Whitepaper: Orphan drugs

Ten years ago, the "Act on the Reform of the Market for Medicinal Products" (AMNOG) which is still the legislative framework governing the market access of innovative prescription drugs, caused a paradigm shift in the examination of the added value and subsequently the reimbursement of medicinal products in Germany. Based on the evidence of available study data, the additional benefit over the existing standard of care is assessed by the most powerful German health authority, the German Federal Joint Committee (G-BA). Depending on the classification of the benefit category, the pharmaceutical manufacturer negotiates the final reimbursed price with the German national association of statutory health insurance funds (GKV-SV).

For orphan drugs, there are specific challenges that the manufacturer faces due to the rarity of the disease. These include small population size and studies of lower evidence levels than the recommended Randomized Controlled Studies (RCT). As a result, there is a considerable uncertainty about the added value of the drug which can lead to intense and sometimes strenuous negotiations with high rebates. Pharmaceutical companies planning to introduce new orphan drugs into the German market should prepare a sound strategy well in advance to ensure a successful market entry. This white paper elaborates on all relevant issues pertaining to the market authorization of orphan drugs in Germany and describes important recommendations for manufacturers to address these challenges.

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In mehr als 30 Seiten erötert das Whitepaper die folgenden Punkte:

1. Before AMNOG: quick overview of the German market access process for orphan drugs
2. The AMNOG legislation changed the pricing landscape of pharmaceutical products in Germany
   • Overview of the AMNOG process
   • First step of the AMNOG process: Benefit assessment
   • Second step of the AMNOG process: Price negotiations
   • Third (optional) step of the AMNOG process: Arbitration
3. 10 years of AMNOG - Insights gained for orphan products
   • Results from the early benefit assessment of orphan drugs
   • Outcome of the benefit assessment for orphan drugs after exceeding the 50 million euros threshold
   • Outcome of the benefit assessment after expiration of the G-BA resolution
   • Registry requirements and implications on the benefit assessment of orphan drugs
   • Challenges related to price negotiation and the arbitration
   • Outcome of the benefit assessment of the most expensive pharmaceuticals
   • Challenges related to the payment of curative gene therapies)
4. Lessons learned from AMNOG and its strategic implications