Does HTA Undermine the Goals of EMA Authorization Pathways? Time to Availability of Drugs Licensed Under Conditional Marketing Authorization Compared to Standard Marketing Authorization

Speaker(s)

Ardito V1, Robinson J2, Ciani O3
1SDA Bocconi School of Management, Bocconi University, MILAN, MI, Italy, 2University of California, Berkeley, Berkeley, CA, USA, 3SDA Bocconi School of Management, Bocconi University, Milan, MI, Italy

Presentation Documents

OBJECTIVES: To expedite the drug approval process, the European Medicines Agency (EMA) has implemented programs, like the conditional marketing authorization (CMA), that grant access based on incomplete evidence at launch. However, while EMA focuses on the overall risk-benefit ratio of treatments, national payers in the European Union (EU) focus on their long-term benefits, cost-effectiveness and budget impact, thus reflecting a misalignment in incentives and missions. As part of the Horizon Europe project HI-PRIX (Grant Agreement: 101095593), this study investigated the time to availability of CMA drugs, compared to standard marketing authorization (SMA), in Italy, Germany, and Spain.

METHODS: Time to availability is defined as “inclusion of centrally approved medicines on the public reimbursement list in a country”. CMA-licensed drugs from 2006-2022 were retrieved. Another group of SMA, drugs comparable in terms of therapeutic indication and time of approval, was identified. For each retrieved drug, the following data was collected: drug information at launch time (orphan status, indication, PRIME status); information on the approval (pathway, authorization date, clinical evidence at approval); reimbursement dates in each national context (using Farmadati, Lauer-Taxe, and BIFIMED data for Italy, Germany and Spain, respectively).

RESULTS: A total of 65 CMA-SMA matched-drugs were analyzed. In Italy, median time to availability was 515 (IQR: 413-766) days for CMA drugs, against median 455 (IQR: 324-664) days for SMA drugs. In Spain, median time was 696 (IQR: 462-1,051) days for CMA drugs, against median 500 (IQR: 378-693) days for SMA drugs. In Germany, median time was 38 (IQRCMA: 26-146; IQRSMA: 27-94) days for both pathways.

CONCLUSIONS: The EMA approval process is being accelerated; payer access is not. Delays in payer coverage and access in major EU nations are counteracting the intent of specific authorization pathways by the EMA, like CMA. Will the Joint Clinical Assessment contribute to accelerating (and standardizing) time-to-access across member states?

Code

HPR99

Topic

Health Policy & Regulatory

Topic Subcategory

Approval & Labeling, Coverage with Evidence Development & Adaptive Pathways, Pricing Policy & Schemes, Reimbursement & Access Policy

Disease

No Additional Disease & Conditions/Specialized Treatment Areas