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Analysing Global Immunisation Expenditure

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OHE and Vifor Pharma present a poster at the last European Conference on Rare Diseases (ECRD) on the Mechanism of Coordinated Access to orphan medicinal products (MoCA) where evidence on avacopan for ANCA-associated vasculitis (AAV) was discussed using the Transparent…
OHE and Vifor Pharma present a poster at the last European Conference on Rare Diseases (ECRD) on the Mechanism of Coordinated Access to orphan medicinal products (MoCA) where evidence on avacopan for ANCA-associated vasculitis (AAV) was discussed using the Transparent Value Framework.
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 (org.s3.amazonaws.com)
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:
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:
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
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