ICER's Value Assessment Framework: Capturing the Patient Experience

Assessing the value of new drugs, diagnostics, or other health system interventions, often called health technology assessment (HTA), can become an academic exercise that loses sight of the fact that these technologies ultimately have a single purpose: to help patients. The patient perspective should therefore be central to HTA. The Institute for Clinical and Economic Review (ICER), a leading HTA research group in the United States, has established a robust strategy for engaging patients so that the entire process is informed by the patient experience and what “value” means to them.

ICER’s reports evaluate the evidence on the comparative clinical effectiveness and cost-effectiveness of new therapies. An ICER report process has distinct phases, starting with announcing the topic for a report, collecting open input, and clarifying the scope of the report. As the report is developed, a draft version is made available for public comment, and the report process culminates with a public hearing at which an independent committee discusses the report, public comment is heard, and a roundtable of patients, clinical experts, and other stakeholders discusses how to apply the evidence to practice and medical policies. Input from patients and patient groups plays an important role in all phases.

The first phase of the process is called “scoping.” During this phase, ICER identifies relevant patient groups and solicits open input¹ from them and all other relevant stakeholders. ICER has developed a Patient Participation Guide² to help patient groups understand the overall HTA process and how they can contribute. Patient input during the scoping phase allows patients to help set the stage for the entire report by sharing information on what living with a certain medical condition is like for different patients, and by identifying the outcomes and other aspects of treatment that are most important to them. While much of the data considered in the report come from rigorous clinical trials, ICER recognizes that these trials may not capture important information about the patient experience with disease and treatments.³ Where feasible, ICER encourages patient groups to gather primary data on the patient experience that can be used to complement clinical trial data. Patient input can provide a guiding influence during scoping:

• In a review of therapies for non—small cell lung cancer (NSCLC),⁴ patient groups noted that levels of distress and anxiety are particularly high for patients with lung cancer. As a result, ICER looked for evidence that the therapies being assessed affected these outcomes. When ICER did not find such evidence, a discussion in the report of other potential benefits and disadvantages of the therapies noted this absence. The report also raised issues about generalizability from randomized trials, as patients in clinical trials may have greater resources, potentially leading to lower levels of distress than experienced by the general population of lung cancer patients.

The perspectives and evidence from patients and patient groups thus help inform ICER’s analysis of comparative clinical effectiveness and guide a description of “other benefits or disadvantages” included in every ICER report. Other benefits or disadvantages include important elements of overall value that may not be captured in the available clinical evidence or in a cost-effectiveness model. These elements include the potential for some treatments to ease family and caregiver burden, offer new modes of administration that can simplify treatment regimens, or allow patients to return to work or other activities earlier in treatment:

• The ICER report on treatments for multiple myeloma discussed input received from patients on the benefit of having oral treatment options available.⁵

These may also include contextual considerations, such as issues of equity:

• Based on patient input, ICER’s NSCLC report noted that lung cancer disproportionately affects patients in lower socio-economic groups who are least able to deal with the financial toxicities of treatment.⁴ The report also noted input from patient groups, pointing out that patients with NSCLC often have high rates of vascular and pulmonary comorbidities, which would have excluded many of them from clinical trials of newer therapies. Some newer therapies, however, are better tole-rated by patients with poor performance status than are older treatments, and so only comparing newer agents to existing treatments in randomized trials with restricted entry criteria misses a potential “other benefit”: the use of these agents in patients who would never have been candidates for treatment previously.

With the patient perspective firmly embedded in the analysis of comparative clinical effectiveness and other benefits or dis-advantages, an initial draft version of an ICER report is posted for a 4-week period to gather public comment. At this point, all stakeholders, including patients and patient groups, see how ICER has described the clinical and cost-effectiveness evidence and put them into context. This public comment period allows patient groups to express concerns if they feel that their perspectives have been inadequately or incorrectly addressed.

