Current Controversies in Rare Diseases and Evidence-Based Advocacy


Moderator: Jamie O'Hara, MSc, HCD Economics, The Innovation Centre, Chesire, UK
Speakers: Sheela Upadhyaya, MSc, NICE - National Institute for Health and Care Excellence, London, LON, UK; Brian O'Mahony, FIBMS, FACSLM, FIOSH, DMLM, Dip SHWW, Irish Haemophilia Society, Dublin, D, Ireland; Mohit Jain, PhD, MBA, BioMarin Europe Ltd, London, UK; Persefoni Kritikou, PhD, HCD Economics, Daresbury, UK

Presentation Documents

The purpose of this forum is two-fold: firstly, describe the process used to identify current controversies in rare diseases conducted to inform the future strategy of the ISPOR Rare Disease Special Interest Group (SIG); secondly, to present the outline of the Key Project scheduled by the Rare Disease SIG, on the development of guidelines for ‘Evidence-Based Advocacy’ (EBA). The Rare Disease SIG will present the results of a consultation process informing the future strategy of the SIG and priority research. Various stakeholders in the area will be consulted, through semi-structured interviews, with four main areas of interest: Regulatory, Health Economic Evaluation & Equity, Data Collection, and EBA. The interviews will elicit the views of the stakeholders on challenges in Rare Diseases and key areas of research. The results will be presented at the forum with an aim to have a dialogue between the panelists and the audience, to identify the medium-term research direction and strategic planning of the SIG, as well as Member Engagement and Key Projects for the SIG. The Rare Disease SIG has already scheduled a Key Project, on EBA. There is no consensus or consistency in terms of how Patient Advocacy Groups (PAGs) can best illustrate unmet need/burden of their respective beneficiary populations. Given the increasingly important role of PAGs (particularly in the Rare Disease space) the purpose of this project is to generate a set of guidelines to empower PAGs to better represent their beneficiaries. The focus will be on evidence generation to limit the welfare losses associated with incomplete information, delivery of healthcare in a more equitable way and use of evidence-based research to advocate for health policy shaping. A multi-disciplinary Working Group of experts will develop a set of guidelines to be published and made freely available to PAGs.

Conference/Value in Health Info

2021-11, ISPOR Europe 2021, Copenhagen, Denmark



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