產業新聞

**Tailored Scientific Advice for biosimilar development Report on the experience from the pilot (2017-2020) **

**Background **
A similar biological medicinal product (biosimilar) contains a version of the active substance of an already authorised original biological medicinal product (reference medicinal product) in the EEA. Similarity to the reference medicinal product in terms of quality characteristics, biological activity, safety and efficacy, based on a comprehensive comparability exercise, needs to be established for the marketing authorisation of a biosimilar product.

According to the applicable guidelines (Guideline on similar biological medicinal products (CHMP/437/04 Rev 1) and Guideline on similar biological medicinal products containing biotechnologyderived proteins as active substance: non-clinical and clinical issues (EMEA/CHMP/BMWP/42832/2005 Rev1) the extent and nature of the required non-clinical in vivo studies and clinical studies depends on the level of evidence obtained in the previous development step(s), i.e. quality and non-clinical in vitro characterisation, and it is recommended to discuss this in advance with regulatory authorities. Such discussions would require the Scientific Advice Working Party (SAWP)/Biologics Working Part (BWP)/Biosimilar Medicinal Products Working Party (BMWP)/Committee for Medicinal Products for Human Use (CHMP) to perform an in-depth review of data accumulated during the initial steps of development and subsequently make recommendations to applicants related to remaining uncertainties and the next steps of the development.

As regular scientific advice is intended to be prospective and would normally not include review of data, SAWP, BWP and CHMP agreed to explore the possibility of a more extensive scientific review of quality and non-clinical in vitro data gained from a specific development programme to allow a discussion on residual uncertainties or simplification of the development programme. Such in-depth review of analytical and functional quality comparability data could allow to further advise on the next steps of the development programme thereby further supporting the development of a biosimilar product in a targeted way.

An adaptation of the scientific advice procedure was seen as a valid instrument to adequately satisfy the concept of the stepwise approach recommended in the aforementioned guidelines. Based on the review of the quality comparability data, advice could be given on:

• differences identified at the quality level and their potential impact on the suitability of the biosimilar approach per se or on the need for and design of additional pre-clinical or clinical investigation.
• whether a reduced, i.e. tailored/simplified clinical development programme could be granted (e.g. waiver of clinical efficacy/safety trial).

To this end, in February 2017 EMA launched a tailored scientific advice pilot to explore further in practice the stepwise approach to development support for new biosimilar developments. The pilot was open to all types of biosimilars and applicants and was decided to run until completion of six scientific advice requests. In April 2020, the pilot was completed with the finalisation of the sixth procedure. Data on the pilot and an analysis of the experience is presented below.

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