AMCP 2026 Spotlights New Era for Specialty Drug Market

The specialty drug market is being reshaped by patent expirations; a faster, less predictable FDA; and precision oncology and rare disease innovations, requiring adaptive planning from managed care professionals, an expert emphasized at the Academy of Managed Care Pharmacy (AMCP) 2026 meeting in Nashville, Tennessee.¹

AMCP Returns to Music City

The AMCP 2026 annual meeting brought together managed care professionals in Music City last week. The organization returned to its roots, as the first-ever AMCP meeting was held there in 1989, but attendance increased from 40 people to approximately 4000.²

Through educational sessions, poster presentations, and more, the meeting explored hot topics, including the federal regulatory landscape, artificial intelligence, and glucagon-like peptide-1 receptor agonist use.

Before adjourning last Thursday, Nicole A. Caffiero, PharmD, MBA, BCACP, a senior clinical pharmacist at Evernorth Health Services, led the final keynote session, “Emerging Specialty Pharmaceuticals: Current Development Landscape and Why It Matters.”¹ As part of the last session, she highlighted specialty market trends shaping the drug pipeline.

Competition-Driven Savings Reshape Specialty Drug Landscape

Caffiero began by highlighting the impact of biosimilar/generic competition with brand drugs. She identified competition as a core, recurring theme in the specialty market, driven by generics and biosimilars as high-cost drugs lose exclusivity, with generics addressing small-molecule drugs and biosimilars addressing biologics.

For decades, patent expiration primarily impacted traditional primary-care drugs. However, following the first major generic wave triggered by Prozac's patent loss in 2001, the industry pivoted toward specialty and rare disease therapies. Now, specialty drugs are beginning to lose exclusivity, with substantial savings opportunities in a highly concentrated number of products.

She highlighted that, over the next 5 years, roughly $137 billion in brand sales are at risk, with about half attributable to specialty medications. In 2026 alone, more than $43 billion in brand sales face generic competition, with more than half of that value in diabetes and other traditional drugs and the rest coming from first-time generic specialty drugs.

Regarding biosimilars, Caffiero noted that the FDA has now approved 83, accelerating sharply to 18 per year in 2024 and 2025. A total of 65 have been launched to date, targeting 17 reference biologics. She highlighted that a cluster of major biologics will generate the next wave, with nearly $80 billion in potential savings through 2030. Beyond 2030, Caffiero explained that future savings will depend on the uptake of a handful of high-impact biosimilars rather than volume alone.

Over the next decade, approximately 118 biologics are expected to lose exclusivity. However, she noted there is currently a “biosimilar void” as a modest number of biosimilars are in development, leaving more than $230 billion in potential spending at risk if competition fails to materialize.

At the same time, the FDA has revised biosimilar guidance to reduce the development burden and streamline clinical requirements. Consequently, major manufacturers such as Sandoz and Teva are expanding biosimilar strategies. If the pipeline fills in and competition expands, Caffiero highlighted that some describe the coming years as a possible “golden decade” for biosimilars.

“While today, the pipeline looks thin, the structural conditions for a golden decade are taking shape, so we will see how this plays out,” she said.

Oncology, Rare Diseases Emerge as the Core of Specialty Innovation

Next, Caffiero underscored oncology and orphan/rare disease as the 2 therapeutic areas that now define the specialty market, as they represent more than 85% of the pipeline.

She emphasized that oncology innovation is not just accelerating but also becoming more precise. With 92 cancer drugs approved overall and 16 novel agents approved in 2025 alone, the field has shifted away from treating cancer broadly toward more biomarker-driven, narrowly defined patient populations.

“We are matching treatment to what is actually driving the tumor, and that shift matters because it's allowing for better outcomes and better tolerability,” Caffiero said. “Really, the takeaway here is that oncology innovation isn't just accelerating; it's becoming smarter.”

Additionally, despite their label, rare diseases have become more central. Since the Orphan Drug Act of 1983, more than 500 therapies have been approved for rare conditions. Although each orphan disease affects fewer than 200,000 Americans, she noted that there are approximately 10,000 such diseases, meaning 1 in 10 Americans live with a rare condition, with more than 95% of those diseases currently lacking an approved treatment. At the same time, orphan drugs accounted for more than half of all FDA approvals in 2025 and make up roughly 40% of the specialty pipeline.

“This tells us where innovation is being directed, and it really reinforces that orphan and oncology drugs are no longer niche areas; they are the core specialty drug development,” Caffiero said.

Faster, Less Predictable FDA Reshapes Specialty Market

Lastly, the FDA is approving drugs more quickly and with more transparency, but it has also become less predictable. She highlighted several workforce disruptions resulting from staffing cuts and leadership turnover. As a result, advisory meetings have halted, with none in the last 9 months of 2025, reducing the external guidance and predictability that sponsors and payers rely on.

Complete response letters (CRLs), which signal FDA rejection or a request for additional data, also rose in frequency in 2025. On top of this, the FDA has begun publicly releasing CRLs shortly after issuance and has made historical CRLs available. Caffiero noted that this increases transparency, but it also makes setbacks immediately visible, raising pressure on sponsors to provide stronger data packages from the outset.

At the same time, speed is being increasingly prioritized with new accelerated review mechanisms. The Commissioner's National Priority Review Voucher is the most aggressive and can compress review timelines from 10 to 12 months down to 1 to 2 months. It enables rapid approvals but leaves less margin for error and time for payers to prepare coverage and management strategies. She underscored that uncertainty has now shifted more to the postapproval period for regulators, payers, and providers alike.

In this new environment, the FDA approved 58 new therapies in 2025, 72% of which were specialty drugs and 52% of which carried orphan designations. The agency also granted over 150 expanded indications, further blurring the line between traditional and specialty categories, as some traditional drugs gained specialty indications and other specialty drugs moved into broader use.

Regarding spending, total pharmacy costs reached $1613 per member per year (PMPY), up $162. Despite being used by only about 2% of patients, specialty drugs now account for roughly 50% of that spending. Caffiero said this statistic exemplifies cost concentration at work, with growth centered on a small number of fast-moving drugs rather than broad utilization trends.

Inflammatory conditions remain the largest specialty spend category at approximately $400 PMPY. Although biosimilars bring savings, she explained that new agents and rising utilization offset these.

Although oncology is the second-largest category by spend on the pharmacy benefit side, 80% of total oncology spend is captured under the medical benefit. Meanwhile, HIV spending is increasing due to utilization growth, but multiple sclerosis spending is declining as generics enter the market.

Planning Amid Uncertainty in an Evolving Specialty Landscape

To conclude, Caffiero emphasized the uncertainty in today’s specialty market, particularly around approval timelines and data required for approval. As a result, she recommended that managed care professionals adopt adaptive planning strategies to prepare for continued unpredictability.

“I just think there's a lot of planning that needs to happen, which there always has been, but I think that the uncertainty is even more than it has been in the past,” she said. “So, just planning, keeping up, and evolving with the data so coverage decisions are not a one-time decision. I think making sure that we continue to evolve as more data comes out in this postapproval period is going to be really important.”

References

1. Caffiero NA, DiPietro R. Emerging specialty pharmaceuticals: current development landscape and why it matters. Presented at: AMCP 2026; April 13-16, 2026; Nashville, TN.
2. McCormick B. AMCP returns to Nashville roots for 2026 meeting. AJMC^®. April 9, 2026. Accessed April 23, 2026. https://www.ajmc.com/view/amcp-returns-to-nashville-roots-for-2026-meeting