XIV INTERDISCIPLINARY EXPERT WORKSHOP “SUCCESSFUL DRUG DEVELOPMENT UNDER COST CONSTRAINTS AND COMPLEX REGULATIONS”

Frankfurt am Main, Germany


Proceedings of the “XIV. Expertengespräch” in Frankfurt am Main, Organized by SanaClis

SanaClis gladly shares the summary report of an insightful and interactive educational event held at the Frankfurt Biotech Innovation Center on 17 May 2019.

Four expert speakers introduced to current challenges in clinical drug development and discussed with an expert audience viable solutions for the way efficiently and successfully moving forward in clinical development operations.

I - Innovative Pharma/Biotech R&D Financing

Daniel Parera, MD, Executive in Residence, TVM Capital Life Science, Munich, Germany presented an innovative risk mitigating and IRR (internal rate of return) maximizing investment strategy for early‑stage therapeutics utilizing a “Project Focused Company investment model (PFC)”.

-      For “Early Stage” investments in a PFC approach, one pre-clinical asset at a maximum of 1.5 years prior to IND is spun-off into a virtual NewCo with the focus on the fields of oncology, CNS/pain, auto-immune, metabolic, or certain cardiovascular diseases. For the PFC, TVM provides full investment to achieve clinical proof of concept with comparably lower risk, faster development time with capital efficiency. This is enabled through the collaboration with Chorus Pharma, an autonomous development unit of Eli Lilly, as an operational backbone.

-      TVM also does syndicated “Late Stage” investments, meaning registrational trials for therapeutics, pre-commercial for diagnostics and commercial stage for digital and Medical Devices.

-      All investments are focused on an exit within 4 years or less.

II - Clinical Trial Transparency: Status Quo and Important Trends

Dr. Matthias Zerm, Lead Expert Clinical Trial Disclosure and R&D Processes Global Clinical Development, Merz Pharmaceuticals GmbH, Frankfurt am Main, Germany reported on the past, present, and future of clinical trial transparency (CTT). The take-home messages of his contribution were as follows:

-      CTT complexity is still increasing, yet its global harmonization remains a utopia. It nowadays encompasses numerous types of clinical documents, lay language summaries, as well as sharing of patient level data.

-      Sponsors need dedicated experts and CTT aspects/procedures built into routine business processes and not consider CTT as an add-on. A shift in mindset is key to achieve efficiency and compliance. Top management should commit to CTT to enable a top-down implementation of necessary standards and procedures. Embracing the CTT reality is key for making use of potential benefits, such as retaining/gaining positive reputation, (competitive) intelligence, learnings from other studies, and potential collaborations.

Full presentation available here

III - CRO Oversight as Practiced by vfa Members

PD Dr. Michael Hennig, Head of Biostatistics & Epidemiology, GlaxoSmithKline, Munich, Germany presented survey results on outsourcing and CRO oversight in clinical trials collected from member companies of the vfa, the German Association of Research-Based Pharmaceutical Companies.

The guiding principles for sponsor oversight as concluded from the survey were transparent communication, clearly defined quality expectations, precise assignments of accountability and responsibility, and a comprehensive documentation of oversight evidence. For globally acting sponsors the alignment of oversight processes at the global and local level adds to this list.

The survey results demonstrate that the currently implemented oversight processes cover all relevant areas to ensure highest quality and integrity of data produced by outsourced clinical trials.

Full presentation available here

IV - Registries as Demanded by the G-BA according to the GSAV

In his first presentation, Professor Sträter, Sträter Rechtsanwälte, Bonn, Germany described new regulations for “application-accompanying data collection” in the German HTA process (so-called AMNOG procedure) and for the use of cannabis, prescribed by physicians in the statutory German health care system. The main topics of the talk were:

-      New Regulations: The legislator authorizes the G-BA (Federal Joint Committee) according to the GSAV (law on more safety of provided medicinal products) to order the implementation of "application-accompanying data collection" for medicinal products being authorized under exceptional circumstances with an incomplete dossier, such as orphan drugs. These data collections are named as registries. Physicians are obliged to participate in these studies in order to prescribe the drugs at the expense of statutory health insurance funds. For patients, however, participation in this data collection is voluntary. PEI and BfArM, the two German regulatory authorities, are to be involved to contribute with their know-how. The remaining questions are which type of study these trials are from a legal perspective and whether such studies are covered by the AMG (German Medicines Act) at all, since they are based on the new regulation in § 35a Para. 3b SGB V (Code of Social Law, fifth edition).

-      Cannabis Medicinal Products: The BfArM must conduct a “non-interventional, supportive data collection” for all cannabis medicinal products. Challenges in this regard are the 100 % off-label use and heterogenous administration and dosage forms which lead to low data quality.

Full presentation (predominantly in German language) available here

Accompanying “Streiflichter” (in German language) available here

V – Methodology of Obtaining Informed Consent according to §§ 40 ff. AMG (German Medicines Act) and §§ 20 ff. MPG (German Medical Device Act) in Consequence of “Zweites DSAnpUG-EU and GSAV”

In his second presentation, Professor Sträter explained the amendment to §40 AMG and §§20, 21 MPG caused by the second DSAnpUG-EU (Act to Adapt Data Protection Law to Regulation (EU) 2016/679) and the GSAV in regard to patient consent and the resulting inconsistencies between German and EU law.

The overall consequences for patient consent to clinical trials are as follows:

-      For drug trials, hand-written consent is retained (due to EU law).

-      According to EU law for medical devices hand-written consent remains binding.

-      Electronic patient consent suffices for pure medical device trials.

Caveat: In view of the EU law, resistance from other EU member states is to be expected.

Full presentation (in German language) available here

 

SanaClis is looking forward to continuing with this series of annual events in Frankfurt am Main on 15 May 2020.