Scientific Advice in the EU

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 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 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 you should 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. Choosing the right procedure for scientific advice (at the European Medicines Agency or at the National Authorities in the EU) and preparing well for the procedure is very important to assure your development program meets the requirements of the Regulatory Authorities.

This webinar will address which procedure is best depending on the circumstances, and how to choose among the National Authorities. In addition it will cover the preparation of the meeting (including briefing document). In addition, the European Medicines Agency offers scientific advice to support the qualification of innovative development methods for a specific intended use in the context of research and development into pharmaceuticals. The advice is given by the Committee for Medicinal Products for Human Use (CHMP) on the basis of recommendations by the Scientific Advice Working Party (SAWP). This qualification process leads to a CHMP qualification opinion or CHMP qualification advice. The procedure will be explained, as well as when it is best to use this procedure. Sometimes it is useful to request scientific advice and protocol assistance in parallel with the United States Food and Drug Administration or with health-technology-assessment (HTA) bodies.

The aim of parallel scientific advice with HTA Bodies is to allow medicine developers to gain feedback from regulators and HTA bodies at the same time, early in the development of a medicine. This helps them to establish the evidence that both parties will need to determine a medicine's benefit-risk balance and value.

Areas Covered in the Session:

• Introduction
• Scientific advice at the EMA
• Qualification of novel methodologies
• Parallel scientific advice: EMA and FDA
• Parallel scientific advice: EMA and HTA bodies
• National scientific advice
• Briefing document for scientific advice

Who Will Benefit:

• Regulatory Affairs personnel
• Senior management
• Project Managers
• Medical writers
• CRAs and CRCs
• QA / Compliance personnel
• Investigators
• Clinical Research Scientists
• QA / QC Auditors and Staff
• Consultants