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A small pharmaceutical company focused on CNS developed a psycholeptic for a defined pediatric population to treat insomnia. The Sponsor wanted to extend the registered target indication to make their centrally authorised medicinal product available to more pediatric patients, including a wider range of neurogenetic disorders and rare diseases. The Sponsor, a longstanding partner of ÃÛÌÒ´«Ã½¡¯s for more than 20 years, contracted us for specialist regulatory consulting to support this indication extension without conducting additional clinical trials. The Sponsor also wanted to secure a 1-year extension to the period of market exclusivity. 

Extending a therapy to a new indication typically requires pivotal clinical trial data within the new target disease or condition. However, the target population at the cross-section of pediatric and rare disease populations limited the options for generating new, additional pivotal trial data. The Sponsor wanted to pursue this extension without new clinical trial data, requiring a strategic regulatory approach. Support had to come primarily from the pivotal study supporting the original marketing authorisation, supplemented with post-marketing data and extensive literature reviews.

Regulatory timelines posed another challenge. To benefit from an additional 1 year of market exclusivity, under Article 14(11) of , authorisation of a new indication must take place within eight years from the date of the first authorisation. Within the time limit, the marketing authorisation holder (MAH) must provide compelling evidence to demonstrate significant clinical benefit in comparison with existing therapies¡ªagain, typically requiring additional clinical trial data. No comparative clinical trial data was available and additional clinical studies were not feasible. This meant ÃÛÌÒ´«Ã½¡¯s Regulatory Consulting Solutions (RCS) team had to build a case for the market protection period based on justifying the significant clinical benefit in comparison to existing therapies in the new therapeutic indication. 

The RCS team supported this submission process from strategy through to execution. A dedicated regulatory project manager (RPM) mapped the entire process and streamlined communication and collaboration, managing both the external and internal stakeholders, including Sponsor teams, EMA contacts, and other ÃÛÌÒ´«Ã½ functions like medical writing and publishing. The RPM drafted the justification to support significant clinical benefit and the updates to the risk management plan and provided counsel on all other aspects of the Type II variation including pediatric and orphan drug considerations and submission strategy. 

Providing sufficient, compelling evidence to justify the target indication extension and market protection extension without new clinical data resulted in a comprehensive regulatory submission package. RCS prepared and published the eCTD dossier that included approximately 1,000 pages and 1,500 hyperlinks. 

A professional medical writer drafted the clinical overview and collaborated closely with the Sponsor to draft responses to the EMA¡¯s questions received during the assessment.

As a result of the comprehensiveness of the documentation and the efficient management of the process, the European Commission granted both indication extension and the additional year of market exclusivity. The EMA made the decision within nine months of the submission, following only one round of questions. ÃÛÌÒ´«Ã½¡¯s ability to create a compelling argument to secure important post-market extensions for the Sponsor reinforced the close collaboration between our two organisations, resulting in a high level of customer satisfaction and an ongoing positive business relationship.