Quality decision making: Procedures and practices in drug development and the regulatory review
7-8th December 2006, Surrey, UK
Benefit and risk for patients, the success or failure of companies’ multi‑million‑dollar research projects, and the credibility of regulatory agencies as guardians of public health all depend on the quality of decisions made at key stages as new medicines progress from laboratory research to clinical development, regulatory review, approval and post‑marketing use.
The Workshop held by the CMR International Institute for Regulatory Science in December 2006 examined the factors that contribute to quality decision making. The relevance of the topic was underlined by contemporaneous events, including the publication in the UK of recommendations from an Expert Scientific Group on first‑in‑human trials, and the decision by a major pharmaceutical company to suspend advanced Phase III development of a cardiovascular medicine.
Scope of discussions
Chaired by Thomas Lönngren, Executive Director of the European Medicines Agency (EMEA), the first session focused on best practices within companies and regulatory agencies. Presentations explored critical decision points across development, the role of organisational culture and quality management, and initiatives to improve the consistency and transparency of regulatory review.
Speakers described national and international initiatives on Good Review Practices, ongoing academic work on embedding quality into regulatory assessment, and exploratory projects to gather structured feedback from both agencies and industry following major regulatory reviews. Early reactions from participants supported the feasibility and value of these approaches.
A structured approach to decision making
The second session examined models to improve the quality and consistency of benefit‑risk decision making. A range of qualitative and quantitative methodological approaches was reviewed, including multi‑criteria decision analysis, alongside less well‑known frameworks that have been investigated in academic and regulatory settings.
Industry and regulatory perspectives highlighted the importance of drawing on epidemiological data, improving the documentation of benefit‑risk judgements, and adopting more consistent procedures and criteria for regulatory decisions. Case studies illustrated the complexity of decisions where substantial patient benefit must be balanced against significant safety concerns.
Including patients’ views
The Workshop concluded with a discussion on the role of patients in decision making. Emphasis was placed on partnership between science, regulators and society, and on the value of incorporating patient perspectives into ongoing discussions about clinical trial design, regulatory review and risk‑benefit assessment.
Syndicate discussions
The Syndicate groups considered best practices for quality decision making by companies and regulatory agencies, as well as the advantages and limitations of transparency during regulatory review. A general formula for good decision making was identified as the combination of clear, well‑defined processes, consistent application, and talented, well‑trained people.
Company processes
Good practices identified for companies included establishing independent and objective project review processes at key milestones, ensuring multidisciplinary input at appropriate management levels, using a target product label to guide development decisions, incorporating health technology assessment considerations early, involving relevant stakeholders such as patients and clinicians, and encouraging data sharing to prevent duplication of effort and improve learning.
Agency procedures
For regulatory agencies, recommendations centred on Good Review Practices, lifecycle management, the use of peer and advisory reviews, quality management systems to monitor performance, and more structured approaches to benefit‑risk assessment using common templates and criteria.
Transparency of decision making
Transparency was recognised as an important contributor to quality decision making, but not as an objective in itself. The Workshop distinguished between passive transparency, through publication of review information, and active transparency, involving greater stakeholder participation. Benefits include increased public confidence and understanding, while disadvantages relate primarily to resource demands and potential misuse of information.