• Synthesizing Evidence to Secure Coverage and Reimbursement

    A key question for payers is whether a drug works as well in the real world as it did in the controlled environment of a clinical trial.

    Before adding a new drug to a health plan formulary and providing reimbursement, payers often require evidence that it is not only safe and efficacious but also effective in the real world. An important way that manufacturers provide this evidence is with formulary dossiers documenting the product and evidence of treatment effect (including comparisons with alternatives); disease characteristics and treatment options; clinical trial results; economic value; and other evidence. A dossier is a single document that ties together all the available data supporting the drug’s value.

    When available, real-world evidence (RWE) can be an essential part of a compelling evidence package for reimbursement submissions and health technology assessments (HTAs). Results from clinical trials may not be sufficient to support premium pricing because observations from populations that fit often-narrow study inclusion criteria may not be generalizable to health plan members. For example, patients with multiple comorbid conditions may be ineligible for a clinical trial, and patients are often less likely to adhere to prescribed treatment when not closely monitored.

    RWE is available from a wide range of sources, including observational studies of data such as patient registries, medical charts or electronic medical records (EMRs), administrative claims, and surveys of patients or providers.

    This evidence can help validate clinical trial results by:

    • Demonstrating safety and efficacy in patients with characteristics that were not included in the trial
    • Showing safety and efficacy over the long term, beyond the initial measurements made in primary study endpoints
    • Showing that patients not treated per a study protocol adhere to the recommended dose and administration schedule and derive the full benefit of treatment
    • Matching patient characteristics across different trials to compare whether one intervention is more effective than others in the relevant setting

    The value of a new treatment or technology depends on an intervention working as expected in the treated population, and an effective evidence package must be more than the sum of its individual parts. Dossiers synthesizing clinical trial results (which have high internal validity) and real-world studies (which are more generalizable) can convincingly communicate value for a new drug or treatment. ■ 


  • "[N]ew evidence describing the actual use and effect of the product in a real-world setting should be developed to inform formulary management across the product lifecycle.”

    –The AMCP Format for Formulary Submissions

  • Anita J. Chawla, Managing Principal
    Dave Nellesen, Principal
    Marcia Reinhart, Manager