SMEs need to face scientific advice head on

Sweden 7-9 October – The number of SMEs failing to achieve marketing authorisations was a hot topic at The Organisation for Professionals in Regulatory Affairs (TOPRA) annual symposium this week.

The symposium, organised in conjunction with the Medical Products Agency, Sweden, looked at the regulatory challenges for and support to SME companies.

Melanie Carr from the EMEA’s SME Office provided the latest information on the EMEA’s SME Initiative, which was introduced in 2006 to promote innovation and development of new medicinal products through a number of incentives including a 90% fee reduction on Scientific Advice.

Ms Carr expressed her delight at the growing number of companies assigned SME status. However, discouragingly, only 43% have been successful in securing a marketing authorisation with a large number of companies not following the Scientific Advice provided.

The SME Day was chaired by Margareth Jorvid, LSM group, Sweden who warned SMEs: “...Scientific Advice is a crucial area for SMEs, therefore when you ask for it you need to be prepared to listen to the answers. If you don’t follow the feedback then the Scientific Advice will not be useful...”

Ms Carr commented: “It’s still early days. We only started our SME initiative in 2006... We have seen that the major objections do run high in areas of clinical efficacy and quality.”

Quality objections included: Process documentation incomplete, process validation incomplete, levels of impurities too high, setting of specifications not justified, lack of demonstrated consistency of lots, comparability between different sites not addressed, lack of GMP certification and stability data lacking.

Clinical objections included: discrepancy between studied patients and proposed indications, insufficient clinical package – one pivotal study, inadequate trial design, efficacy not demonstrated to significantly robust level, primary endpoint is not statistically significant, choice of dose not sufficiently justified, predefined criteria for clinical relevance not met, Inconsistency in statistical methods between protocol and report, multiplicity issues and data do not allow comprehensive evaluation of safety profile.

Melanie Carr concluded the meeting with the recommendation that SMEs seek Scientific Advice as early as possible and avoid premature filing. And, with the good news, that many of these companies, whose first application failed, are continuing to develop their application and are now in the stage of resubmitting the data or have resubmitted the data.

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