The journey from scientific discovery to patient access for new therapies in the European Union (EU) is a complex and lengthy process. While the recent announcements of the positive opinion from the Committee for Medicinal Products for Human Use (CHMP) for VYJUVEK® brings hope, it's essential to understand that several stages remain before treatments become widely available.
The EU employs a centralised procedure for the approval of certain medicines, especially those derived from biotechnology, intended for advanced therapies, or addressing rare diseases. This process ensures that once a medicine is authorised, it can be marketed across all EU member states.
CHMP Opinion: The European Medicines Agency's (EMA) CHMP evaluates the medicine's safety, efficacy, and quality. A positive opinion from the CHMP is a significant milestone, indicating that the committee recommends the medicine for approval.
European Commission Decision: Following the CHMP's positive opinion, the European Commission reviews the recommendation. If they agree, they grant a marketing authorization, allowing the medicine to be sold throughout the EU.
National-Level Decisions: Despite EU-wide marketing authorization, individual countries make decisions regarding pricing and reimbursement. These decisions are crucial as they determine whether the medicine will be accessible to patients through national healthcare systems. This stage can vary significantly between countries and often involves negotiations that can extend over months or even years.
On February 28, 2025, Krystal Biotech announced that the CHMP adopted a positive opinion recommending the approval of VYJUVEK® for the treatment of dystrophic epidermolysis bullosa (DEB). The final decision from the European Commission is anticipated in the second quarter of 2025.
While this news is promising, it's important to recognise that:
Pending European Commission Approval: The CHMP's positive opinion is advisory. The European Commission has the authority to grant marketing authorization, a decision expected in the coming months.
National Adoption Timelines: After EU-wide authorization, each member state will assess VYJUVEK® for inclusion in their healthcare systems. This involves evaluating the therapy's cost-effectiveness and negotiating pricing, processes that can vary in duration across countries.
While regulatory milestones like a positive CHMP opinion are encouraging, they represent early steps in a broader process. Even after EU-wide approval, the availability of a new therapy can differ between countries based on national decisions. The path from a positive CHMP opinion to widespread patient access in the EU involves multiple stages, each with its own timelines and challenges. The period between EU approval and patient access can range from several months to years, depending on the efficiency of national procedures and negotiations.
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