Regulating personalised medicines
14-15th April 2003, Surrey, UK
The unravelling of the human genome and advances in genetic research are now opening up new horizons in the understanding of the science behind the ‘variability among individuals’. At the forefront of this research is the integration of pharmacogenomics and pharmacogenetics into the discovery and development of new medicines. Such medicines have the potential to be ‘personalised’ or tailored for optimal efficacy and minimal risk in the individual patient.
The implications of these developments for pharmaceutical research and regulation and the impact on practical and economic aspects of drug development and healthcare delivery were discussed at a Workshop convened by the CMR International Institute for Regulatory Science in April 2003 in Nutfield, Surrey, UK. It was clear from the discussions that the pharmacogenomic era has begun in earnest and that it is no longer a question of whether it will deliver a new generation of medicines, but when.
A concern that was re-iterated throughout the workshop was that, whilst pharmacogenomic research must be conducted within an appropriate regulatory framework, premature and inappropriate additional regulation could hamper development of the science. Dialogue between industry and regulators and a willingness to share experience of the issues is critical to moving forward.
In many cases pharmacogenomics may be integrated into the development process as one of many tools to improve discovery and increase knowledge of disease aetiology. The ‘ultimate’ personalised medicine, however, is one that is marketed in combination with a genetic diagnostic test, or with a labelling requirement that such testing be undertaken. This raises many regulatory issues that are yet to be resolved in relation to ensuring the sensitivity and specificity of tests and setting standards for their control.
There are also significant unresolved issues related to the economics of developing medicines for targeted subsets of the patient population rather than the traditional ‘one product fits all’ approach. There will also be major implications for the ‘payers’ who will need to be convinced of the cost benefits of medicines which will almost inevitably be higher priced, and will carry the cost of additional diagnostic testing.
There are also practical and economic issues for healthcare delivery infrastructures in moving towards an era where diagnostic genetic testing, as a prerequisite to prescribing medicines, could become the norm, rather than the exception. By its very nature, personalised medicine will mean greater involvement of patients in decisions about their treatment. A concerted effort is needed to present the new technology in a positive light using language and terminology that can be understood by the lay public.