Value Assessments: Incorporating Patient Benefits and Perspectives in ICER Reviews of Allergy and Asthma Treatments

New treatments for patients living with moderate to severe asthma and serious allergies have been on the radar of the Institute for Clinical and Economic Review (ICER), which has developed value assessment reports for those treatments. In recent years, ICER has reviewed a therapy for severe asthma with eosinophilia (2016), five biologic therapies for moderate to severe asthma (2018), and in June 2019 ICER completed its value assessment of three potential treatments for peanut allergy. A common thread among these reviews is participation by Tonya Winders, CEO and President, Allergy & Asthma Network (AAN), a national non-profit organization dedicated to ending the needless death and suffering due to asthma, allergies, and related conditions through outreach, education, advocacy, and research.

Asthma and allergies affect millions of Americans. The Centers for Disease Control and Prevention estimates that 20.4 million American adults and 6.1 million children have asthma; of those, about 5 percent -10 percent have severe asthma. Each year, asthma is associated with 14 million physician visits, 1.8 million emergency room visits, and 440,000 hospitalizations, accounting for $82 billion in treatment costs.

Peanut allergies affect an estimated 1.5 percent-4.5 percent of children. Managing food allergies primarily focuses on avoiding exposure to peanuts, though up to 39 percent of people with peanut allergy may be accidentally exposed and experience a reaction each year. Two promising treatments for peanut allergy are currently undergoing Food and Drug Administration review and a third, described as a “non-commercialized oral immunotherapy,” involves introducing increasing amounts of peanut allergen to desensitize the patient. The “non-commercialized oral immunotherapy” is in use; all three treatments, however, are the subjects of an ICER assessment.

In a conversation with the National Pharmaceutical Council (NPC), Ms. Winders discusses her involvement in the peanut allergy value assessment process and shares her experiences in working with her members and ICER.

NPC: Given your involvement in three different value assessments by ICER, how has the process changed?  How has your approach changed, if at all?

AAN: I had no idea who ICER was or how the process worked in my first ICER review years ago, so I was ill-prepared and quite overwhelmed when I was invited to participate on the live meeting panel less than one month in advance. During the second review in 2018, I engaged early and often with ICER, offering the patient perspective at every stage of the process. I was disappointed with the lack of integration of the feedback into the final report and voting process. In the third review in 2019, I was engaged at each stage actively, but my approach changed. I obtained an outside health economist to help draft our comments and offer clear specifics on how ICER needed to adapt their economic model to better capture patient outcomes. While ICER was more receptive to the economist’s comments and did slightly adjust the utility based on comments, it did not change the outcome and final vote, which was disappointing.

NPC:  Many patient advocacy groups grapple with whether to engage individual patients in the review process, and, if so, how and at what stage.  How does AAN engage members in value assessments?

AAN: Allergy & Asthma Network has consistently offered to be engaged at every step of ICER’s process. We have participated in pre-scoping discussions with ICER, provided formal written comments on the scoping and draft evidence reports, attended the live meetings and provided testimony. We also keep our members, who include patients, families, caregivers and health care providers, aware of what ICER is and how we are working on their behalf at each stage of the review process. We do not ask members to write ICER throughout the process because the matter seems to be far too technical for the vast majority of our members. We do engage a few select members to attend and support our comments with specific patient testimonials.

NPC: AAN and others have noted in comments to ICER that the impact of disease on daily tasks of living are most important to patients and caregivers. ICER and others rely on QALYs (quality-adjusted life years) as a measure of patient benefit to assess value.  How well do QALYs capture the benefit of a treatment from a patient’s perspective?

AAN: We do not believe ICER’s value framework nor QALYs adequately reflect the impact on activities of daily living, especially in common chronic conditions that do not have high rates of ER visits, hospitalizations or mortality. QALYs are typically used in epidemiology to make country-to-country and disease-to-disease comparisons, and reflect benefits and “value” more from a payer perspective than a patient or societal perspective.

NPC: In addition to the health economist, have you engaged with experts outside of the Allergy & Asthma Network to help guide your responses? If so, what types of expertise have you sought and why? Did you find it helpful? Why or why not?

AAN: We have always involved medical reviewers in the development of our comments. In the 2019 review, we also employed a health economist. We found it is essential to have this area of expertise due to the technical nature of the ICER review documents. [The economist] helped me to understand how a small difference in the utility can have a significant impact on the cost-effectiveness evaluation. Our medical reviewers are also frustrated by their discussions with ICER and reported that committee members often have no relevant medical expertise in the therapeutic area they are discussing.  Consequently, they cannot understand the consequences on a patient’s life, beyond the reported population health data. Most medical reviewers report their comments to ICER were completely disregarded and in no way influenced the scoping, draft or final guidance.

NPC: ICER’s value assessments tend to focus on new treatments, including some that have not finished the FDA review process.  You’ve raised concerns about relying on clinical trial data to make assessments of value.  What are the limitations with clinical trial data that concern you?  What would you like to see instead? How has ICER responded?

AAN: Clinical trial data are designed for one purpose – to ensure the product meets clinical endpoints to achieve FDA approval.  These data were never intended to be used in head-to-head cost-effectiveness evaluations. I have implored ICER to wait until manufacturers and payers have true real-world impact data and cost-effectiveness data before moving forward with these reviews; however, it has gone unheard.

NPC:  Given your experiences, why do you feel it is important to continue to engage with ICER?

AAN: I believe it is our responsibility to the community we serve to be engaged in this process. I often say we must be “at the table to avoid being on the table.” Innovation breeds hope for patients, but innovation without access simply breeds frustration. It is difficult to have a third-party organization like ICER make population-based value assessments based on limited data, with total disregard for an individual patient’s history, including burden of disease.

NPC: Any recommendations for other groups who have not engaged in value assessments, but are interested in finding impactful ways to get involved?

AAN: Engage early and often and reach out for help from qualified health economists, preferably those who are familiar with the disease/therapeutic area under consideration. Use every opportunity to bring the patient voice forward in the process. Do not let the economics and evidence presented discount the emotional impact of those living with the disease daily.

NPC: Thanks for your time and candor.  Sharing your experiences and the lessons you’ve learned will help others understand the process and how to engage.

AAN: Thank you for this opportunity to share our experiences. I hope this helps other groups to engage more effectively and to stay the course with ICER even when frustrated or overwhelmed.