The European Medicines Agency (EMA) can give scientific advice and protocol assistance to companies involved in developing medicines. For human medicines, scientific advice and protocol assistance are given by the Committee for Medicinal Products for Human Use (CHMP) on the recommendation of the Scientific Advice Working Party (SAWP).
Scientific advice is when the Agency 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.
Companies can request scientific advice from the EMA at any stage of development of a medicine, whether the medicine is eligible for the centralised authorisation procedure or not.
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-authorisation application. Such major objections can significantly delay the marketing of a product, and, in certain cases, may result in refusal of the marketing authorisation. Following the Agency’s advice increases the probability of a positive outcome.
The Agency gives 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 CHMP 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-authorisation application.
Scientific advice received from the Agency is not legally binding on the Agency or on the sponsor with regard to any future marketing-authorisation 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 authorisation 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.
Requesting scientific advice
Companies can request scientific advice or protocol assistance either during the initial development of a medicinal product before submission of a marketing-authorisation application or later on, during the post-authorisation 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.
For details on how to apply, see how to submit a scientific-advice or protocol-assistance request.
Scientific advice on post-authorisation safety studies (PASS)
Starting in July 2015, EMA is launching a 12-month pilot to encourage companies to seek scientific advice for PASSs for medicines. This voluntary, optional procedure will help to improve the design of studies meant to collect further information on a medicine's safety once it is on the market. This pilot will build on the expertise of the Agency's Pharmacovigilance Risk Assessment Committee. The main focus of the pilot will be non-imposed PASSs, which are studies that are not a condition to the marketing authorisation.
For more information, see:
- Question 10 on 'Scientific advice for safety studies NEW July 2015' on our page Post-authorisation safety studies: questions and answers.
Parallel scientific advice with the Food and Drug Administration
The Agency provides scientific advice and protocol assistance in parallel with the United States Food and Drug Administration (FDA):
Parallel scientific advice with health-technology-assessment bodies
The Agency also offers parallel scientific advice with health-technology-assessment (HTA) bodies. The aim of this is to allow medicine developers to gain feedback from 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.
A pilot for parallel scientific advice was launched in July 2010. Around 25 procedures had been finalised or were ongoing as of November 2013, covering indications such as diabetes, heart failure, lung cancer, breast cancer, pancreatic cancer, melanoma, mesothelioma, asthma, rheumatoid arthritis, multiresistant infections, food allergies, diabetic gastroparesis, Alzheimer’s disease, depression, osteoporosis and three rare conditions.
The parallel procedure 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.
A draft best practice guidance for EMA-HTA parallel scientific advice was published for public consultation in May 2014.
Applicants for these procedures are welcome to arrange a discussion by emailing email@example.com.
- How to submit a scientific advice or protocol assistance request
- Fees and fee reductions for scientific advice
- Qualification of novel methodologies for medicine development
- Scientific advice and protocol assistance: Regulatory and procedural guidance
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- European Medicines Agency guidance for applicants seeking scientific advice and protocol assistance (14/10/2014)
- CHMP protocol assistance scientific advice briefing document template (18/06/2010)
- Template letter of intent for request of scientific advice or protocol assistance (28/07/2015)
- Template letter of intent for request of parallel health-technology-assessment scientific advice / protocol assistance (28/07/2015)
- Template letter of intent for request of qualification of novel methodologies to the Scientific Advice Working Party (26/06/2015)
- The dates of 2016 Scientific Advice Working Party meetings and deadlines for submission of scientific advice, protocol assistance, qualification of biomarkers and parallel European Medicines Agency-health-technology-assessment requests (17/09/2015)
- The dates of 2015 Scientific Advice Working Party meetings and deadlines for submission of scientific advice, protocol assistance, qualification of biomarkers and parallel European Medicines Agency / health-technology-assessment requests (28/07/2015)