This article is part of a multi-part series on the topic of targeted medicines.
Navigating regulatory frameworks for targeted medicines can be daunting due to their novelty and complexity1.. Ensuring compliance with evolving regulations and addressing regulatory concerns about safety, efficacy, and quality requires expertise and resources.
For example, regulatory agencies may require extensive data on biomarker validation2, patient selection criteria, and long-term follow-up to support approval decisions. Given that complexity, navigation of the regulatory pathway for targeted therapies also requires close collaboration between sponsors, researchers, and regulatory authorities.
Companies should engage with regulatory agencies early in the development process to gain insights into regulatory requirements and expectations. Such collaboration can help identify potential challenges and ensure that development plans align with regulatory requirements.
In fact, experience shows that companies that develop targeted medicines and collaborate with regulatory agencies that have experience with targeted medicines benefit in several ways:
- Expert guidance: Regulatory authorities that have experience with targeted medicines can provide expert guidance and advice for companies, helping companies navigate complex regulatory pathways and avoid potential pitfalls.
- Efficiency: Regulatory agencies with the right experience can help streamline the regulatory review process, reducing delays and expediting the approval timeline for targeted medicines 3. Their familiarity with regulatory standards enables them to efficiently assess submissions and provide timely feedback.
- Risk mitigation: Experienced regulatory agencies can help companies identify and mitigate potential regulatory risks early in the development process. By proactively addressing regulatory concerns and ensuring compliance with regulatory requirements, companies can minimize the likelihood of regulatory setbacks and delays.
- Enhanced credibility: Companies that work with reputable and experienced regulatory agencies can enhance the credibility of their regulatory submissions. Regulatory agencies with records of thorough and rigorous reviews lend credibility to the approval process and instill confidence in stakeholders such as investors, healthcare providers, and patients.
- Global influence: Regulatory agencies that have experience working other global health authorities can help companies navigate regulatory requirements across multiple jurisdictions. Their knowledge of regional differences in regulations and their relationships with regulatory authorities worldwide can facilitate market access for targeted medicines on a global scale.
Contributors:
Dennis Earle
Dennis has 10 years of oncology consulting experience and more than twenty years of experience in strategic planning, program management and clinical development within the bio-pharmaceutical industry.
Christian K Schneider
Christian is Chief Medical Officer, Strategic Product Development Consulting, and Vice President at PharmaLex.
Jörg Schneider
Jörg is a translational biopharmaceutical expert with 22+ years of experience.
