Workstream 1

Price and the economics of ATMPs: Enabling a stakeholder dialogue in support of patient access and innovation.

 

Project scope

Advanced therapeutic medicinal products (ATMPs) represent a paradigm shift in healthcare, offering potentially curative therapies for patients with rare and complex diseases. Yet pricing of these therapies is subject to increasing scepticism, with concerns about the fairness of the innovation model and sustainability of expenditure on ATMPs in rare diseases.

The ambition of this workstream was to create a dialogue on the topic of pricing and economics of ATMPs, so as to improve the understanding around the ATMP innovation model, as well as the implications for sustainable health systems and innovations. The key objectives were:

  • To help contextualise prices within the broader ATMP innovation model through collective exploration
  • To explore the economics of development of ATMPs in rare diseases
  • To enable an open dialogue across stakeholders and advance mutual understanding of the pricing issues of ATMPs

 

Stakeholder engagement

To achieve these goals, a discussion document was first developed within the RARE IMPACT Working Group, structured in five sections:

  • Overview of biopharmaceutical innovation model
  • Characteristics and value of ATMPs in rare diseases
  • Economics of ATMP innovation in rare diseases
  • Sustainability of ATMP innovation (manufacturer’s perspective)
  • Sustainability of ATMPs for society (other stakeholder’s perspective)

These themes were then covered in a series of three stakeholder dialogue workshops, attended by representatives from Working Group member companies, patient organisations, policy makers, payers and physicians. Within the workshops, content of the discussion document was utilised to guide discussions and acted as a ‘straw man’ for participants to respond to. Furthermore, areas of alignment, misalignment and information gaps around different topics were identified and captured, as well as potential actions to address them. Accordingly, key discussion points and insights from the dialogue workshops were summarised in a short report.

 

Discussion document (PDF) Summary of dialogue workshops (PDF)