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Rising to the Challenge with Innovative Therapies:

How to Take Advantage of Regulatory Initiatives to Speed Access

Given the urgent need to help oncology and rare disease patients facing limited treatment options, many health authorities have established expedited review pathways and have formed international partnerships that optimise their regulatory resources. Their common aim is to accelerate access to innovative therapies, improve agencies’ review efficiency and tackle scientific challenges in a more unified manner.

Each key market’s expedited review pathway has its own set of criteria, processes and expectations. For example, while the US Food and Drug Administration’s (FDA’s) Breakthrough Therapy designation emphasises intensive guidance and rolling review, the European Medicines Agency’s PRIME scheme focuses on early scientific support and enhanced dialogue. Japan’s Sakigake designation and China’s Priority Review pathway offer additional incentives but have their own distinct eligibility requirements and procedural nuances.

The collaborative programs between health authorities vary in formality and depth, ranging from simultaneous regulatory submissions, joint reviews and shared assessments to independently made—but harmonised—decisions among participating countries.  For example, FDA’s Project Orbis enables concurrent submission, review and decision making for oncology products across multiple countries, while the World Health Organization’s (WHO’s) Collaborative Registration Procedure allows participating countries to conduct abridged reviews using assessments made by WHO or other stringent regulatory authorities.

Despite these initiatives, the regulatory landscape remains complex. Overlapping objectives, differing procedures and multiple jurisdictions can make it challenging for sponsors to identify the most efficient path for securing approval of new therapies across multiple countries. In practice, these programs do not always simplify the process but require careful navigation as part of a broader global development strategy. The key challenge is: How can companies maximize regional regulatory opportunities to align data requirements, avoid duplication of effort and accelerate timelines?

The answer lies in proactive planning, with extensive regulatory intelligence and cross-functional collaboration from early development stages onward. Most sponsors find that adopting such a comprehensive, tailored approach requires specialized resources that an experienced Contract Research Organisation (CRO), such as ICON, is uniquely positioned to provide.

We recommend that sponsors address the challenge with this broad advice in mind:

  • Begin planning early. This means defining your target product label early in development to ensure your clinical program generates the necessary data to support it. And it means engaging cross-functional teams in each target country well in advance of developing a global dossier.
  • Become and stay familiar with the regulatory landscape. A foundational knowledge of the coordinated review options available and how they align with the expedited approval pathways will inform your regulatory and clinical development strategies. Since regulatory frameworks are dynamic, it is important to monitor their evolution continuously.
  • Map out the criteria and timelines of expedited programs across major markets. This will allow you to synchronise clinical trial design, regulatory engagement and submission planning.
  • Engage regulators proactively.  By treating health authorities as strategic partners, you can gain valuable feedback on your target product profile, clinical development plan and regulatory expectations. This should help de-risk your program and align it with local requirements.
  • Design with the end in mind. Consider how your product will be evaluated in each market. Tailor your clinical trial designs to reflect local standards of care and regulatory expectations and to generate the data needed to support the desired label claims in each jurisdiction.
  • Remain flexible. Regulatory advice can vary across review teams (Health Authorities) and timelines and may diverge from published guidance. If access to a collaborative pathway is not possible, be prepared to pivot to sequential, country-specific submissions.
  • Leverage the experience of partners. Consider partnering with a company that has deep expertise, coordinates activities across functions and demonstrates operational agility. 

By capitalising on Health Authorities’ momentum in creating expedited approval pathways and collaborative assessment programs, sponsors can accelerate global access to critical therapies. However, developing an integrated strategy is inherently complex and requires careful planning and adequate resourcing. Time invested early in formulating a global approach will yield significant benefits at the marketing authorisation stage.

Accelerating-access-collage

For a more detailed examination of how submission regulations are converging, where they differ and how to meet them most efficiently, download our whitepaper, Accelerating access: Expedited and harmonised pathways for innovative therapies. 

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