Mission
To disseminate information and provide leadership related to health economics and outcomes research of precision medicine and advanced therapies.
Goal
- Establish a forum for members to engage in discussion related to the challenges of translating precision medicine and advanced therapies into clinical practice.
- Promote education related to the health economics of precision medicine and advanced therapies.
- Investigate the demonstration of value and the use of innovative payment models for precision medicine, cell therapies, gene therapies, and tissue-engineered products
ISPOR - Precision Medicine and Advanced Therapies
The term "precision medicine" is often described as providing "the right patient with the right drug at the right dose at the right time.” More broadly, precision medicine (also known as personalized medicine) may be thought of as the tailoring of a medical treatment to the individual’s characteristics, needs, and preferences during all stages of care, including prevention, diagnosis, treatment, and follow-up. Advance therapies (also known as regenerative medicine), which include cell therapies, gene therapies, and tissue-engineered products are used in the precision medicine approach to care. These advanced therapies are often one-time treatments that are potentially curative. Together, precision medicine and advanced therapies reflect some of the most cutting-edge medical technologies available, but these therapies have unique considerations and are very different than traditional medications. The Precision Medicine and Advanced Therapies Special Interest Group seeks to explore issues related to the approval, payment, utilization, and evidence development related to the use of these technologies so they can be better used to improve the population’s health.
Manuscripts and Reports
- Zimmermann, B, Spinner, D. S, Snyder, S.R, Purser, M. Can We Demonstrate the Value of Next-Generation Sequencing Approaches Within Traditional Value Frameworks? Value and Outcomes Spotlight. 2021; 7(5)
- Faulkner, E, Holtorf, A-P, Walton, S, et al. Being Precise About Precision Medicine: What Should Value Frameworks Incorporate to Address Precision Medicine? A Report of the Personalized Precision Medicine Special Interest Group. Value in Health. 2020; 23(5):529 – 539
- Faulkner E, Annemans L, Garrison L, et al. Challenges in the Development and Reimbursement of Personalized Medicine—Payer and Manufacturer Perspectives and Implications for Health Economics and Outcomes Research: A Report of the ISPOR Personalized Medicine Special Interest Group. Value in Health. 2012;15(8):1162-1171
Presentations
- Virtual ISPOR Europe 2020: Can We Demonstrate The Value Of Next-Generation Diagnostic Testing Approaches Within Traditional Value Frameworks?
- ISPOR Europe 2019: ISPOR personalized precision medicine special interest group- translating genomic technologies into clinical practice – are we falling short? – What are the challenges? Over-coming the challenges? – Are we inadvertently creating disparities
- ISPOR 2019: ISPOR personalized precision medicine special interest group- leveraging real world evidence to address uncertainty for transformative and curative therapies
Journal Club
- "Intrapatient Comparisons Of Efficacy In A Single-Arm Trial Of Entrectinib In Tumour-Agnostic Indications" July 2021
- “A review of methodological considerations for economic evaluations of gene therapies and their application in literature” April 2021
- “Shedding Light on Reimbursement Policies of Companion Diagnostics in European Countries” May 2020
- "Long-term Survival and Cost-effectiveness Associated with Axicabtagene Ciloleucel vs Chemotherapy for Treatment of B-Cell Lymphoma" January 2020
Leadership:
Chair-Elect
Renske ten Ham, PharmD
Chair
Melanie Whittington, MS, PhD
Past Chair
Molly Purser, MBA, BS, PhD
Working Groups:
Member Engagement:
These activities allow for a variety of members to participate and also facilitates disseminating content.
Co-Chairs:
Clarissa Higuchi Zerbini
Key Project
Health Technology Assessment of Next Generation Diagnostic Testing Approaches
Joint project with the Medical Devices and Diagnostics Special Interest Group
Co-Chair:
Susan Snyder, MBA, BS, PhD
Daryl Spinner, MBA, BS, PhD
Objectives and Rationale of the Key Project
In the past 2-3 years there has been an explosion in the availability and utilization of next generation testing (NGT) that includes multiple genes/genomic biomarkers. A recent report indicates that between March 2017 and 2018 more than 800 new multigene panels entered a market with more than 8,500 already existing panels in the US alone. While the compiled evidence for most NGTs is sparse to non-existent, the total amount of data being generated through routine use, and thus potentially available for evaluating their real-world clinical utility in HTA decision-making is substantial. Despite these recommendations and the increasingly prevalent use of such tests, few HTAs have been conducted and even fewer are reimbursed by payers. The challenges of defining and assessing the value of NGS testing approaches including data gaps, clinical practice issues, reimbursement/ funding considerations, and potential solutions will be addressed.