Masterstudiengang "Drug Regulatory Affairs"
Stratified medicine: A new challenge for the academia, industry, regulators and patients ***
Prof. Dr. Dr. Thomas Bieber (Abschlußjahr: 2012)
Stratified medicine (SM) is the natural outcome of the progress in biomedical sciences and particularly in genomics which is about to generate a tectonic paradigm shift in our view to approach healthcare. It bears great expectations and hopes with regard to the availability of better medicinal products, especially for life-threatening diseases but also for other diseases with unmet medical needs. However, before reaching this aim, the stakeholders will face various challenges and the pressure for collaborative partnerships is substantial with regard to the increasing costs for R&D. Thus, public-private partnerships, such as translational medicine hubs, are expected to become of increasing value for the mandatory cross-talk between academia, national governments through their organizations, pharmaceutical industry, diagnostics industry and patient organizations or foundations.
Stratification of patients populations need the use of biomarkers which have undergone a thorough clinical validation and qualification process before being “fit-for-use” and have been included in clinical trials, eventually as surrogate endpoints. However, these processes need the availability of biosamples of high quality, handled under standardized conditions and flanked by appropriate clinical phenotypic information. The co-development of a drug with its companion diagnostic needs not only a strong and efficient partnership between diagnostics and pharmaceutical companies but also needs to be supported proactively by the regulators and by the release of appropriate guidelines.
For the pharmaceutical industry, SM signifies a shift from the blockbuster model to a niche-buster model. However, current analyses suggest that all stakeholders, including the industry, regulators, patients and payers, could benefit on the long term if patients have timely access to the correct medicines. The use of new strategies and tools for the clinical development such as adaptive trial design are expected to increase its efficiency and to foster the approval of more targeted and safe drugs.
The complex (co-)development process of diagnostic and drugs requires a proactive involvement of the regulatory bodies. Guidelines must be generated in an early and ongoing dialogue between regulators, academia and industry in order to optimize the development process and the way to marketing authorization for more efficient and safe drugs. Regulators will also have to establish guidance and new regulatory framework for the direct-to-consumer industry (DTC) of personal genomics (PsG) for which the clinical utility remains to be established.
Finally, the collection of the material for biobanks will be dependent on the patients and patient organizations. Foundations and initiatives such as EUPATI should be instrumental in the information of affected individuals by providing adapted educational programs. Thereby they could substantially contribute to their health literacy, a key element for their privacy, autonomy of choice and decision with regard to SM and PsG.