Development & Regulatory Strategy

Scientific Advice is a formal process where pharmaceutical companies consult regulatory agencies, such as the EMA or national authorities, for guidance on scientific and technical aspects of drug development. This ensures that development plans, including preclinical and clinical studies, align with regulatory expectations, optimizing the chances for successful approval.

Pharmaceutical companies and other sponsors involved in the development of medicinal products, including drugs and biologics, are eligible to request Scientific Advice. This includes entities developing innovative therapies, generic drugs, biosimilars, and advanced therapy medicinal products (ATMPs). The procedure is particularly relevant for those seeking guidance on complex or novel aspects of their development plans to ensure regulatory alignment.
Also, it is important take into consideration financial aspects of RMS state fees during MAA as well during whole product lifecyle.

Obtaining an Orphan Drug Designation (ODD) offers significant benefits, including market exclusivity for seven years in the U.S. and up to ten years in the EU, tax credits for clinical trial costs, and exemptions from certain regulatory fees. Sponsors may also become eligible for grant funding and receive regulatory assistance from agencies like the FDA or EMA. Additionally, ODD can lead to faster review processes, with potential for priority review and accelerated approval, helping to bring the drug to market more efficiently.

It's recommended to apply for Orphan Drug Designation (ODD) as early as possible in the drug development process, ideally before starting clinical trials. The application can be submitted at any stage before the submission of a marketing application (such as a New Drug Application [NDA] or Biologics License Application [BLA] in the U.S.). Early designation can help sponsors take full advantage of the benefits, such as market exclusivity, tax credits, and regulatory guidance, throughout the development process.

A Pre-Authorization Good Manufacturing Practice (GMP) inspection is typically triggered when a company submits a marketing authorization application (MAA) or a similar regulatory submission (e.g., New Drug Application [NDA], Biologics License Application [BLA]) to a regulatory agency like the FDA or EMA.