What’s important to know about real-world evidence data collection in Germany?

In this post, I’ll summarise the key points on the real-world evidence data collection in Germany, that the G-BA can request since 2020.

In an early benefit assessment, the G-BA determines the so-called additional benefit of an intervention. This is based on data submitted by the manufacturer on the efficacy, safety, or health-related quality of life, demonstrated with the intervention.

However, some products receive regulatory approval due to a special status:

  • Drugs with conditional approval
  • Drugs authorized under exceptional circumstances
  • Orphan drugs

The aim of this special approval route is to make the treatments available to patients as quickly as possible. This often means that the data available at the time of approval and early benefit assessment is immature or insufficient to derive meaningful conclusions.

Since 2020, the G-BA can request for these cases that manufacturers collect data on the real-world use (German: “Anwendungsbegleitende Datenerhebung”). This means that better, more relevant, data will become available, while patients are treated with the product in clinical practice. The G-BA will then re-assess the drug after several years (§35a SGB V) using this collected real-world evidence (RWE), in addition to data from clinical trials.

The G-BA website includes a list of all RWE data collections requested by the G-BA. Currently, Zolgensma in SMA is the only product on that list.

Process of requested real-world data collection in Germany

The G-BA can request RWE data collection immediately upon launch of the drug in the German market, or at a later point. The process is as follows:

  1. The plenum within the G-BA determines whether real-world data collection is required.
  2. Then, in the next 6 months, the G-BA or IQWiG develop a draft concept, which outlines:
    • the type, duration, and extent of the data collection,
    • the key questions to be answered,
    • the methods of data collection,
    • the evaluation of the collected data.
  3. The G-BA shares the draft with external experts, such as the BfArM, the PEI, medical societies, the AkdÄ, and the manufacturer, for commenting.
  4. The plenum of the G-BA will now finalize the concept plan within another 6 months. The G-BA can also decide to restrict reimbursement of the drug in question to prescribing by clinicians who are participating in the data collection. This should ensure that the data collection covers every use of the drug.
  5. Finally, the manufacturer now has to conduct the study. They can either set up a new study or use existing registries, if these already capture the required data. In that case, participating clinicians should refer to the registry data.
  6. The manufacturer evaluates the collected data and has to share the analyses with the G-BA.
  7. Every 1.5 years, the G-BA assess
    • whether the data are being collected or not,
    • if the data is sufficient for a benefit re-assessment,
    • or if there is a need to amend the concept plan to increase the meaningfulness of the data.

No real-world evidence collection for tabelecleucel in Germany

Tabelecleucel is a somatic cell therapy, classed as an ATMP, for the treatment of Epstein-Barr virus associated post-transplant lymphoproliferative disorder. It is an orphan drug that is undergoing an accelerated EMA assessment since Nov 2021. As such, the product does qualify for G-BA-requested RWE collection.

However, on 5 May 2022, the G-BA has decided that it will not require real-world data collection on the use of tabelecleucel. Even though there will be a need for more data at the time of the benefit assessment, it would be very difficult to conduct these required studies nationally. The target patient population is extremely small and experts estimated there are between 6 and 20 patients in all of Germany. Moreover, the population is very heterogeneous, and there is no consistent use of treatment alternatives that could form a study control arm.

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Anja Pownell

One thought on “What’s important to know about real-world evidence data collection in Germany?”

  1. Pingback: IQWiG finds problems with the use of RWE in manufacturers' dossiers

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