A Faster Future for Medicines

From accelerated approval to breakthrough therapy designation, over the years regulators have expanded opportunities for getting promising drugs to patients faster.

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When the monoclonal antibody treatment Leqembi (lecanemab) first appeared in clinical trial readouts, the Alzheimer’s community watched closely. Early clinical trial data showed that the therapy, designed to target amyloid-beta plaques, could in fact reduce the sticky accumulations long considered a defining feature of the disease. For a field marked by decades of failed trials and incremental advances, it felt like a shift. And in January 2023, on the strength of the data, the FDA granted Leqembi approval for medical use in the US.

The pathway that made this possible was the FDA’s Accelerated Approval program, a mechanism created in the 1990s to speed access to treatments for serious conditions with unmet medical needs. The idea was straightforward: if a drug can demonstrate an effect on a well-validated surrogate endpoint—such as beta-amyloid reduction—it may be reasonably likely to confer clinical benefit, even if full evidence of such a benefit is not yet available.

Accelerated Approval is not an exemption from proving clinical benefit. Companies are still required to run confirmatory trials, but for diseases like Alzheimer’s, where trials can stretch for years, the program offers a pragmatic compromise between urgency and rigor.

Indeed, after securing accelerated approval, the drug’s sponsors completed the Clarity AD Phase 3 randomized, controlled trial, which demonstrated a measurable clinical benefit and in June 2023, the FDA converted Leqembi’s status from accelerated to full approval.

Tools for Speeding Development

Accelerated Approval is just one tool in the FDA’s toolbox for encouraging and expediting drug development. Several other designations are available to sponsors, each aimed at addressing different gaps in treatment landscapes. Priority Review, for example, shortens the FDA’s review timeline from the standard 10 months to 6 months for therapies that could represent significant improvements in the treatment, diagnosis, or prevention of serious conditions. Fast Track designation offers more frequent interactions with the FDA and the possibility of rolling review, allowing companies to submit completed sections of their application early rather than waiting for the entire dossier to be ready.

Breakthrough Therapy designation goes a step further. Reserved for drugs with preliminary clinical evidence suggesting substantial improvement over existing options, it folds in all the benefits of Fast Track while providing intensive FDA guidance and senior-level involvement. While sponsors can request these designations at various stages, timing matters: Breakthrough Therapy requests, for example, are ideally submitted no later than the end of Phase 2 to fully leverage the program’s advantages. Importantly, these designations are not mutually exclusive; a single therapy can qualify for multiple pathways depending on the evidence and clinical context.

Marketing on the Global Stage

Globally, regulatory agencies have developed parallel strategies to speed time to market. The European Medicines Agency offers accelerated assessment, which can cut review timelines from 210 days to 150 for products deemed of major public health interest. In the UK, the Medicines and Healthcare products Regulatory Agency recently launched the International Recognition Procedure, allowing the agency to lean on authorizations from trusted regulators to streamline its own decisions. Japan’s Pharmaceuticals and Medical Devices Agency likewise maintains several expedited pathways, including priority review and the Sakigake Designation for innovative therapies originating in Japan.

Despite these efforts, there is no universal application that unlocks global approval. Each jurisdiction requires its own review, often with its own data expectations and timelines. For companies, this patchwork can be costly and complex, underscoring the need for early planning around target markets and regulatory strategies. To help streamline the application process, the International Council for Harmonisation created the Common Technical Document, a standardized format for marketing applications that is now adopted by numerous regulatory bodies worldwide.

Leqembi’s winding but ultimately successful journey through Accelerated Approval in the US offers a clear illustration of why these pathways matter. Expedited programs can provide companies with meaningful strategic advantages, helping them move promising therapies through development more efficiently. More importantly, they can bring life-altering, and sometimes life-saving, treatments to patients years earlier than traditional timelines would allow.

References

  1. van Dyck CH, Swanson CJ, Aisen P, et al. Lecanemab in Early Alzheimer's Disease. N Engl J Med. 2023;388(1):9-21. https://www.nejm.org/doi/full/10.1056/NEJMoa2212948.
  2. US Food & Drug Administration (FDA). “Guidance for Industry Expedited Programs for Serious Conditions – Drugs and Biologics.” 2014. https://www.fda.gov/media/86377/download.
  3. US Food & Drug Administration (FDA). “Development & Approval Process | Drugs.” https://www.fda.gov/drugs/development-approval-process-drugs.
  4. European Medicines Agency (EMA). “Accelerate assessment.” https://www.ema.europa.eu/en/human-regulatory-overview/marketing-authorisation/accelerated-assessment.
  5. Medicines and Healthcare products Regulatory Agency (MHRA). “Guidance - International Recognition Procedure.” https://www.gov.uk/government/publications/international-recognition-procedure/international-recognition-procedure.
  6. ICH. “M4 : The Common Technical Document.” https://www.ich.org/page/ctd.

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