Innovation for Small Populations: What Should we Pay for?

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 than7,000 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 thought-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.

Full details and booking available here.

2021 THEME

With its devastating and far-reaching consequences, the pandemic has made clear that health and wealth are inextricably interlinked. The rapid development and introduction of new interventions to fight COVID-19 has also demonstrated that the value of healthcare interventions go far beyond narrowly defined patient benefits.

What is the real value of a highly effective vaccine for COVID-19? What wouldn’t a society pay to rid itself of the virus?

Whilst the broader value of treatments and vaccines are acknowledged at the height of the pandemic, it is critical that in the post-pandemic recovery period, that health systems invest in interventions that create the greatest value.

The 2021 Masterclass Series will take attendees through the journey of exploring value from multiple perspectives, providing tools to identify consolidated and novel sources of value, and generate compelling evidence to demonstrate them.

Whilst value assessments are necessary to ensure that healthcare expenditure is optimal, there is also a need for a common understanding of what constitutes value, capturing what matters most to patients, society, and payers.

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

OHE’s 2021 Masterclass may be virtual but its excellence will be real. Join OHE experts and a faculty of international thought-leaders in this stimulating and insightful series on Demonstrating Value.


  • To gain a deep-dive on theory and real-world evidence on the current debate on the value of innovative interventions to patients, healthcare systems and society, and anticipate future developments
  • To discuss and understand opportunities and barriers for the incorporation of broader sources of value which matter the most to patients and society, in terms of payers and decision-makers acceptability and evidence requirements
  • To understand the optimal approaches and solutions to improve patients, health systems and industry outcomes in areas of high unmet need such as rare diseases


Thursday, 9 December 2021