Regulatory Strategy in Clinical Development (EU)
Case study
Challenge
A large pharma customer sought expert regulatory support from ICON for a paediatric rare disease development program. Competitors were behind in development but there was a desire to maintain advantage through an optimised regulatory development plan. As a paediatric condition with treatment starting shortly after birth, ICON staff were required to carefully consider regulatory and ethical perspectives at every stage.
Solution
An ICON Regulatory Consulting Strategist was assigned to the project to provide the required support. Working closely with the customer’s team they contributed expertise to the regulatory activities, including:
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Provided comprehensive input on regulatory aspects of the development plan, outlining various scenarios which detailed opportunities for faster registration, special designations of interest, the optimal timings for health authority interactions and the pros and cons for each approach.
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Advised on various label claims, such as those relating to a disease modifying indication or specific limitations that may arise from different development approaches. Helped to outline which regulatory considerations may factor into future label discussions and whether they could be mitigated throughout development.
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rovided regulatory input on the selection of clinical endpoints in the absence of guidance that would meet both regulatory and payer expectations. Prepared for and supported parallel consultations with regulators and payers, to pressure test these ahead of the pivotal trials.
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Provided leadership or support on the delivery of the described services, such as orphan drug designation and scientific advice.
Outcome
ICON delivered on project timelines and expectations for the duration of the project, providing regulatory expertise and leadership to the project team in areas where it was not available within their own organisation. By appointing an ICON Regulatory Strategist, they were able to benefit from the strategist’s extensive expertise in the paediatric and orphan drug development space. Additionally, they benefited from the flexibility of applying those services as and when they were required, to achieve project success. Through ICON’s Regulatory Strategist’s contributions, the customer had a comprehensive RDP and Paediatric strategy detailing their abbreviated pathway to market. As a result, they were confident to stand behind this strategy in meetings with HAs and Payers and a successful orphan drug designation.
ICON continues to provide support to the customer as development continues.