All ICER reports and public meetings include discussion of explicit factors in an overall judgment of value labeled “potential other benefits or disadvantages” and “contextual considerations.” It is important to address these factors because all too often what matters most to patients is poorly captured in the available clinical trial data. Sometimes this occurs because surrogate outcome measures do not reflect true patient-centered outcomes; but even when trials do capture the clinical outcomes that matter most to patients, there are other aspects of value that fall outside traditional definitions of “health” and that are important for patients, their families and caregivers, and society.
The information on these factors included in ICER reports is largely based on discussions with patients and clinical experts, and is vitally important as a part of any decision about value and fair pricing. Therefore, one of the goals of ICER reports and public meetings is to highlight these factors and make them more tangible for decision-makers, since this is an area that many payers and other policymakers will not have the time or ability to consider fully on their own. To help bring greater focus to these considerations, our independent appraisal councils cast votes on a 1-5 scale to indicate their judgment of the relative importance of each factor to an overall judgment of long-term value for money for that treatment. Further details about the factors that are voted upon are provided below, and broader information about the entire process of how ICER and the appraisal committees evaluate new drugs can be found in ICER’s Value Assessment Framework.
Contextual considerations refer to social or ethical priorities that shape to some extent how the value of any effective treatments for a particular condition will be judged. Independent appraisal committee members vote on a scale of 1-5 on the relative priority that any effective treatment should be given in respect to two considerations:
- Short-term risk of death for patients without treatment. Decision-makers may wish to give added priority to treatments for conditions that present a high short-term risk of death. This category captures what some ethicists have called a “rule of rescue” and the sense that even relatively small absolute gains in lifetime may be of higher priority when patients otherwise have very little time left before they are likely to die. The quantitative measure of “proportional” QALY (or evLYG) shortfall is sometimes used to inform this consideration.
- Magnitude of lifetime impact on individual patients. Decision-makers may also wish to give greater priority to treatments for conditions that represent a very large “burden” on patients throughout the life course. Some ethicists and others believe that greater priority should be accorded to treatments for conditions that affect patients over many years as opposed to conditions in which the negative effects still leave many years of full health during a lifetime.
Potential Other Benefits or Disadvantages
Potential other benefits or disadvantages are meant to capture the effects of a treatment beyond improvements in health. They represent the non-clinical benefits or disadvantages that a treatment may bring to patients, their loved ones, and/or society.
Independent appraisal committee members vote on a scale of 1-5. Votes of 1-2 represent relative disadvantages; 3 represents no effect; and votes of 4-5 represent relative benefits. The categories for votes are shown below:
- Patients’ ability to achieve major life goals related to education, work, or family life. This category is intended to capture the possibility that even if health benefits are fully captured in the evidence, that their long-term or “spillover” effects may be under-represented. For example, a long-term treatment may bring potential other benefits such as being able to choose a different career track, go to college, travel, or start a family. In some uncommon cases, a new treatment may bring disadvantages, like severe side effects that make it difficult to work or participate in social activities.
- Caregivers’ quality of life and/or ability to achieve major life goals related to education, work, or family life. This category looks at similar benefits or disadvantages of treatments as the previous one, but from the perspective of caregivers for patients with the condition.
- Patients’ ability to manage and sustain treatment given the complexity of regimen. This category considers whether a new treatment will be more or less burdensome for patients to take. Reduced complexity may help patients stay on their treatments as indicated.
- Society’s goal of reducing health inequities. This category looks at whether a new treatment can help address inequities in the health care system. For example, a new and effective treatment for sickle cell disease would specifically benefit patients in the Black community, which has historically been underserved and been the subject of systemic racism throughout the medical system.
- Other. Some additional factors may be identified before the public meeting and added as a specific vote. If an important potential other benefit or disadvantage is brought up for consideration during the public meeting, there may be a vote taken, or the item may be added as a “consensus” factor to the list compiled for the Final Report.