Scientific Advice in the EU - By Compliance Global Inc.

Overview

Scientific advice helps the company to make sure that it performs the appropriate tests and studies, so that no major objections regarding the design of the orphan indication tests are likely to be raised during evaluation of the marketing-authorization application. Such major objections can significantly delay the marketing of a product, and, in certain cases, may result in refusal of the marketing authorization. Following advice from the Regulatory Authorities increases the probability of a positive outcome.

The Regulatory Authorities give scientific advice by answering questions posed by companies. The advice is given in the light of the current scientific knowledge, based on the documentation provided by the company. It is not the role of the Regulatory Authorities to substitute the industry's responsibility for the development of their products.

Scientific advice is prospective in nature. It focuses on development strategies rather than pre-evaluation of data to support a marketing authorization application.

Scientific advice received from the Regulatory Authorities is not legally binding on the Authorities or on the sponsor with regard to any future marketing authorization applications for the medicine concerned. Protocol assistance is the special form of scientific advice available for companies developing designated orphan medicines for rare diseases.

In addition to scientific advice, companies developing an orphan medicinal product can receive answers to questions relating to the criteria for authorization of an orphan medicine. These include:

The demonstration of significant benefit within the scope of the designated orphan indication;
Similarity or clinical superiority over other medicines. This is relevant if other orphan medicinal products exist that might be similar to the product concerned and which have market exclusivity in the same indication.
Companies can request scientific advice or protocol assistance either during the initial development of a medicinal product before submission of a marketing authorization application or later on, during the post-authorization phase. Scientific advice and protocol assistance are particularly useful to companies developing a medicinal product when:

There appears to be no or insufficient relevant detail in European Union guidelines or guidance documents, or in Pharmacopoeia monographs, including draft documents or monographs released for consultation;
When the company chooses to deviate from the available guidance in its development plan.

Parallel scientific advice with the EMA and FDA may lead to an increased dialogue between the two agencies and sponsors from the beginning of the lifecycle of a new product, a deeper understanding of the bases of regulatory decisions, and the opportunity to optimize product development and avoid unnecessary testing replication or unnecessary diverse testing methodologies. Parallel scientific advice should focus primarily on important breakthrough drugs or important safety issues in the following areas which have been identified as clusters of interest between the agencies: Oncology, Vaccines, Orphan Drugs, drugs in the Paediatric Population, Nanotechnologies, Advanced Therapies, Pharmacogenomics and Blood products.

Parallel Scientific Advice procedures are conducted under the auspices of the confidentiality arrangement between the European Commission, the EMA, and FDA.

The parallel procedure with the HTA agreement helps to establish the evidence that both parties will need to determine a medicine's benefit-risk balance and value. and has four stages:

Pre-notification: Applicants engage early in informal discussions with HTAs and the Agency, announcing their intentions for the procedure, product and timescale, and specifying which HTAs will participate.
Pre-validation: Includes a teleconference with HTAs to discuss the scope, wording and clarity of the questions, and whether the material provided is sufficient to answer the questions posed. Applicants circulate their own documents throughout the procedure.

Meeting: A face-to-face meeting between all stakeholders, lasting approximately four hours, for which the applicant prepares the agenda according to priorities.
Outcome: Meeting minutes are circulated by the company for individual written HTA agreement. Regulatory scientific advice is provided in a CHMP advice letter. Sponsors may choose to share this letter with the participating HTA bodies.
Why Should You Attend

Scientific advice is when the Regulatory Authorities gives advice to a company on the appropriate tests and studies in the development of a medicine.

This is designed to facilitate the development and availability of high-quality, effective and acceptably safe medicines, for the benefit of patients.

Areas Covered in this Webinar

This webinar will address which procedure is best depending on the circumstances, and how to choose among the National Authorities. In addition, it will c