Value assessment frameworks have emerged as tools to respond to demand expressed by patients, clinicians, payers and other stakeholders for greater health care decision support, particularly to define and measure the relative value of treatment options. In this NPC-funded study conducted by the Lewin Group, four existing value frameworks were compared head to head to understand how each framework would approach the same condition, multiple myeloma, and why the results may be similar or differ. Ultimately, each framework arrived at a different result because of variations in inputs and methodologies, shedding light on how these tools may be used when applied to a wider set of clinical conditions and providing next steps to derive more thorough understanding from existing frameworks.
Four frameworks were studied:
- The American Society of Clinical Oncology (ASCO) Conceptual Framework to Assess the Value of Cancer Treatment Options
- Memorial Sloan Kettering Cancer Center‘s (MSKCC) DrugAbacus
- The National Comprehensive Cancer Network (NCCN) Evidence Blocks™
- The Institute for Clinical and Economic Review’s (ICER) value assessment framework tool
The research looked at the patient population for each value framework, treatments analyzed, the evidence base, clinical outcomes of interest, measures of cost/affordability and the main output of the value assessments. Comparisons of these frameworks highlight important differences and analytic challenges, including the different methodologies, inputs, outputs and assumptions of each that yield important differences in results, even between frameworks with the same main intended purpose and audiences.
By examining these four value frameworks, researchers found:
- A major concern is the relative timing for assessments of new treatments. For some treatments, although NCCN and ICER looked more broadly at the evidence, only one clinical trial was available at the time each framework was applied to assess multiple myeloma treatments.
- Patients with multiple myeloma value some outcomes that may be lesser or non-priorities for some or all frameworks, including:
- health-related quality of life;
- ease of use;
- management of toxicities and side effects (including low-grade, chronic side effects); and
- financial toxicity (i.e., patient cost burden of therapies).
- Updates to multiple myeloma assessments are necessary, given that treatments and the evidence base for this disease are rapidly evolving.
Based on these findings, recommended next steps for framework-makers and health care stakeholders include:
- Considering the assessment timing for conditions such as multiple myeloma
- Reaching out to multiple myeloma patients and clinicians early and maintaining ongoing outreach to better understand:
- patient-centered and clinically relevant outcomes;
- comparators that are relevant to therapeutic options for patients and clinicians; and
- how patients, clinicians and others who are not the primary target audiences of an assessment may be affected, directly or indirectly, by how stakeholders will use the results of those assessments.
- Ensuring that frameworks are explicit and otherwise transparent about multiple methodological aspects, e.g.:
- how and why particular regimens were selected for assessment;
- the sources of evidence used (including who can submit evidence);
- protocols and criteria for inclusion/exclusion of evidence;
- how data are entered and used in scoring, equations, algorithms, models, etc., with examples included with the methodology; and
- integration of expert stakeholder and patient input.
- Making sure that frameworks are more explicit and otherwise transparent about additional aspects, e.g.:
- intended audience(s) and purpose(s);
- limitations of frameworks and output; and
- guidance on use of frameworks.
- Incorporating provisions for prompting assessment updates (periodically or with the availability of new evidence). The report also detailed how the various frameworks address this challenge.