Reimagining Value-Based Deals Through Risk-Pooling Models

While manufacturers are producing one-time therapies with a decade or more of durable benefit, the systems built to pay for them still run on assumptions from decades ago, pointed out Chester B. Good, MD, MPH, senior medical director at the UPMC Center for Value-Based Pharmacy Initiatives and professor of medicine and pharmacy at the University of Pittsburgh School of Medicine, to open a panel on value-based contracting at the National Association of Specialty Pharmacy's Inspire 2026 conference.

Good was joined by Tay Salimullah, founder of Renovamen Advisors, and Donald Vidic, executive advisor at Free Market Health, to discuss why value-based contracting has cooled since its peak years and what might revive it for cell and gene therapies.

Value-Based Contracting Timeline

Good, who helped build UPMC's value-based contracting program after being recruited in 2017, said interest in these deals was strong when the Affordable Care Act first pushed value into the conversation, and at least one of UPMC's original contracts remains among its best performing. That momentum broke in 2020, he said, when COVID-19 disrupted existing agreements, and a wave of regulatory change followed, including the Inflation Reduction Act's drug pricing provisions, Most Favored Nation policy, and Medicaid best-price rules. Two of those actions have since moved from proposal to practice: the Medicare Drug Price Negotiation Program created by the Inflation Reduction Act put its first negotiated prices, covering 10 Part D drugs, into effect on January 1, 2026,¹ and a Most Favored Nation executive order signed in May 2025 directed federal agencies to tie US drug prices to the lowest amount paid by other wealthy nations.² Rising rebates without corresponding drops in net cost have added further pressure, Good said, leaving fewer new contracts even as cell and gene therapy pipelines expand.

Salimullah described himself as a former manufacturer executive who helped bring pediatric chimeric antigen receptor (CAR) T-cell therapy out of the laboratory of Carl June, MD, an immunologist at the University of Pennsylvania's Perelman School of Medicine,³ and later oversaw treatment of more than 5000 babies with spinal muscular atrophy (SMA). Those experiences track 2 of the field's highest-profile products: tisagenlecleucel (Kymriah), the CAR T-cell therapy Novartis developed from research at the University of Pennsylvania, which became the first FDA-approved CAR T-cell therapy in August 2017 for patients up to age 25 with relapsed or refractory B-cell precursor acute lymphoblastic leukemia,⁴ and onasemnogene abeparvovec (Zolgensma; Novartis Gene Therapies), which the FDA approved in 2019 for children younger than 2 years old.⁵

Citing abeparvovec during the panel, Salimullah said its pivotal trial enrolled children only up to about 6 months old, even though the approved label covers children up to age 2. FDA review documents show the trial's 36 patients were dosed between about 2 weeks and 8 months old, consistent with what he described, while the labeled population extends considerably further, to children just under 2 years old.

Salimullah argued the underlying clinical case for these therapies is no longer in question: patients treated with a single infusion in 2020 remain durably well as of late 2026, he said, and children treated for SMA have shown durable benefit for more than 10 years in some cases. What has historically stalled value-based arrangements for these therapies is the payer side: the ability to track patients as they churn between plans and the challenge of paying now for a benefit that may not be realized or captured by the same payer for years. But he argued those barriers no longer hold up given how far tracking technology has come since these conversations began roughly five to six years ago.

"There's no excuse for one-time, multimillion-dollar therapies that are going to hit the market, that are just going to address the root cause of the disease, disrupt the natural history, or halt the disease," Salimullah said.

Specialty Pharmacy's Limited Role

Vidic said specialty pharmacies are well positioned to support value-based deals because they have monthly contact with patients, unlike physicians who may see them only every few months. But he said specialty's participation has been limited by fragmented data: commercial, Medicare, and self-funded plan business is scattered across lines that don't give any single pharmacy a full view of a patient's medical claims or cost offsets.

Without agreement between payers and manufacturers on which outcomes to measure, he said, specialty pharmacies are "waiting patiently to be a part of the solution" rather than driving it.

The Road Ahead for High-Cost Therapy Deals

Looking ahead, Salimullah pointed to emerging models that pool risk across multiple health plans through reinsurance, paired with per-member fees and longitudinal data platforms run by third-party technology companies, as a way to guarantee patients access on the day a therapy is approved rather than leaving smaller employer plans to carve out coverage on their own. He framed the shift as inevitable rather than optional, predicting that some form of outcomes guarantee will become standard for high-cost one-time therapies regardless of what it is called.

"Better to embrace it," Salimullah said, "and make sure we get these therapies to patients who desperately need them."

References

1. Key Facts About Medicare Drug Price Negotiation. KFF. Accessed September 24, 2026. https://www.kff.org/medicare/key-facts-about-medicare-drug-price-negotiation/
2. Rogers HA. Most-Favored-Nation Prescription Drug Pricing Executive Order: Legal Issues. Congressional Research Service. June 5, 2025. Accessed September 24, 2026. https://www.congress.gov/crs-product/LSB11319
3. Joszt L. FDA approves tisagenlecleucel, the first CAR-t cell therapy in the United States. AJMC^®. August 30, 2017. Accessed September 25, 2026. https://www.ajmc.com/view/fda-approves-tisagenlecleucel-the-first-car-t-cell-therapy-in-the-united-states
4. FDA approves tisagenlecleucel for B-cell ALL and tocilizumab for cytokine release syndrome. US Food and Drug Administration. August 30, 2017. Accessed September 24, 2026. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-tisagenlecleucel-b-cell-all-and-tocilizumab-cytokine-release-syndrome
5. FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality. US Food and Drug Administration. May 24, 2019. Accessed September 24, 2026. https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease