Since 2020, the G-BA can request real-world evidence data collection in Germany. However, so far this was only implemented for one product, Zolgensma. Now, on 3 Nov 2022, a second product, Fedratinib (Inrebic), was added to the list of products with RWE collection.
Background
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. Up to now, 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:
- The plenum within the G-BA determines whether real-world data collection is required.
- 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.
- The G-BA shares the draft with external experts, such as the BfArM, the PEI, medical societies, the AkdÄ, and the manufacturer, for commenting.
- 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.
- 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.
- The manufacturer evaluates the collected data and has to share the analyses with the G-BA.
- 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.
RWE collection for fedratinib
Fedratinib had been evaluated by the G-BA on 2 Sep 2021 under the abbreviated orphan drug benefit assessment. The G-BA saw a hint of a not-quantifiable additional benefit, since the available scientific data did not allow a quantification.
Subsequently, on 3 Nov 2022, the G-BA decided on the RWE collection for the oral JAK inhibitor fedratinib (Inrebic). The manufacturer must collect real-world use data through a national registry study using the GSG-MPN registry (“German Study Group – Myeloproliferative Neoplasien”). The data collection is specific to the use in adults with primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia-myelofibrosis, who have not been previously treated with a JAK inhibitor and for whom ruxolitinib represents the appropriate comparator.
If the existing registry can’t fulfill the data requirements sufficiently, then the manufacturer is obliged to set up an alternative data platform, which can be used for the complete collection and later evaluation of the treatment data.
As the next step in the RWE procedure, the manufacturer now has to align with the G-BA on the study protocol and the statistical analysis plan. The registry study can only start after this.
Restriction of use
From the start of the study onwards, fedratinib will only be allowed to be used by physicians who are involved in the data collection.
The exact start time will be determined after the study protocol and statistical analysis plan have been agreed, which is expected in the second half of 2023.
Further reading related to RWE
- IQWiG flags up legal barriers to registry-based RCT
- IQWiG finds problems with the use of RWE in manufacturers’ dossiers
- IQWiG methods for RWE collection for products of same active ingredient class
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