The outcomes of clinical research quite regularly are only discussed within the scientific community and are seldom spread to the general public. With the release of the Clinical Trial Regulation 536/2014 in 2014, informing the public about clinical trial results will become mandatory in Europe once it actually enters into application towards the end of 2018. Lay language summaries intend to improve the transparency of clinical research in general and may help to return results of clinical trials to the individual patients concerned.
EFGCP and EFPIA have joined forces and in early May 2017 organised a workshop with stakeholders from patient and consumer health organisations, regulatory authorities, pharmaceutical industry and academia to discuss and develop best practices towards implementation of lay summaries. Specific guidance aimed at providing sponsors with recommendations and templates relating to meaningful lay summaries will be available in due course. On behalf of the EMA/EU Commission, a multi-stakeholder task force, led and coordinated by the UK Health Research Authority, drafted a European guideline on Summaries of Clinical Trial Results for Lay Persons.
The EFGCP and EFPIA workshop addressed important aspects of lay summary guidance that had been debated in detail by the HRA task force. These focused, for example, on: acceptable literacy levels (and specific tests to ensure easy readability); use of language (English mandatory plus languages of trial participants); reporting of adverse reactions (causal relationship to trial medication as judged by the investigator); reporting of outcomes (primary only to avoid “cherry-picking” among secondary); and inclusion of patients / patient organisation representatives in the lay summary writing process. The workshop audience discussed whether lay summaries generally should undergo an ethical review. Feasibility (e.g. time constraints, organisational process and funding) seems to preclude involvement of an ethics committee in the disclosure of a lay summary after trial completion and when patients are no longer on investigational treatment.
European-US alignment initiatives regarding lay summary concepts (e.g. initiated by TransCelerate) point out that they need to convey fair and balanced messages using strictly non-promotional language. Therapeutic changes must not be based on the results of a single trial presented in a lay summary and, accordingly, references to the approval status of a compound should not be made.
The guidelines available currently are a starting point for the implementation process that lies ahead of sponsors and authorities. The entire process needs to be oriented towards the target audience and patient organisations need to be involved strongly. User-acceptability testing, although not conducted by default for each lay summary, may help to avoid “data dumps”, to find creative and effective solutions, and to overcome current challenges. As a next step, following the release of the guidance documents, a multi-stakeholder task force should establish a road-map on best practices for the implementation of lay summaries. Both EFGCP and EFPIA stand firmly behind this development and are ready to work with stakeholders to make lay summaries meaningful, in order to meet the needs of the public and enhance the understanding of clinical research in general.
About the authors
Kerstin Breithaupt-Grögler studied medicine at the University Frankfurt, Germany (1976-1983). After state approval as physician and medical thesis (doctor of medicine, summa cum laude) worked as Junior Research Fellow at Center of Physiology, University Frankfurt (1983-1985).
From 1986 to 1995 Research Physician / Clinical Investigator at Center of Cardiovascular Pharmacology, Mainz-Wiesbaden, Germany and Board Certification in Clinical Pharmacology. Since 1995 working as freelance medical writer and consultant in clinical pharmacology. More than 20 years experience in planning, conduct, evaluation and reporting of Phase I to III clinical trials, writing of expert reports, scientific presentation and publication (28 original papers, more than 75 abstracts and oral presentations).
Founding member of the German Association of Applied Human Pharmacology (AGAH e.V.,Hamburg), Board Member since 1997; President 2012-2014, Past President since 2014,member of scientific programme committees, conduct of workshops and conferences,training courses in exploratory medicines development, qualification of study nurses.
AGAH Delegate to EUFEMED (European Federation for Exploratory Medicines Development,Brussels). Representing EUFEMED in the HRA (Health Research Authority) EU Taskforce Group, which is developing recommendations regarding the ‘EU Guidelines on Summaries of Clinical Trial Results for Laypersons’ intended for the European Commission Ad hoc Working Group.
Sini Eskola is working as Regulatory Affairs Director at the European Federation of Pharmaceutical Industries and Associations (EFPIA) since Feb 2014. She has a degree in pharmaceutical sciences (MSc) from the University of Helsinki. In EFPIA her main focus areas are leading and coordinating the regulatory policy and advocacy activities on clinical trials and transparence, including implementation of Clinical Trials Regulation, pharmacovigilance, regulatory information technology and environmental, health and safety aspects. She has previously worked over 5 years at AstraZeneca R&D Global Regulatory Affairs in Sweden and prior to that as an Executive Director of Finnish Pharmacists’ Society. She has vast experience as practicing community pharmacist. She is currently a member of the Executive Committee of Industrial Pharmacy Section of International Pharmaceutical Federation (2011-now).
Ingrid Klingmann is a physician, specialized in General Medicine, Clinical Pharmacology and Pharmaceutical Medicine with over 30 years of experience in different senior operational and managerial functions in pharmaceutical companies, CROs and academic sites, focussing on clinical trial management, ethical and regulatory aspects. Since January 2003 she has her own pharmaceutical development and site management support consulting company. Dr. Klingmann is Chairman of the Board of the European Forum for Good Clinical Practice (EFGCP). On behalf of EFGCP she was and is involved in different FP7- and IMI-funded projects (ICREL, PatientPartner, PharmaTrain, EUPATI, Combacte-Magnet) and with her company in the FP7-funded paediatric LENA project and the IMI-project SPRINTT. Currently Dr. Klingmann is also President of PharmaTrain Federation. Dr. Klingmann is Module Chair in the post-graduate Master Course in Regulatory Affairs at the University of Bonn, Germany, and is lecturer in the Diploma Course in Clinical Trial Practices and in the ECPM course at University of Basel and in the Pharmed course at Université Libre de Bruxelles, Belgium.1