Regulatory and HTA scientific advice for SMEs
Results of a survey on the current state of play from EuropaBio and the Deerfield Institute.
Brussels, 17 March 2015 – EuropaBio, the European Association for Bioindustries, and the Deerfield Institute have partnered to carry out a survey on the current state of regulatory and health technology assessment (HTA) advice with a special focus on small and medium sized enterprises (SMEs). The findings suggest early regulatory scientific and HTA advice may help SMEs to better prepare and manage their clinical programs toward facilitating both regulatory approval and positive HTA recommendations, and therefore contribute to their increased competitiveness.
“Despite agreement amongst all stakeholders that early interactions between regulatory, HTA authorities and SMEs would be positive, ensuring that these interactions actually take place is not straightforward. Given that many SMEs focus on the early stage of development and can be quite limited in terms of personnel and resources, the process of seeking such advice can be perceived as lengthy, formal, and cumbersome”, said Miriam Gargesi, Director for Healthcare Biotechnology at EuropaBio. However, the findings also show that both the authorities and the SMEs acknowledged the need to become more flexible, and to diversify and increase the number of interactions between them.
“Early dialogue activities have the potential to reduce drug development uncertainties related to regulatory and HTA assessments. While existing HTA scientific advice programs have made a good start and EuropaBio member companies have reported positive experiences so far, there are a number of other needs to be met, particularly in the areas of information, training and coordination”, said Paolo Morgese, Director of European Research for the Deerfield Institute and Chair of EuropaBio’s HTA Topic Group.
Phase III clinical trials are the last and vital development step before submitting healthcare biotech products for approval. While the costs for these trials are rising, the success rate is still 50% at phase III, according to the UK's Centre for Medicines Research. Success at phase III clinical trials is not the end of the story, as drugs will still have to go through regulatory and HTA evaluation processes before being accessible to European patients. Reviews from the European Medicines Agency, and to a larger extent from HTA authorities, have proven to be a significant challenge for biotech SMEs, often resulting in very limited access to new biotech drugs.
Against this context and the resulting significant financial burden incurred during phase III clinical trials, companies, regulatory and HTA authorities are starting to work together at earlier stages of drug developmentin order to make drug development more efficient. This could eventually help companies better understand what data the authorities need earlier in the process, thus leading to better designed clinical trials and fewer drugs failing at the approval stage, or not being reimbursed.
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