Proceedings of the
“XV. Expertengespräch” Online,
Organized by SanaClis

6th November 2020

SanaClis is pleased to share the summary report on an insightful and interactive educational event of the series of annual drug development expert workshops, held online on 6 November 2020.

Four expert speakers expanded on the current challenges in clinical drug development and market access. They discussed with an expert audience viable solutions for the way efficiently and successfully moving forward in clinical development operations. As in preceding years, Dr. med. Johanna Schenk, FFPM, GFMD, Executive Consultant and Company Officer at SanaClis DE GmbH, moderated the meeting.

SanaClis announces the rescheduling of the XV Interdisciplinary Drug Development Expert Workshop

I - FROM "REAL-WORLD DATA" TO "REAL-WORLD EVIDENCE" - BASIC METHODOLOGICAL REQUIREMENTS

Annette Lehn, Dipl.-Math., Institute of Biostatistics and Mathematical Modeling, Goethe University Frankfurt, Frankfurt/Main, Germany presented the propensity score method as a statistical tool for the analysis of data from non-randomized controlled clinical studies beside conventional multiple regression analysis.

Randomization remains the ‘gold standard’ for controlled interventional studies. However, there is opportunity for improvement, as Collins et al. 2020 suggest, such as in the reliability of study results, recruitment, quality, and effective follow-up. Data from randomized controlled interventional studies are collected under near ideal conditions and do not always apply to the real world. Thus, interest increases in the evidence that non-randomized controlled clinical studies can provide.

The propensity score method finds increased usage in the analysis of non-randomized studies and enables these to provide results of similar reliability as the ‘gold standard’.

Full presentation available (in German language) here.


II - Faster and better drug development with RWE data - the IMI EHDEN project


Dr. Johann Pröve, Chief Scientific Officer, Cyntegrity Germany GmbH, Frankfurt/Main, Germany introduced the IMI EHDEN (Innovative Medicines Initiative, European Health Data & Evidence Network), a cross-database flagship project that provides previously unavailable patient data for clinical research challenges.

The 22 partners from academia, small- and mid-sized companies, non-profit organizations, and EFPIA (European Federation of Pharmaceutical Industries and Associations) endeavor to harmonize anonymized data from over 100 million patient records provided by data partners in 12 countries. Through the intrinsic federate structure, the data remain with the respective data source.

A community has evolved around the project, involving academia, industry, regulators, payers, governments, and non-governmental organizations, from which all participants benefit. In addition, the EHDEN project offers education via the EHDEN Academy and publishes its outcomes in scientific papers.

Besides data issues, the IMI EHDEN project also addresses data quality & interoperability, analysis transparency & reproducibility, governance & legal issues, business challenges, ethics, questions about privacy, ethics and security, and demonstrating value in their work.

Full presentation available here.


III - International research meets national benefit assessment - two Incompatible worlds?

Dr. med. univ. Manuela Bamberger, Value & Access Strategy Consultant, spoke about the national early benefit assessment in Germany and its compatibility with international research conditions in clinical trials.

Since 2011, for the purpose of pricing, the scientific procedure of the early benefit assessment assures that costs of new medicines do not exceed those of established drugs without having demonstrated additional benefit on the basis of clinical trial data. Thereby, challenges at the international level are varying therapy standards and concomitant medication that do not reflect the therapy standard in Germany.

Differing requirements for medicine approval and the early benefit assessment can lead to study endpoints that are of no relevance for the early benefit assessment, such as progression-free survival, ECOG performance status, and the overall response rate in oncology trials. Patient-relevant endpoints for the early benefit assessment are standardized in four categories: extension of life span (mortality), reduction of symptoms (morbidity), improvement of the quality of life, reduction of side effects, which should be considered in study planning.

Full presentation available (in German language) here.


IV – Application-Accompanying Data Acquisition – ABDE – G-BA requirements – ASSESSMENT Methods used by IQWiG

Professor Sträter, Sträter Lawyers, Bonn, Germany described the new study type of application-accompanying data acquisition (ABDE) which was created during the Pharmaceutical Market Restructuring Act (AMNOG) procedure.

Orphan drugs and medicines that address unmet medical needs can be approved without a complete medical dossier. The most recent example is Zolgensma against spinal muscular atrophy (SMA) which was approved on the basis of a single-arm trial. One injection costs 2 million USD.

The goal of ABDE is to reduce the costs of such medicines. This shall be achieved with a better data situation, obtained with the new study type, supporting the early renegotiation of the reimbursement with the National Association of Statutory Health Insurance Funds (amendment of § 35 section 3b SGB V with § 58 VerfO G-BA, chapter 5; effective from 26 October 2020).

The Federal Joint Committee (G-BA) determines the type, duration, scope and evaluation of an ABDE under the approval of the EC and the national competent authority (NCA). The ABDE must be application-accompanying (observational study, case-control study, or registry study - EU-GVP product registries - after § 67 section 6 AMG) and excludes randomized blinded clinical trials.

Full presentation (in German language) available here.

Accompanying “Streiflichter” (in German language) available here.

Amendment of § 35 section 3b SGB V with § 58 VerfO G-BA, chapter 5 (in German language) available here.


This year’s event was held in honorable memory of Dr. Holger Maria Rohde, who sadly passed away suddenly and unexpectedly on 24th June 2020. The Expertengespräch attendees remember Dr. Rohde as a valued and active participant of past events.

SanaClis is looking forward to continuing with this series of annual meetings on 30 April 2021, if possible again face to face at the Frankfurt Innovation Center Biotechnology (FIZ).