HEALTH ECONOMIC OR TECHNOLOGY ASSESSMENTS
As physicians, patients, advocates, health plans, drug manufacturers, and policymakers search for tangible solutions to address increasing healthcare costs and access issues in the United States, Institute for Clinical and Economic Research (ICER) drug reviews and other health economic assessments (HEAs) and value discussions have become commonplace today. For purposes of this website page and Lupus and Allied Diseases Association's Elevating the Patient Voice in Health Economic Assessments Initiative, HEAs represent Health Technology Assessments (HTAs) and Value Assessments (for example ICER Drug Reviews).
64% of patient advocacy organizations who participated in HEAs and had a positive experience indicated that their community did not benefit from the assessment by gaining access to the new treatment.
LADA's Elevating the Patient Voice in Health Economic Assessments Initiative
LADA actively engaged and participated in a HEA through ICER for Lupus Nephritis (LN) therapies over an 8-month period during 2020-2021. By drawing on our own unique experiences in using the patient and caregiver voice to drive the review to assess the drugs’ worth based on what people living with lupus and lupus nephritis and their caregivers value most in a treatment, we were able to effectively contribute to the assessment. The involvement of LADA and other lupus community participants and advocacy stakeholders ensured that the patient and caregiver viewpoints were properly accrued and accounted for in the lupus nephritis drug evaluation. In this case, the process yielded a positive assessment of both drugs being appraised, which has not been the usual outcome of these reviews. We would like to note that although the review was positive, it has not resulted in the lupus community gaining access to both treatments.
As a takeaway from this experience, LADA sought to better understand the collective patient advocacy experience in HEAs in order to demystify the process and encourage and prepare other patient advocacy organizations in different disease states to strategically engage and empower their own communities to effectively participate. LADA developed and conducted a survey between February and April 2022 to gauge the knowledge and experience of U.S. based patient advocacy organizations concerning HEAs and to inform the degree and structure of potential educational resources needed to better prepare groups to participate.
The survey was shared with a diverse group of patient advocacy stakeholders throughout the United States ranging from large national umbrella and chronic disease foundations to small grassroots rare disease advocacy organizations. The key findings from this survey illuminate the importance of including the patient voice in the HEA process while revealing that there is willingness among patient advocacy organizations to become engaged and identifying areas of opportunity to better prepare groups to be important participants in the HEA process.
63% of patient advocacy organizations that participated in HEAs responded that despite the outcome of the assessment, the overall participation experience was positive, demonstrating that they are open to being engaged and sharing their community perspectives.
Key findings:
73% of patient advocacy organizations included actual patient and caregiver stakeholders in their HEA processes.
46% of patient advocacy organizations who participated in HEAs indicated that their view of the final result of the assessment was neither positive nor negative for their community.
64% of patient advocacy organizations who participated in HEAs and had a positive experience indicated that their community did not benefit from the assessment by gaining access to the new treatment.
63% of patient advocacy organizations that participated in HEAs responded that despite the outcome of the assessment, the overall participation experience was positive, demonstrating that they are open to being engaged and sharing their community perspectives.
69% of patient advocacy groups that did not participate in HEAs indicated that they had not been asked to participate.
72% of patient advocacy groups that did not participate in HEAs, indicated that they would engage if they understood how to effectively participate through educational forums, had adequate resources, and were able to connect with organizations who had participated in HEAs.
The results of this groundbreaking Health Economic Assessments Participation Survey for U.S. based Patient Advocacy Organizations yields significant learnings for the broader advocacy and value assessment community while underlining potential opportunities for the LADA Team to further investigate and address. To review the report and summary CLICK HERE
72% of patient advocacy groups that did not participate in HEAs, indicated that they would engage if they understood how to effectively participate through educational forums, had adequate resources, and were able to connect with organizations who had participated in HEAs.
Definition
The definition of health technology assessment (HTA) is provided below, with important clarifying information provided in 4 accompanying notes:
HTA is a multidisciplinary process that uses explicit methods to determine the value of a health technology at different points in its lifecycle. The purpose is to inform decision making in order to promote an equitable, efficient, and high-quality health system.
Note 1: A health technology is an intervention developed to prevent, diagnose, or treat medical conditions; promote health; provide rehabilitation; or organize healthcare delivery. The intervention can be a test, device, medicine, vaccine, procedure, program, or system.
(Definition from the HTA Glossary: http://htaglossary.net/health-technology).
Note 2: The process is formal, systematic, and transparent, and it uses state-of-the-art methods to consider the best available evidence.
Note 3: The dimensions of value for a health technology may be assessed by examining the intended and unintended consequences of using a health technology compared with existing alternatives. These dimensions often include clinical effectiveness; safety, costs, and economic implications; ethical, social, cultural and legal issues; and organizational and environmental aspects, as well as wider implications for the patient, relatives, caregivers, and the population. The overall value may vary depending on the perspective taken, the stakeholders involved,
and the decision context.
Note 4: HTA can be applied at different points in the lifecycle of a health technology, i.e., pre-market, during market approval, post-market, through to the disinvestment of a health technology.
References
HTA Glossary. Definition of health technology assessment. http://htaglossary.net/health-technology-assessment. Accessed September 15, 2022.
O’Rourke B, Oortwijn W, Schuller T. The new definition of health technology assessment: a milestone in international collaboration. Int J Technol Assess Health Care. 2020:1–4. doi: 10.1017/S0266462320000215.
Resources
National Health Council Value Classroom
https://nationalhealthcouncil.org/education/value-classroom/
HTAi – The international association of Health Technology Assessment organizations
https://htai.org/interest-groups/pcig/
EUPATI – a European initiative to support patient involvement in clinical research, regulatory process and HTA
https://toolbox.eupati.eu/search-toolbox/category/health-technology-assessment/
ICER Lupus Nephritis Drug Review
https://icer.org/assessment/lupus-nephritis-2021/
Patient-centered health technology assessment: a perspective on engagement in health technology assessment by three patient organizations and a health technology assessment body. Published in International Journal of Technology Assessment in Health Care. Open access link.
National Library of Medicine self-study course on Health Economics
https://www.nlm.nih.gov/nichsr/edu/healthecon/01_he_08.html
CDC Office of the Association Director of Policy & Strategy
https://www.cdc.gov/publichealthgateway/pheconomics/index.html
Beyond guidelines – tools to support patient involvement in health technology assessment
https://g-i-n.net/wp-content/uploads/2021/06/HTA-final-for-online-publication-.pdf
EU Patient Involvement in Health Technology Assessment (HTA)
https://www.eu-patient.eu/policy/Policy/HTA/
Patient Voice Initiative
https://www.patientvoiceinitiative.org/patient-experience-and-participation/
CADTH Framework for Patient Engagement in Health Technology Assessment
https://www.cadth.ca/cadth-framework-patient-engagement-health-technology-assessment
ICER for Patients
https://icer.org/patients/
Please feel free to forward additional resource suggestions to Kathleen Arntsen at kathleen@ladainc.org