Global drug development: Issues for the pharmaceutical industry and regulatory authorities
26-27th May 2004, Tokyo, Japan
Overview
Although most of the major pharmaceutical companies are moving towards simultaneous development and registration of new drugs in Europe and the US, the full integration of Japan into global development plans has yet to be achieved by many companies. It was therefore appropriate and timely that the CMR International Institute Workshop on Global Drug Development should take place in Tokyo, providing an opportunity for senior executives from industry and regulatory agencies to discuss the scientific issues and perceived regulatory hurdles to be overcome, on the way forward, towards fully integrated drug development.
Among the concerns that were discussed was the so-called ‘Japan gap’ – the lag time between filing new drug applications in the Western world and filing in Japan, which can result in a delay of several years before Japanese patients benefit from medicines that are available to patients in other parts of the world.
New Japanese Agency
The timing of the Workshop was also particularly opportune as it took place less than two months after the inauguration of the new Japanese Pharmaceuticals and Medical Devices Agency (PMDA), in April 2004. The Keynote presentation by the Senior Executive Director of PMDA, Dr Osamu Doi, gave delegates the opportunity to learn, at first hand, about the structure and objectives of the new agency. Of particular interest were plans to streamline the review process, to introduce a fast track system for the clinical investigation and registration of priority medicines, and to strengthen safety monitoring.
Syndicate recommendations
Following the customary format for Institute Workshops, all participants were involved in lively interactive Syndicate discussions. The Syndicates were asked to look at the immediate steps that could be taken to overcome barriers to global development and also to develop a vision for the future. Among the areas identified for action in the short to medium term were improving safety monitoring and information exchange through a global safety database, and taking steps to maximise the benefits of the harmonisation achieved through ICH, by improving the consistency of interpretation and implementation of guidelines. The need to develop guidance on establishing clinical endpoints and on adopting surrogate markers was also among the recommendations from the Syndicates.
A New Development Paradigm
Looking to the future, there was consensus that the time is right to initiate an international debate on a ‘new paradigm’ for drug development and regulatory review. It was recommended that the CMR International Institute for Regulatory Science should take a lead in this and build such discussions into its work programme for the coming years.
Among the visions for the future were proposals that a formal review of new medicines should take place at the end of Phase II rather than waiting for completion of Phase III, and that some innovative products that address unmet medical need could be released for early marketing on a ‘trial’ basis, with intensive safety monitoring. It was proposed that ‘rolling reviews’, as established in the US, should become the norm and even taken a stage further. It was envisaged that data could eventually be held in a central ‘data warehouse’, accessible to the authorities, that allowed reviews to take place on a continuous basis, ultimately obviating need for conventional submissions or final study reports.
Innovation and Change
In a thought-provoking presentation, Dr Murray Lumpkin, Principal Associate Commissioner, FDA, underlined the obligations of regulators to engage with industry in a debate on how to bring about change where this would optimise the development and review of innovative new therapies. He encouraged companies to become involved in the debate generated by the FDA white paper Innovation or Stagnation: challenge and opportunity on the critical path to new medical products. The belief that the time is right to start discussing some radical changes of philosophy and practice in global drug development was one of the main themes to emerge in the Workshop discussions.