Regulatory solutions for uncharted scientific waters

Not all therapeutic areas have scientific guidelines or established precedents on which to base clinical development plans. This can be particularly problematic in rare diseases and even more so in those with predominantly pediatric populations. ICON’s Regulatory Consulting Solutions (RCS) staff recently assisted a large pharma partner by providing regulatory leadership to project teams developing novel endpoints for a rare neurodevelopmental disorder, guiding the regulatory strategy necessary to obtain agreement from international regulatory authorities and payers for novel endpoints. This support ensured project team, executive and investor confidence in these novel endpoints ahead of their use in pivotal studies.

This rare disease affects pediatric populations. It can be identifiable very early, even shortly after birth and diagnosis is possible from as little as 6 months. In addition to dealing with vulnerable pediatric populations, the Sponsor was faced with the challenge of no scientific guidelines, no established precedent and no appropriate or validated instruments necessary to demonstrate clinical efficacy. With so little predetermined guidelines and yet a competitive market, gaining agreement with key regulatory authorities for any endpoint selection was an essential step on the path to pivotal trials.

ICON RCS Regulatory Strategy staff provided EU regulatory leadership to a development team of therapeutic, health economic, biostatistics and other experts on setting the engagement strategy with regulators for a novel endpoint strategy. 

As part of the regulatory strategy, RCS provided: 

• Regional and scientific expertise to identify relevant regional scientific guidelines with recommendations to support endpoint selection
• Regulatory insight gained from extensive experience, sharing regulatory perspectives on endpoint proposals
• Regulatory coordination, including setting and obtaining agreement for the regulatory authority engagement strategy and guiding the team during agency meeting preparations 

Leveraging ICON’s experience with regulatory authority meeting processes, the ICON RCS team members were instrumental in making the case to EMA and EU HTAs for a parallel consultation between payers and regulators. This gave the Sponsor the opportunity to garner insights from both sets of stakeholders in one meeting, providing clarity on the regulatory and potential economic value of the proposed endpoint.

ICON’s experienced RCS experts provided the Sponsor with a consultative partner to help them navigate regulatory guidelines and requirements despite the absence of established guidance or precedent for this particular therapeutic. RCS brought extensive knowledge of regulatory expectations and insights into their unique perspectives to inform effective endpoint selection. 

Furthermore, knowledge of cutting-edge European regulatory procedures helped secure a parallel consultation between payers and regulators during a period when such interactions are severely limited by HTA capacity. Streamlined interactions reduced the overall time needed for independent regulatory procedures. Collectively these steps helped to ensure confidence in the proposed endpoint strategy supporting further development in an area of unmet medical need.