Orphan drugs inherently face a number of challenges in their development and value assessment. In this blog post, we describe the Mechanism of Coordinated Access (MoCA) initiative, and discuss why it can be beneficial to all parties involved and how it can improve decision making for – and access to – orphan drugs in European countries.
There is considerable variation in the degree and speed of adoption of orphan drugs across European countries. Could a joint European process expedite value assessment and funding decisions for these interventions in individual countries?
MoCA is a voluntary, non-binding, free-of-charge collaboration between stakeholders, created under the European Commission Process on Corporate Responsibility in the Field of Pharmaceuticals. The Medicines Evaluation Committee of the European Social Insurance Platform (MEDEV) initiated MoCA pilots in 2013. The MoCA process and discussions are non-binding, and no recommendations are published. Key benefits of the MoCA process include increased predictability of outcomes of reimbursement processes, effective evidence generation plans (both pre- and post-launch), and sharing of expertise among countries.
So far, 15 companies have participated in pilot projects (see, for example, Hughes-Wilson, 2014) involving a series of multi-country level discussions between national payers, patients (recruited by EURORDIS – Rare Diseases Europe), applicant companies, and experts from the pharmaceutical industry around evidence development and reimbursement schemes. MoCA pilots are bespoke to the needs of the applicant company: there are no set procedures and timelines vary from pilot to pilot.
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 TVF includes four criteria: unmet need, relative effectiveness, response rate and degree of certainty. For each of the four criteria, a product can be scored using a categorical scale with three levels (‘low’, ‘medium’, and ‘high’).
In principle, having a pan-European assessment of new orphan drugs before the start of national pricing and reimbursement processes can be optimal for two reasons. First, the evidence base of orphan drugs near their launch can be limited (in some cases based on phase II trials), therefore it might be more efficient to review it centrally rather than at the individual country level. Second, some countries, particularly the smallest, might not have the required capacity and expertise to conduct an in-depth assessment of orphan drugs, especially those targeting very rare conditions which often affect a few patients in one country. Given the different ability and willingness to pay for health interventions of individual countries, a joint assessment should focus on the clinical benefit, leaving considerations about value for money, affordability, and social values to national processes. This is in line with the recent European Commission proposal on ‘joint clinical assessment’.
However, in the case of orphan drugs, because of the limited pool of patients affected and clinical experts across Europe, there is a case to develop a value framework that could capture a wide range of elements that are relevant to these medicines and the conditions they target. This could structure an early dialogue between key stakeholders at the central level. For any multi-dimensional value framework, a series of steps are required, including agreement on the value dimensions, their definition and their relative importance, and how to combine them.
The TVF is an initial guide to discussing value aspects of a new orphan drug and is not meant to support a quantitative value assessment. However, it has the potential to be further developed to create a valuable tool for national decision making. More work could be conducted to this end, focussing on areas such as:
- the selection and definition of criteria to allow their separate measurement and ensure they comply with recommended properties such as non-overlap;
- a method to score the medicine under discussion according to the criteria, potentially more granular than the three-category proposal;
- a method to weight the various criteria from different perspectives, acknowledging the relative importance of the dimensions of value, while allowing individual countries to use the results to inform further discussions around trade-offs in funding decisions.
Achieving consensus among the members of a multi-country consortium may prove difficult, though recommendations on value frameworks for orphan drugs are available for implementation (see Annemans et al, 2017, for example). For applicant companies, it is advisable to initiate the process at an early stage of the product lifecycle, when designing Phase 3 (or even earlier, for products with adaptive development designs), to inform evidence generation for payers’ submissions and post-launch real-world use. Payers and patients need to weigh the benefits of participating in MoCA at an early stage of product development against the risk of committing resources to discuss a product with considerable uncertainty, including whether the product will reach the market at all.
Towse, A. and Garau, M. (2018). Appraising Ultra-Orphan Drugs: Is Cost-Per-QALY Appropriate? A Review of the Evidence. OHE Consulting Report. RePEc
Zamora, B., Maignen, F., O’Neill, P., Mestre-Ferrandiz, J. and Garau, M. (2017). Comparing Access to Orphan Medicinal Products (OMPs) in the United Kingdom and Other European Countries. 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