ISSUE:

With small trial sizes, lack of active comparators and limited knowledge of the prognostic value of biomarkers complicating the generation of evidence at the assessment stage, precision oncology therapeutics are experiencing difficulties in meeting the evidence requirements of reimbursement agencies.

As HTA agencies and payers prepare to review the expanding pipeline of precision oncology therapeutics, how can we introduce new approaches and greater flexibility into the assessment of these products across their lifecycle to help address uncertainties and share risk across stakeholders?

OVERVIEW:

Scientific advancements over the past two decades have ushered in a new paradigm of precision medicine, which is changing drug development and care delivery. Precision oncology, in particular, is redefining how we view cancer, from an organ-specific to a molecular disease, with therapeutics targeting increasingly smaller, better-defined populations based on their molecular profile. With cancer treatment moving toward stratification based on the molecular makeup of a tumour, randomized control trials have shifted to smaller, single arm and basket trials involving a subset of individuals (sometimes using surrogate endpoints or no comparators).

Given the evidence limitations of these trials, including the lack of similar therapies with which to make comparisons, HTA agencies are faced with significant challenges in assessing these products, ultimately hindering their adoption and the ability of patients to benefit from these innovations.

The session will aim to:

• Present an overview of some of the complexities associated with generating and assessing evidence for precision oncology therapeutics [5 minutes];
• Discuss and debate potential approaches to addressing these challenges while balancing the potentially conflicting priorities of HTA agencies and payers (represented by Dr. Brian O’Rourke), patients (represented by Anne-Pierre Pickaert) and clinicians (represented by Dr. Jean-Yves Blay) [40 minutes]; and
• Use interactive online tools (e.g. polls) to gather questions and feedback from the audience [15 minutes].