Friedhelm Leverkus (Director Health Technology Assessment & Outcomes Research)

1) Which “application-accompanying data collections” have already been carried out, nationally and internationally?

2) What study/registry designs are used?

3) What levels of evidence can be generated?

4) How do registry data differ from those generated in RCTs?
They differ on 3 levels:

5) How has this data been incorporated into the benefit assessment so far?

Dr.-Ing. Janine Garbe, MHBA (Head Evidence-Based Medicine/HEOR)
1) Which “application-accompanying data collections” have already been carried out, nationally and internationally? 2) Which study/registry designs have been used?

3) What levels of evidence can be generated?
At the German level, the level of evidence accepted by the payers/HTA authorities is low. However, the importance of registry data in international approval is increasing: in the area of rare diseases, there are examples in which an approval or indication extension was based solely on registry data or that newborn screening was initiated in the USA

4) How do registry data differ from those generated in RCT?

5) How has this data been incorporated into the benefit assessment so far?
So far, the data has not been used in the benefit assessment

Dr. Andreas Kress (Head of Market Access, Health Policy, Key Account Management)

1) Which “application-accompanying data collections” have already been carried out, nationally and internationally?

2) Which study/registry designs have been used?
Prospective & retrospective observational studies; cohort studies; Mono group or parallel group designs

3) What levels of evidence can be generated?
For example, gaps in care are identified, data on the safety and effectiveness of new therapies are obtained and information is generated on the management of adverse events. EbM Class III to IIa

4) How do registry data differ from those generated in RCT?
In RCTs, the study population is very strictly regulated and limited by excluding comorbidities, for example. In return, the real patient population is recorded in the registers and the NIS, including all comorbidities and concomitant medications in any age structure (possibly restrictions for pediatric patients)

5) How has this data been incorporated into the benefit assessment so far?
Market access is increasingly being discussed with the GKV-SV (e.g. size of subpopulations), but currently not as part of the early benefit assessment

Dr. Susanne Schach, (Real World Data Director at Roche Pharma)

1) Which “application-accompanying data collections” have already been carried out, nationally and internationally?

2) Which study/registry designs have been used?

3) What levels of evidence can be generated?
Data from clinical routine per se have a risk of bias. Methodological procedures such as statistical adjustments can partially, but not completely, compensate for this

4) How do registry data differ from those generated in RCT?

5) How has this data been incorporated into the benefit assessment so far?
Data from registries, NISs and secondary data analyzes are already used today to describe the epidemiology and everyday care of the patients concerned. Real world data has also been used to demonstrate effectiveness, for example as an external control arm in single-arm studies - but not yet accepted by IQWiG/G-BA - methodological reasons were given