Innovation for Small Populations: What Should we Pay for?

Developing products for small populations presents unique but well-known challenges. However, despite significant policy changes in Europe and the US there remains significant unmet need with only 5% of the almost 8,000 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 Masterclass of 2020 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.


Demonstrating Value: evolving with innovation explores this lively technical and policy debate where the evolving and sometimes challenging nature of innovative interventions is not always matched by value assessment paradigms applied by country decision makers.

Value assessments are necessary to ensure that healthcare expenditure is optimal but there is a need for a common understanding of what constitutes value, capturing what matters most to patients, society, and payers. The 2020 Masterclass Series will take attendees through the journey of exploring value from different perspectives and providing tools to identify consolidated and novel sources of value, and generate compelling evidence at different stages of the product life-cycle to demonstrate them.

Other Masterclasses in the Series

Exploring value: novel elements for breakthrough treatments

Patient preferences: what do patients value?

Adaptive Pathways: generating evidence of value


100% of previous attendees said that they would recommend our Masterclass events – here’s what they’ve said:

“Excellent exploration of a complex issue, with good balance of the theory and the practice.”

“Overall a well-rounded course. The course provided an in-depth understanding of the challenge ahead of curative medicines."

"Highlighted all of the challenges and the various viewpoints. A realistic view of current and emerging situation."

“Very comprehensive deep dive into the issues and reason for OHE recommendations.”

“A very helpful event with some very high level experts covering subjects which could be difficult and contentious. Light into informational darkness.”


Each event in our Masterclass Series provides a highly collaborative space guided by candid expert perspectives. During the event we encourage open discussion of the topic, and believe that through this we have the potential to unlock transformative insight and reveal solutions to the challenges we face every day.

As the world’s oldest and most well-respected health economic research institute, we’re uniquely placed to bring you the latest in research, expertise and real-world evidence, as well as first-hand advice from global thought leaders. Together we can improve decisions surrounding healthcare, and deliver better outcomes for patients, payers and industry.

Click here for registration

Thursday, 3 December 2020