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Using Randomized Trials, Real-World Evidence to Design Value-Based Contracts

June 04, 2019

According to recent data, real-world evidence (RWE) and randomized controlled trial evidence (RTC) significantly contribute to implementing successful value-based (VBC) or outcomes-based contracts.

“The use of value-based contracts, or performance-based risk sharing agreements has increased between manufacturers and payers to mitigate growth in drug and health care costs,” explained researchers Amanda Patrick, Todd Gorsuch, and Jeremy A Rassen of Aetion, New York, NY.

Currently known barriers to implementing outcomes-based contracts include:

  • Correctly estimated contact risk due to uncertainty and real-world target product performance;
  • Sufficient data infrastructure to execute and track outcomes;
  • Transparency and alignment and defining and measuring outcomes; and
  • Establishing causality between the biopharmaceutical intervention and target outcome(s).

In a project managed by Aetion—a health care technology company that delivers real-world evidence to determine which treatments work best, for whom, and when—the researchers compared the economic outcomes to the manufacturer under threshold assumptions drawn from RTC data vs outcomes based on RWE, identifying VBC structures as either: (1) absolutely threshold-based contracts, where the manufacturer is required to provide refunds on drug or medical costs if the outcome rate fails to meet a predetermined level or (2) comparative effectiveness performance contracts, where the manufacturer might be required to provide refunds if the performance improvement of drug vs standard of care comparator fails to meet the predetermined threshold.

Data was used from the phase 3 international, double blind randomized trial of SAC/VAL (PARADIGM HF). Researchers estimated the differential cost of care if the real-world benefit differed from the benefit reported in the RCT. “We assumed 2 million HF patients, 20% uptake of SAC/VAL, and a $12000 cost per HF hospitalization,” explained Ms Patrick and colleagues.

When implementing the hypothetical outcomes-based contracts, researchers identified results in two main categories, patient characteristics’ impact on absolute risk and impact on relative risk. “Under an absolute threshold based contract, a 1% absolute higher baseline risk in real world vs the trial would translate to 4000 excess cardiovascular deaths and HF hospitalizations per year, with consequent economic impact to the manufacturer of up to ~$29 million,” explained researchers. “In a recently published RWE paper, the 6-month risk was ~4% higher than that observed in PARADIGM-HF.

Other significant findings from the research project included:

  • VBCs are typically based on RCT findings but they are implemented in the real world.
  • In order to anticipate how the drug will likely perform in the local population, RWE on patient characteristics and baseline rates of outcomes is needed in addition to the RCT results.
  • For VBCs based on comparative effectiveness, risk adjustment is key to obtaining valid results.
  • Reliance on RCT evidence alone carries significant risks both for manufacturer and payer.     

This information was presented at ISPOR’s 2019 Annual Meeting.

Edan Stanley


Patrick A, Gorsuch T, and Rassen JA. Informing the design of value-based contracts: use of randomized trial and real-world evidence. Poster presented at: ISPOR; May 18-22, 2019; New Orleans, LA.

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