Innovation for Small Populations: What Should We Pay for?

Virtual | 14:00 - 17:30 GMT | Thursday 9 December
15:00 - 18:30 CET | 09:00 - 12:30 EST | 08:00 - 11:30 CST

£375.00(per ticket)

Developing interventions for rare conditions presents unique but well-known challenges, such as small and heterogeneous populations, limited knowledge of the disease, and lack of validated endpoints to predict long term effects.

Despite significant policy changes in Europe and the US, there remains significant unmet need with only 5% of the more than 7,000 rare diseases having treatments available today.

Generating the right incentives to stimulate research and development into new therapeutic solutions for rare diseases, whilst also managing concerns around health system affordability, is a critical issue of global relevance.

The final OHE Masterclass of 2021 will tackle this issue and discuss solutions for moving forward in the area of orphan and ultra-orphan diseases.

Based upon the most recent therapies for rare conditions, this Masterclass will provide essential insights on how to articulate value propositions of orphan medicines, by exploring the elements of value that matter most to patients, their families and carers, but also areas where cost-offset (in the healthcare system and beyond) can arise.

Join OHE’s global though-leaders and other industry experts who will discuss the highly controversial issue of value and prices of orphan medicines, and how to set a fair reward to private R&D investments. The patient perspective on the challenges to gain access to orphan medicines and on collaborative solutions to address uncertainty and data gaps to accelerate access to promising therapies will also be explored.


Delegates Join

Welcome and Introduction

HTA for Orphan Medicinal Products (OMPs)

1. Are health conditions been salami-sliced to drive exceptionalism?
2. Can evidence in support of Health Technology Assessment (HTA) submissions for OMPs meet the same standards as medicines for more common conditions?
3. What is society willingness to prioritise treatments for rare conditions?

QALYs for OMPs

4. Does the quality-adjusted life year (QALY) metric capture all the elements of value relevant to rare conditions?

Case study: Project Hercules

A UK based initiative to improve the evidence base of Duchenne treatments


Research and Development (R&D) for OMPs

5. Is Market Exclusivity (ME) needed to incentivise the Research & Development (R&D) of Orphan Medical Products (OMPs)?
6. Are R&D costs for individual OMPs lower than those for medicines for more common conditions?
7. What is a fair price for OMPs?

Sustainability of OMPs

8. Is the total expenditure for OMPs sustainable for individual health systems?

Panel Discussion

Summary and Reflections

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