A company experience of the first MoCA pilot project

Background Pricing and reimbursement authorities often lack sufficient information to decide about Orphan Medicinal Products (OMPs), because of pricing, high levels of uncertainty and small datasets, amongst others. EU Member States share similar challenges as they seek to include OMPs appropriately in their healthcare systems. The “Process on Corporate Responsibility in the field of Pharmaceuticals”, launched in 2010 under the Belgium EU Presidency, included a project on a “Mechanism of Coordinated Access to Orphan Drugs” (MoCA), to explore whether a collaborative approach could create opportunities for more timely, sustainable and equitable access to OMPs. Twelve EU Member States, industry, patients’ representatives and other stakeholders developed a potential mechanism for such cooperation in the MoCA Recommendations. EU Member States adopted these as part of the formal conclusions of the Process on Corporate Responsibility on 17 April 2013. The Recommendations identified points where voluntary cooperation could smooth the process of evaluation, by sharing information and data along a coordinated, dialogue-based pathway; as well as a first draft “Transparent Value Framework” (TVF) to provide a possible structure for national pricing and reimbursement discussions.