The European Medicines Agency (EMA) has recently published a booklet covering the process undertaken for medicines for human use and which are authorised through the EU centralised procedure. With a user-friendly layout, it communicates the steps that are taken from the initial research stages of a new medicine to when it is eventually marketed to the public.

The convenient and accessible manual explains how the EMA provides scientific advice to support the timely and sound development of high-quality, effective and safe medicines, for the benefit of patients. Medicine regulators have unique knowledge and experience of how medicines should be developed, and are able to provide specific scientific advice where existing general guidelines only address general situations. Scientific advice is all about advising medicine developers on the most appropriate way to generate robust evidence on a medicine’s benefits and risks. The advice provided builds on existing scientific guidelines but, more importantly, is tailored to the specific medicine and the group of patients in need of care.

Patients are often involved in scientific advice. They are invited to share their real-life perspective and experience in relation to a particular medicine in their disease area. This can help medicine developers and regulators better understand what will work for that patient group and what they consider important.

Moreover, discounts in application fees have been made available to certain groups. There is a 75% fee reduction for medicines for rare diseases (a.k.a. ‘orphan medicines’), whereas SMEs are entitled to a 90% fee reduction.

Questions during scientific advice can relate to quality aspects, methodological issues, clinical aspects and non-clinical aspects. Some of the questions to be addressed may include:

• Are the patients to be included in a study sufficiently representative of the population for whom the medicine is intended?
• Does the study last long enough and include enough patients to provide the necessary data for the benefit-risk assessment?
• Is the medicine being compared with an appropriate alternative?
• Are the plans to follow the long-term safety of the product appropriately designed?


Read the booklet in full here for more information.