Table 5 Summary of potential and realised benefits of MoCA as a multi-stakeholder approach in Europe

Payers input early enough to support a clinical development programme adapted for downstream decision-making

Insight into potential “downstream” problems impacting anticipated availability, opportunity for direct interaction with HTAs and payers

Enhanced awareness of the condition and the actual unmet medical need to support potential future comparative assessments

Better budget impact awareness and predictability; insights into potential treatment-eligible population(s), epidemiological data; opportunity for early understanding of treatment setting or pathways (e.g., hospital vs. outpatients setting, first or later line therapy…)

Quicker and broader availability of an individual OMP and increased equity across EU MS

Increased predictability and planning for launch timing, likely uptake and barriers

More efficient Scientific Advice (if advice is taken into account)

Better data for HTA (if advice is taken into account)

Unique platform for multi-stakeholder and multi-country engagement on an individual OMP; opportunity to discuss potential parameters for managed entry agreements

Better understanding of needs and expectations from decision-makers in determining access and availability questions

Clear understanding from all data-customers about data-requirements to support value demonstration, initial P&R decisions and maintenance

Input into consolidated post-authorisation data-collection in conjunction with other data-customers – avoiding fragmentation and supporting inclusion of EMA tools [19] including Guidance and link with EUnetHTA / HTA tools, e.g., REQqueST [20]

Dialogue that includes the payer / budget-holder perspective on a given OMP, especially for countries where the HTA / payer agencies are split

Sharing of expertise with different MS / transparent understanding of clinical value, natural history, and related organisational aspects

Ability to inform and engage with OMP developers to secure relevant endpoints, expectations and needs are included

Guidance on prospectively designed post-marketing evidence generation / data-gathering / requirements based on gap identification from all data customers from earliest phases of development / market launch planning

Secure HTAs and Payers are aware of the regulatory rationale for decisions on endpoints and their relevance to the therapeutic area in question

Better, coordinated follow up and collection of PROs and real-life experience