The Benefits of Early Engagement with Payers and Patient Representatives: The Case Study of a MoCA Pilot Project on ANCA-associated Vasculitis

Article by: Gayathri Kumar, Martina Garau

The Office of Health Economics in collaboration with Vifor Pharma is presenting a poster on a pilot with the Mechanism of Coordinated Access to orphan medicinal products (MoCA) for avacopan in ANCA-associated vasculitis (AAV) at the European Conference on Rare Diseases (ECRD).

MoCA is a voluntary, non-binding, free-of-charge collaboration among stakeholders, created under the European Commission Process on Corporate Responsibility in the Field of Pharmaceuticals. The process provides patients, payers and companies with the opportunity for early dialogue; to discuss unmet needs of the disease, and the value that a novel treatment could bring to patients and healthcare systems.

As part of MoCA, the Transparent Value Framework (TVF) was created to structure a discussion about the value of new orphan drugs, prior to national pricing and reimbursement submissions. The framework includes four criteria: unmet need, relative effectiveness, response rate and degree of certainty, using a categorical scale with three levels (‘low’, ‘medium’, and ‘high’) to score the product under discussion, as showed below.

Figure 1: TVF criteria. Source: WG MoCA-OMP Transparent Value Framework.pdf (

OHE supported Vifor Pharma in a pilot for their medicine avacopan in ANCA-associated vasculitis (AAV). This involved three meetings with payer representatives from 8 EU countries as well as EURORDIS and representatives from the patient group Vasculitis International. The meetings provided stakeholders with the opportunity to discuss:

  • the disease area and clinical trial program for avacopan,
  • an adaptation of the TVF disaggregating the relative effectiveness criterion into subcategories, and suggested outcomes to show the degree to which the criteria were met by avacopan, and 
  • suggested scoring using the pre-agreed TVF criteria and the outcomes of avacopan clinical trials.

OHE supported Vifor Pharma in adapting the TVF by disaggregating the relative effectiveness criterion into subcategories, identifying outcomes for each criterion, and suggest the scoring using the pre-agreed TVF criteria and outcomes of avacopan clinical trials.

Suggestions for how to further improve the MoCA process include:

  • further definition of TVF methods to refine the measurement and scoring of its criteria;
  • ensuring a broader and consistent representation of payers from the participating countries, e.g. through publicly promoting the benefits of the process;
  • keeping the process voluntary and non-binding while providing more funding to ensure it is run effectively;
  • aligning with other pan-European regulatory and Health Technology Assessment (HTA) initiatives, such as PRIME and the Joint Clinical Assessment (JCA);
  • continuing to build Patient Groups capabilities to facilitate technical discussions on market access topics.

In conclusion, the MoCA process provides a valuable opportunity for early exchange of information and perspectives on the value of an orphan medicine while it is at the development stage. Integrating the patient perspective allows for a better understanding of the condition and unmet need by all participants. The TVF is a simple and pragmatic tool to inform discussions around the value of a novel orphan medicine.


Related research

Towse, A. and Garau, M. (2018). Appraising Ultra-Orphan Drugs: Is Cost-Per-QALY Appropriate? A Review of the Evidence. OHE Consulting Report. RePEc

Garau, M. and Devlin, N. (2017). Using MCDA as a decision aid in Health Technology Appraisal for coverage decisions: opportunities, challenges and unresolved questions. In “Multi-Criteria Decision Analysis to Support Healthcare Decisions” Marsh, K., Goetghebeur, M., Thokala, P., Baltussen, R. (Eds.). Springer book. DOI

Posted in Pricing and Reimbursement, Other Public Policy | Tagged Events