Spring Intro 2023

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03/03/2023

Research and Development Phase

Multiple rounds of scientific advice*

– Sponsor can ask for guidance from EMA about best methods/study design ● Early topics for discussion might include: – General advice on novel class of medicinal products – Appropriateness of toxicological and pharmacological tests – Choice of animal models

*Options now exist to do parallel EMA-HTA advice or parallel FDA EMA advice

– Interpretation and implementation of EU guidelines ● Later stage topics for discussion might include: – Aspects of study design including choice of endpoints, comparator, population studied, size of safety database – Appropriateness of the statistical analysis plan – Manufacturing, release, stability, comparability – Acceptability of the programme for either a conditional marketing authorisation or a marketing authorisation under exceptional circumstances

Access

Submission and validation of MAA

Research and development (several years before MAA)

Pre submission (~18 months before MAA)

EU Commission decision

Evaluation of MAA (~ 1 year)

EMA opinion

Continuous Safety Monitoring

EMA link: scientific advice & protocol assistance

The Organisation for Professionals in Regulatory Affairs

Research and Development Phase

Priority Medicines (PRIME)

Aims to enhance support for the development of medicines that target an unmet medical need ● Eligibility

– Offer major therapeutic advantage over existing treatments , or benefit patients without treatment options – Must be able to demonstrate the potential benefit to patients with unmet medical need using early clinical data ● Benefits include: – Rapporteur appointment to help build knowledge prior to MAA – KO meeting to receive guidance on overall development plan / Regulatory strategy – Dedicated PoC at EMA (otherwise not assigned until Eligibility confirmed)