The new draft guidance from European Medicines Agency (EMA) aims to optimise the use of registry-based studies as a source of real-world evidence. The guideline’s primary focus is on the use of patient registries for studies by marketing authorisation applicants and holders (MAAs/MAHs). The guideline’s scope includes disease and condition registries, such as those for patients with a particular disease or disease characteristic, a condition such as pregnancy, or molecular or genomic features.
In the post-authorisation phase, patient registry-based studies can be useful for both safety and efficacy studies. Registry-based post-authorisation studies can help with assessment of effectiveness of adapted drug dosing schemes. They can also aid understanding of the effectiveness and safety of medicinal products in a broader clinical disease-related context and a more heterogeneous patient population.
Registries are important for patients with rare diseases who may receive advanced therapy medicinal products (ATMPs) such as gene therapy, since approval of these products is often based on small patient populations. According to the guidance, post-authorisation activity planning should begin in the pre-authorisation phase for ATMPs.
For pre-authorisation studies, observational evidence from patient registries can be helpful since the registries can supply information about population characteristics which can help to identify valid surrogate endpoints.
Registry-based study:
- Investigation of a research question or hypothesis using data from an existing patient registry or from a registry newly set-up for the study.
- Timelines of this study are driven by the collection/extraction and analysis of the data relevant for the specific study objective(s).
- Patient enrolment in these studies are defined by research objective(s) – may be a subset of a registry population; in case of a clinical trial, allocation to treatment (e.g. with randomisation) is to be documented; representativeness and generalisability of the study results to be analysed and documented.
- Data collection is restricted to what is needed by the research question including data on potential confounders and effect modifiers; additional data collection may also be required; if such additional data includes subject monitoring outside SmPC and normal clinical practice, the legislation for clinical trials apply; study may involve primary data collection or secondary use of data.
- Additional quality assurance to be performed for the study data; quality control to be prospectively defined and assessed with a risk-based approach; for RRCTs, data quality control involves central adjudication of events and treatment complications.
Also included is guidance on study protocol, population, and data management. The guideline also includes an annex detailing general considerations on constructing and using patient registries.
Draft guidance document from EMA: https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-registry-based-studies_en.pdf
Leave a Reply