populations.

Longer dosing intervals and subcutaneous delivery may shift sites of care and staffing. Psychedelic therapies, Caffiero said, are simple to administer but require a new ecosystem: preparation, a supervised session lasting as long as 10 hours, and integration follow-up. On timing, Koch said, “There’s probably a 3-month window for anticipated approval where it’s ideal to start getting some information and preparing for a drug.”

Meyer cautioned that some plans start 2 years early, only to see negative phase 3 results or a complete response letter. Koch said commercial plans can narrow coverage with inclusion/exclusion criteria, which may make planning more predicable than in Medicaid.

Caffiero stressed that “there’s a difference between priority and urgency.” For example, a prior authorization request arriving in the inbox cannot wait.

Key Takeaways

Caffiero summarized 4 overall lessons learned: prepare for evolving evidence, prioritize by expected impact, separate urgency from priority, and plan operations before approval. “Pipeline readiness isn’t just knowing what’s coming; it’s anticipating the decision that the therapies will create and preparing your organization to make those decisions when the therapy reaches the market,” she said.

Meyer and Koch said combining a gene therapy with ongoing treatment is generally investigational if the 2 were never studied together, and Meyer urged manufacturers to include standard of care in trials.

“Payers are going to follow the evidence, and if the evidence isn’t there, then it just isn’t there,” she said.

References

1. Compilation of CDER new molecular entity (NME) drug and new biologic approvals. FDA. Updated May 12, 2026. Accessed October 28, 2026. https://www.fda.gov/drugs/drug-approvals-and-databases/compilation-cder-new-molecular-entity-nme-drug-and-new-biologic-approvals
2. Myshko D. PSG: specialty drug spending predicted to increase 32% by 2028. Managed Healthcare Executive^®. July 30, 2026. Accessed October 28, 2026. https://www.managedhealthcareexecutive.com/view/psg-specialty-drug-spending-predicted-to-increase-32-by-2028
3. McCormick B. FDA approves first treatment for Sanfilippo syndrome type A. AJMC^®. September 21, 2026. Accessed October 28, 2026. https://www.ajmc.com/view/fda-approves-first-treatment-for-sanfilippo-syndrome-type-a