A revised draft report based on this further input becomes the subject of a public meeting of one of ICER’s independent appraisal committees. Each of these committees includes practicing clinicians, methodology experts, and public representatives with a background in patient advocacy. At the public meeting, there are numerous opportunities for additional patient input. First, with invited patient community representatives at the table, the information in the report on comparative clinical effectiveness and other benefits and disadvantages is debated by the appraisal committee and formally weighed as part of a vote on the long-term value for money of the treatment under consideration. Before votes are taken, there is another opportunity for public comment during which other patients and/or representatives of patient groups have the opportunity to speak. The cumulative impact of hearing from the invited patients and additional public commenters contending with issues of illness, disability, treatment, adverse effects, and insurance coverage is substantial in informing the way that the evidence is interpreted by the committee in its votes.

Following the voting, each meeting concludes with a moderated roundtable discussion that includes invited patients, expert clinicians, and representatives from the payer and manufacturer communities. The goal of this discussion is to frame recommendations for how best to apply the evidence to guide practice as well as medical policies, such as coverage and pricing determinations. Here, again, patient input is central:

• At the ICER meeting on multiple myeloma, patients pointed out that as they live longer and are on treatment longer, manufacturers are receiving more money over time for their treatment. This led to a recommendation that manufacturers consider this effect when setting prices.⁵
• The ICER report on treatments for moderate-to-severe plaque psoriasis found that all the targeted immunomodulators provided reasonable long-term value for money.⁶ As such, the report recommended that insurers consider abolishing step-therapy requirements—a major concern raised by patients.

Without input from patients, ICER’s reports would lack critical perspective on how a condition affects day-to-day life, the financial and insurance challenges patients face in accessing their treatment, information about what outcomes or adverse effects hold most importance to patients, and much more.² The traditional system through which manufacturers develop and price new drugs, and through which insurers evaluate evidence and make coverage decisions, has lacked transparency and offered few opportunities for patient input. We believe that ICER’s efforts to incorporate the patient perspective—through engagement in the HTA process, and through fostering a public dialogue—is an important step towards a more evidence-based healthcare system that can achieve high value while being grounded in clinical experience and firmly centered on advancing the best quality of care for all patients.

ABOUT THE AUTHORS

David M. Rind, MD, is chief medical officer, Institute for Clinical and Economic Review, Boston, MA.

Sarah K. Emond, MPP, is executive vice president and chief operating officer, Institute for Clinical and Economic Review, Boston, MA.

Address for Correspondence

David M. Rind, MD

Chief Medical Officer, Institute for Clinical and Economic Review

2 Liberty Square, Ninth Floor, Boston, MA 02109

E-mail: drind@icer-review.org

REFERENCES

1. Guide to open input. Institute for Clinical and Economic Review website. http://icer-review.org/wp-content/uploads/2016/02/ICER-Guide-to-Open-Input-012417.pdf. Accessed March 30, 2017.

2. Perfetto EM, Oehrlein E, Boutin M, Reid S, Gascho E. Value to whom? the patient voice in the value discussion. Value Health. 2017;20(2):286-291. doi: 10.1016/j.jval.2016.11.014.

3. Patient participation guide. Institute for Clinical and Economic Review website. https://icer-review.org/patient-participation-guide/. Published January 2017. Accessed March 30, 2017.

4. Treatment options for advanced non-small cell lung cancer: effectiveness, value and value-based price benchmarks. Final evidence report and meeting summary. Institute for Clinical and Economic Review website. https://icer-review.org/wp-content/uploads/2016/10/MWCEPAC_NSCLC_Final_Evidence_Report_Meeting_Summary_110116.pdf. Published November 1, 2016. Accessed March 30, 2017.

5. Treatment options for relapsed or refractory multiple myeloma: final evidence report and meeting summary. Institute for Clinical and Economic Review website. https://icer-review.org/wp-content/uploads/2016/06/MWCEPAC_MM_Final_Evidence_Report_061016V3.pdf. Published June 9, 2016. Accessed March 30, 2017.

6. Targeted immunomodulators for the treatment of moderate-to-severe plaque psoriasis: effectiveness and value. Final evidence report. Institute for Clinical and Economic Review website. https://icer-review.org/wp-content/uploads/2016/11/NE_CEPAC_Psoriasis_Evidence_Report_FINAL_012317.pdf. Published December 2, 2016. Accessed April 3, 2017.

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