Mission

By identifying key challenges in assessing the value of innovative therapies, including advanced, emerging, and orphan* therapies, we aim to advance research and policy dialogue to support informed healthcare decision-making and foster equitable access for all patients.

*Includes treatments for rare diseases with and without regulatory orphan drug designation.

Goal

  1. Leadership – Lead HEOR community in issues on value assessments of emerging and orphan therapies and contribute to the relevant active consultations.
  2. Research – Identify priority topics, conduct research, and provide a platform for membership to engage on key projects that contribute to advance decision-making approaches for emerging and orphan therapies.
  3. Education – Facilitate educational exchange with membership on the latest HEOR trends and policy topics pertaining to emerging and orphan therapies.

Manuscripts & Reports

  1. 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)
  2. Challenges in Research and Health Technology Assessment of Rare Disease Technologies Disease Terminology and Definitions—A Systematic Global Review

Webinars

  1. The Global Socioeconomic Impact of Rare Diseases: A Call for Action

Leadership


Chairs

Ka Keat Lim, MSc, RPh, PhD

Lecturer in Health Economics, Queen Mary University of London
London, LON, United Kingdom

Carina Righetti, MSc, BSc

Director Pipeline Strategy & Operations for Emerging Government Programs, J&J Innovative Medicine
Titusville, NJ, United States

Past Chair

Joel Iff, PharmD, PhD

Executive Director, Global Access, Value & Evidence, Sarepta Therapeutics
Cambridge, MA, United States

Renske ten Ham, PharmD

Assistant Professor, Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht
Amsterdam, NH, Netherlands

Working Groups



Key Project

In development


    Member Engagement

    These activities allow for a variety of members to participate and facilitates disseminating content in webinars, journal clubs, conference sessions and meetings, and online communities. 

    The Emerging and Orphan Therapies Online Community offers active ISPOR members a platform to engage in discussions, share resources, exchange experiences, and stay informed on SIG-related topics and updates. Join the ISPOR Emerging and Orphan Therapies Special Interest Group and connect with the online community today! Not yet an ISPOR member? Join today!

    EMERGING AND ORPHAN THERAPIES ONLINE COMMUNITY - FOR MEMBERS

    Questions or ideas? Please send an email to orphantherapiesSIG@ispor.org.

    Join this Special Interest Group

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