Module 6

02/10/2024

Regulatory Pathway Plan Obtaining scientific advice with agencies can help mitigate regulatory riskd

Considerations

Challenges

Identify which agency

A high resourced team required

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• National; EMA; FDA; JP; EMA/HTA; EMA/FDA,

• Preparation and delivery to short timelines: briefing package; A-LoQs • Development plans are too premature to discuss with the agency,

• Identify the appropriate stages during development and the actual timing: PRIME; Ph2; Ph3; MAA ready

Identify gaps in the development plan

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Data not ready to share with the agency

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Identify the topics of engagement

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• Readiness to discuss key decisions on certain aspects of the development plans with the agency. In some cases substantial detail is required • Willingness to change development plans upon health authority discussions (otherwise justified) • PRIME: EMA PRIME coordinator and Rapporteur updated periodically on development plans and dates of future engagement timelines and topics and progress check on changes. Important when product is earlier in development and engagement may be less frequent. • Resolving divergences between HA vs HTABs

• Identify whether the company is ready to share any data: preclinical, clinical, pharmacology, quality. Ideal to seek endorsement • Define company position for each topic of engagement and be ready to present the development plans • Frame the questions in way to spark scientific discussion. Avoid posing too many “ Do you agree….” type questions. • Planning is key. E.g.: EU CHMP SA can take up to 7 to 8 months from briefing book preparation to actual CHMP written advice.

The Organisation for Professionals in Regulatory Affairs

Lecture 1: Part 1 – Develop and Shape the Brand

25

Clinical Development Strategy: Define Pathway to MA Acceleration to MA is dependent on designs, disease and development

Traditional development pathway • Distinct clinical phases

Accelerated development pathway • Unique adaptive clinical trial designs that combines Ph1 & Ph2 evaluates can help to accelerate the product development and thus could meet the unmedical need earlier Potential to apply for acceleration of MA with or without EMA PRIME assistance or FDA breakthrough •

The Organisation for Professionals in Regulatory Affairs

Lecture 1: Part 1 – Develop and Shape the Brand

26

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