Patient-reported outcomes must play critical role in oncology value frameworks

Issue: May 25, 2016

ByEthan Basch, MD

When I sit down with a patient to discuss starting a new cancer treatment, one of the first questions I am asked is how that treatment will make the patient feel. Will it improve their fatigue or pain? Will it cause side effects like nausea or neuropathy? Will it improve or worsen their physical functioning?

Oncologists and patients require this information in order to understand the “value” of a treatment — is it worth the tradeoffs given that patient’s disease status, personal preferences and insurance status.

Recently, interest has risen in quantifying the overall value of cancer treatments, by combining the benefits, harms and costs of treatments into single metrics that enable cross-therapy comparisons, formulary prioritization and assessments of whether pricing is reasonable. Example frameworks include the ASCO “Value Framework;” the European Society for Medical Oncology (ESMO) “Magnitude of Clinical Benefit Scale;” the Institute for Clinical and Economic Review (ICER) “Value Assessment Framework;” the Memorial Sloan Kettering “Drug Abacus;” and the National Comprehensive Cancer Network “Evidence Blocks.”

These frameworks largely depend on data from published clinical trials and available summary cost information. With the exception of the ICER approach — which bases efficacy estimates on quality-adjusted survival — none of the approaches explicitly requires consideration of “patient-reported outcomes,” such as symptoms or quality of life, to tabulate overall value. Notably, ASCO, ESMO and NCCN are explicit that their frameworks are intended for use by clinicians and patients to make informed decisions. But, it is unfathomable how this can occur meaningfully without prominent consideration of patient-reported outcomes.

This represents a major limitation of these frameworks and places their validity into question.

Another major limitation across frameworks is overreliance on severe (grade 3 to grade 4) toxicities for calculations of harms. Most patients also care about low-grade toxicities, especially when these become chronic. This information is usually not well collected in regulatory clinical trials, and necessitates consideration of post-marketing evaluations or comparative effectiveness/real-world data.

In summary, including the patient perspective in generation of value estimates is essential to assure that frameworks are relevant to the users of drugs. This can be achieved by:

Including real-world data in calculations (not just regulatory clinical trials), and by encouraging such evidence development when it is lacking;

Explicitly requiring inclusion of patient-reported outcomes, such as symptoms and quality of life in calculations, and penalizing scoring for drugs that do not provide such information (as has been done for some clinical practice guidelines);

Incorporating information on low-grade (and not just high-grade) toxicities; and

Including patient representatives in consensus processes for value frameworks to assign weights and overall value to specific drugs.

References:

Cherny NI, et al. Ann Oncol. 2015;doi: 10.1093/annonc/mdv249.

Institute for Clinical and Economic Review. Value Assessment Project. Available at: icer-review.org/methodology/icers-methods/icer-value-assessment-framework. Accessed April 27, 2016.

Memorial Sloan Kettering Cancer Center. Drug Abacus Tool. Available at: drugabacus.org/drug-abacus-tool. Accessed April 27, 2016.

National Comprehensive Cancer Network. Evidence Blocks. Available at: nccn.org/evidenceblocks. Accessed April 27, 2016.

Schnipper LE, et al. J Clin Oncol. 2015;doi:10.1200/JCO.2015.61.6706.

For more information:

Ethan Basch, MD, is a University of North Carolina Lineberger Comprehensive Cancer Center member, director of the cancer outcomes research program and professor in UNC–Chapel Hill’s department of medicine in the division of hematology and oncology. He can be reached at ebasch@email.unc.edu.

Disclosure: Basch reports no relevant financial disclosures.