Author: Lukas Schoppmeyer, Senior Consultant, Market Access

Since 2019, following the adoption of the German Act for greater safety in the provision of medicinal products (GSAV), Section 35a of the German Social Code Book V (SGB V) has enabled the implementation of routine practice data collection (Anwendungsbegleitende Datenerhebung, AbD). This instrument was introduced to address evidence gaps for certain medicinal products – particularly orphan drugs as well as medicines granted conditional approval or approval under exceptional circumstances – following market authorization. The objective of the legislation was to establish a mechanism that generates a more robust evidence base for these products, thereby enabling renewed benefit assessments and subsequent price negotiations. 

In practice, however, significant challenges have emerged that often render the implementation of an AbD impractical or even unfeasible. Since the introduction of the legislation, only four routine practice data collections have been initiated or are currently ongoing out of a total of 23 procedures requiring an AbD. 

The high number of required AbDs that have either not been implemented or were discontinued is primarily attributable to small patient populations within the approved indication and real‑world clinical practice, a lack of meaningful treatment alternatives, and the unsuitability of existing registries to address product‑specific research questions. In addition, the involvement of multiple stakeholders and the high administrative complexity result in disproportionally long procedural timelines, often extending over several years. The development of study protocols and statistical analysis plans, as well as the initiation, recruitment, follow‑up and long‑term observation phases of AbDs, take substantial time. To date, no AbD followed by a reassessment of the added benefit has been completed. The first reassessment is planned for Zolgensma® in July 2027—six and a half years after the initial benefit assessment. 

These prolonged timelines raise fundamental questions regarding the relevance of AbD results at the time of final analysis. On the one hand, randomized controlled trials for the respective medicinal products are often conducted in parallel – for example in earlier lines of therapy – leading to shifts in both the approved indication and the use of the product in clinical practice. On the other hand, standards of care within the indication may evolve, meaning that AbD results could ultimately rely on comparisons against outdated therapeutic approaches. 

Accordingly, since the beginning of this year alone, an AbD has already been discontinued or not initiated at all for three active substances in the treatment of relapsed or refractory diffuse large B‑cell lymphoma, as well as most recently for valoctocogene roxaparvovec in the treatment of severe hemophilia A. 

Against this background, there is a clear need for adjustment from both the perspective of the German Federal Joint Committee (G‑BA) and the pharmaceutical industry. This ranges from improved generation of care‑relevant, validated prospective data via suitable medical registry infrastructures – potentially initiated already during the marketing authorization process – to greater acceptance of statistical models and testing approaches for the comparison of data derived from single‑arm or placebo‑controlled studies, as increasingly advocated by industry. 

Nevertheless, under the current legal framework, pharmaceutical companies that decide not to conduct an AbD following a G‑BA request are required to enter renewed negotiations with the National Association of Statutory Health Insurance Funds (GKV‑SV) within a short timeframe. In these negotiations, an agreed reimbursement amount must be established that results in annual treatment costs reduced by an unspecified but “reasonable” margin compared with the previously agreed price. 

From a market access perspective, this creates a clear problem: if the G-BA requests an AbD, but this is not feasible in practice, pharmaceutical companies face significant economic risks. Against this backdrop, the German Kintiga team supports companies as a strategic partner in evaluating options, risks, and consequences when dealing with AbDs in market access. 

 

Glossary of Abbreviations

• AbD – Anwendungsbegleitende Datenerhebung
  (Routine practice data collection conducted alongside use of a medicinal product)
• AMNOG – Arzneimittelmarktneuordnungsgesetz
  (Pharmaceutical Market Restructuring Act)
• G‑BA – Gemeinsamer Bundesausschuss
  (German Federal Joint Committee)
• GKV‑SV – GKV‑Spitzenverband
  (National Association of Statutory Health Insurance Funds)
• GSAV – Gesetz für mehr Sicherheit in der Arzneimittelversorgung
  (German Act for Greater Safety in the Provision of Medicinal Products)
• RCT – Randomized Controlled Trial
• SGB V – Sozialgesetzbuch Fünftes Buch
  (German Social Code Book V – Statutory Health Insurance)
• SoC – Standard of Care

Sources

• Drucksache 19/8753 SGB
• Anwendungsbegleitende Datenerhebungen und Beschränkungen der Versorgungsbefugnis – Gemeinsamer Bundesausschuss 
• Valoctocogen Roxaparvovec zur Behandlung der schweren Hämophilie A: Anwendungsbegleitende Datenerhebung wird beendet – Gemeinsamer Bundesausschuss
• AbD-Impulsvorträge aus Pharmadialog AG3 Januar 2026