Chair: Anna Berglind Vice Chair: Christoph Gerlinger
The purpose of the regulatory committee is to provide the Biostatistical viewpoint on regulatory issues. This includes promoting best practise, reviewing regulatory policy and guidelines, driving debate on future guidance, and engaging with statisticians in European regulatory agencies.
The committee meets approximately every 2 months by teleconference/webex and face to face once or twice a year.
The regulatory committee will achieve its aims by:
- Review of draft regulatory guidelines.
- Expert groups.For areas of major interest, the committee will form expert groups to discuss issues with the aim of forming an industry consensus to drive debate on future regulatory policy.
- Communication with the membership of the EFSPI organisations.
- Meetings with statisticians in regulatory agencies.
- Cross-industry initiatives.
The committee members are: Julie Anderson, Anna Berglind, Maylis Coste, Erika Daly, Anne Danniau, Daniel Evans, Chrissie Fletcher, Lesley France, Christoph Gerlinger, Teppo Huttunen, Kerry Gordon, Jürgen Hummel, Frances Lynn, Kaspar Rufibach, Antony Sabin, Ruthild Sautermeister, and Florian Voss
Link to PSI organogram (updated every year with annual report, members only access)
Committee operating guidelines | Committee resources folder
Our links to various regulatory guidelines can be found here
Link to pages with archived material on topics of special interest to the group.
2016 has been another exciting year for the Regulatory Committee. During the year the committee held seven regular committee meetings, held meetings with the EMA’s Biostatistics Working Party and the MHRA statisticians in London, and led the review of several regulatory guidance documents (more detail below). The Regulatory expert group on Subgroups continued the work on developing a best practice for subgroup analysis, and held a training event on subgroups at the PSI conference in Berlin May 2016. The group is currently finalizing a paper on the topic to be submitted to Pharmaceutical Statistics.
In June 2016 the chair of the Regulatory Committee transferred to Anna Berglind from Christoph Gerlinger. Christoph is now the Co-Chair of the committee, taking over that role from Lesley France. In addition, committee members have contributed to EMA and industry workshops on extrapolation, Clinical Trial Transparency and anonymization; estimands, as well as the successful 1st EFSPI Workshop on Regulatory Statistics held in Basel in September 2016.
Meetings with statisticians from regulatory agencies
The EFSPI/PSI regulatory committee met with the EMA’s BioStatistics Working Party on September 30, 2016. Topics discussed included: guideline on multiplicity Issues in clinical trials; statistical methodology for the comparative assessment of quality attributes in drug development; control of Type I error in bioequivalence studies, treatment switching in oncology; guidance on data monitoring committees; confidentiality of interim results; role of non-randomized data in regulatory decision making; and estimands.
On November 23 2016 the Regulatory Committee met with the MHRA statisticians in London. Topics discussed included: estimands; real world data; EMA publication of trial data; first in human studies; confidentiality of interim results; use of R and other specialist software; single arm trials for drug approval; parametric survival curve modelling; evaluation of safety and quantitative benefit-risk in drug development; and the role of statisticians and quantitative methods in drug development decision making.
One of the outcomes of these meetings is that the Regulatory Committee is initiating a workgroup on the topic of Confidentiality of interim results.
The regulatory committee commented on the following documents during the year:
A major area of focus during 2013 has been clinical trial transparency. The regulatory committee reviewed the EMA data transparency policy and contributed to the cross industry comments.
Members of the regulatory committee organised a session at the PSI conference to inform PSI members of the EMA transparency proposals. Committee members also contributed to the EFSPI Industry's position on access to clinical trial data and the EFSPI/PSI meeting which was held in August 2013. In the light of the large number of comments received on the policy the EMA have chosen to take more time to consider the comments. The EMA Board meeting agreed a set of key principles on 12 December 2013. The key principles include:
- a stepwise approach for implementation
- preparation for the publication of clinical study reports redacted as appropriate;
- development of a methodology for de-identification of patients;
- definition of a standard format for the submission of data.
The principles also foresee the introduction of preliminary steps prior to data access designed to address the risk of possible unfair commercial use of data, while ensuring proactive and non-selective access (‘use control’ not ‘access control’). The policy and an implementation plan was discussed at the March 2014 EMA Management Board meeting. A good overview of the current status of the regulatory agency activities is available to review.
Support for ABPI Meetings.
The ABPI asked the PSI Regulatory committee to help with a technical workshop in March 2013. Through the ABPI, members of the regulatory committee also became involved in activities on transparency led by the Welcome Trust.
Members of the regulatory committee participated in the ABPI/PSI workshop on technical standards for data sharing for old, current and future clinical trials on 5 December 2013, organised by the PSI chair.
Regulatory Document Review
A key activity of the committee is to compile and submit PSI/EFSPI comments on draft regulatory guidelines. The regulatory committee has also worked closely with Special Interest Groups (SIGs) to alert them to regulatory documents and to engage their expertise.
During 2013 the Regulatory committee worked with SIGS to submit consolidated comments on:
EMA - Baseline Covariate Document
FDA Enrichment document
The regulatory committee attempted to collate comments on a number of other regulatory documents but did not attract comments from PSI membership.
Multiplicity Expert Group
An article for SPIN was written to provide the high level information to members on the multiplicity expert group meeting early in 2013 and full details of the Multiplicity Expert group proceedings were published in Pharmaceutical Statistics. Multiplicity: discussion points from the Statisticians in the Pharmaceutical Industry multiplicity expert group Pharmaceutical Statistics Volume 12, Issue 5, September/October 2013, Pages: 255–259,
Regulatory Links to HTA
The other key area to highlight is the increasing links between regulatory authorities and HTA organisations. In 2013 there was the first joint work plan with EUNetHTA, a network of European health technology assessment organisations, outlining key areas of collaboration and the first workshop bringing the regulatory and HTA worlds together. The Regulatory committee have been maintaining close links with the HTA SIG on this topic and we expect to see this area evolving during 2014.