Evidentiary requirements in clinical development: Synchronising phase 3 requirements to meet multiple needs
31st March – 1st April 2011, Geneva, Switzerland
A drug development programme is by its very nature serial, acquiring knowledge and generating evidence about a new medicine though clinical trials that each build upon preceding studies. However, companies today are increasingly obliged to consider more than the traditional requirements of quality, efficacy and safety, as success is now measured by achieving both regulatory approval and reimbursement. In the current environment, the regulator and the payer, who make separate decisions, review new medicines at essentially a similar time point and therefore often with the same underlying data. as companies plan future development programmes: the question is, how can inclusion of health technology assessment (HTA) requirements be conducted without making the drug development programme overly burdensome or complex?
The key area for debate surrounds phase 3 development, specifically, how practical changes to drug development design might improve efciency, reduce duplication and mitigate against a potential mismatch of outcome of regulatory and payer approval in terms of the clinical evidence. There are several experiments (pilot projects) in regards to parallel advice from both regulators and payers on the clinical development of a new product. The participants at this workshop considered whether such advice is leading to better decision making and by discussing the different pilots, sought to identify which aspects of parallel advice programmes are most effective. The workshop comprised numerous presentations by topic experts and a discussion of specific topics in the context of syndicate discussion groups.
Workshop objectives
The objective of this workshop was to answer several questions:
- Is there a collective responsibility to develop a high-quality evidence pool?
- As drug development is based upon the serial development of evidence, what is the role of the regulatory and HTA agencies in participating in the knowledge build-up and how can industry avoid the temptation to overcomplicate evidence development to try to meet conflicting requirements?
- Which phase 3 requirements can be aligned?
- The key technical areas within drug development programmes where the differing requirements of the regulator and the payer might be aligned were discussed.
- What is the best approach to achieving this alignment?
- Discussions were focused on how such alignment might occur in practice, considering whether this would best occur on a drug-by-drug basis, by therapy area, or in forums.
- Does shared scientific advice help?
- As payers are now beginning to offer advice on development programmes, the workshop examined the current experience of pilot joint or parallel scientific advice and sought to identify which approaches to advice work best for all parties.