Scientific advice: EMA or national guidance on development before submission
Scientific advice is formal guidance from EMA or a national authority on the studies and data a developer should generate before applying for an MA.
Article 57(1)(n) of Regulation (EC) No 726/2004 gives EMA the task of advising companies on the conduct of the tests and trials needed to demonstrate the quality, safety and efficacy of a medicine. The advice is prepared by the Scientific Advice Working Party and adopted by the CHMP. For designated orphan medicines the same service is called protocol assistance under Article 6 of Regulation (EC) No 141/2000 and is available at a reduced fee. National competent authorities offer their own scientific advice, which is useful for products that will go through the decentralised procedure, and fees for EMA advice are set in Regulation (EU) 2024/568.
Advice can be sought at any stage, on quality, non-clinical or clinical questions, and can be repeated as the programme develops. The company submits a briefing document with specific questions and its own proposed answers, and EMA responds in writing after a discussion meeting where needed. Parallel consultation with health technology assessment bodies is possible for products that will need a joint clinical assessment, and EMA also offers qualification of novel methodologies such as biomarkers and digital endpoints.
The advice is not legally binding on either side, but the CHMP is expected to explain any departure from it at assessment, and applicants who ignore advice are expected to justify their choice. In practice a documented request for advice and a programme built on the answers reduces the risk of major objections at Day 120 and is routinely cited in assessment reports.
What goes wrong: asking broad questions that produce broad answers, rather than putting a specific proposal to the agency for confirmation; seeking advice too late to change the pivotal trial; and taking advice from one national authority and assuming other Member States will share it in a decentralised procedure. Companies also lose the benefit when they change the programme after the advice without going back for follow-up advice.
Under the new pharma package (not yet in force), scientific advice gains weight, because one route to an extra year of market protection requires clinical trials with a relevant comparator in line with EMA scientific advice; see exclusivity changes.
Timing
EMA runs scientific advice to monthly submission deadlines published for each year. After validation the standard procedure takes 40 days, or 70 days where the working party needs a discussion meeting with the company or additional expertise. The written advice is adopted at the CHMP plenary that follows the working party's conclusion. Follow-up advice on the same questions is handled in the same cycles, and national advice follows each authority's own timetable, often a few weeks from request to meeting.
Used in
Development teams research the guidance and precedents behind a development question before asking the agency in Workbench, with cited answers from EMA and ICH.
Related terms
- CHMP: Committee for Medicinal Products for Human UseThe CHMP is the EMA committee that assesses centralised applications and referrals for human medicines and adopts the opinions behind Commission decisions.
- Centralised procedure: single EU marketing authorisation via EMAThe centralised procedure grants one marketing authorisation valid across the EU and EEA after a single EMA assessment and a Commission decision.
- Accelerated assessment: shortened centralised assessment for major public health interestAccelerated assessment shortens the CHMP's review of a centralised application from 210 to 150 days for a medicine of major public health interest.
- JCA: joint clinical assessmentA joint clinical assessment (JCA) is the EU-level review of a new medicine's relative effectiveness under the HTA Regulation, shared by all Member States.
- Orphan designation: status for medicines for rare conditionsOrphan designation is EU status for a medicine for a rare condition, bringing fee reductions, protocol assistance and market exclusivity once authorised.
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