The Issue

An important challenge for biopharmaceutical policy is balancing the objective of bringing new drugs to market quickly with the need to maintain rigorous safety standards. This challenge became especially relevant during the COVID-19 pandemic, when the rapid development and approval of vaccines showcased the potential of streamlined regulatory processes. While accelerating the time it takes to get drugs to market can save lives and address urgent public health needs, it also raises questions about unintended consequences, particularly regarding the quality and safety of approved therapies.

The Evidence

A new study published in The Review of Economics and Statistics highlights how regulatory innovation—particularly the FDA’s Breakthrough Therapy Designation (BTD) program—can reduce the time to market for critical drugs while maintaining safety standards, a key point for policymakers shaping the future of drug regulation.

Launched in 2012, the BTD program is designed to expedite the development of drugs targeting serious conditions for which there are not yet effective medical treatments. The program provides significant engagement and guidance from senior regulators throughout the development process for new drugs whose early evidence suggests there will be substantial improvement over existing treatments.

The drug-development and approval process for novel drugs involves several steps: drug discovery and preclinical testing, which can take as long as six years; then clinical research (spanning Phase I through Phase III trials), which can take up to nine years; and finally submission of a New Drug Application (NDA) to the FDA for review, which can take more than a year.

Figure 1: The drug-development process

Within this process, the BTD program has been found to:

1. Reduce clinical development time: The program reduced late-stage clinical development times by 23 percent. This reduction occurred in the period from Phase II trials (which test for efficacy and further evaluate safety) through the NDA submission stage, and in the period from Phase III trials (which aim to confirm efficacy and monitor side effects) through the NDA submission stage. This can save millions of dollars in trial costs and enable faster market entry for life-saving drugs.

2. Maintain safety standards: A primary concern with faster drug approvals is whether expedited timelines lead to safety compromises. The new study found that drugs approved through the BTD process were just as safe after approval as similar drugs approved through regular processes, illustrating that strict safety checks work well and underscoring the need to keep these high standards in place.

3. Support less-experienced firms: Less experienced firms saw a greater reduction in Phase III through NDA submission times compared to more experienced ones, suggesting the guidance and organizational support that the FDA provides through the BTD program are particularly valuable for them. While highly experienced drug developers often possess the regulatory expertise needed to design efficient trials, less experienced firms typically lack these resources. The support the BTD program provides helps bridge this gap, fostering competition and expanding the diversity of drug developers.

4. Streamlined clinical trial designs: The BTD program offers regulatory guidance to “ensure that the design of the clinical trials is as efficient as practicable, while scientifically appropriate.” Simplified designs help trials achieve their enrollment targets and produce requisite regulatory-grade data, addressing a key challenge that causes 20 percent of cancer clinical trials to fail. This finding underscores the potential of regulatory guidance to enhance clinical trial efficiency without compromising drug safety.

Takeaways for Policymakers

▪ Regulatory innovation is critical: The positive impact of the BTD program demonstrates how regulatory innovation can reduce clinical development timelines without compromising safety. Policymakers could explore expanding similar initiatives to address unmet medical needs, while ensuring rigorous oversight to ensure safety.

▪ Less experienced firms benefit the most: Programs like BTD illustrate how targeted regulatory support can help less experienced firms innovate and compete. Policymakers could enhance this impact by introducing additional incentives, such as grants, tax credits, and tailored funding programs, to foster diversity among drug developers.

▪ Long-term monitoring can spur continuous improvement: Ongoing evaluation of expedited approval pathways is important to ensure their long-term safety, efficacy, and societal impact. Policymakers could support longitudinal studies to refine such programs, mitigate potential risks, and uphold public confidence in these initiatives.

Charting the Future of Drug Regulation

The FDA’s BTD program exemplifies the potential of targeted regulatory innovation to streamline drug development without compromising safety. Its success offers valuable lessons for policymakers navigating the balance between speed and safety in approving life-saving therapies. By expanding and adapting frameworks like BTD and fostering innovation among less experienced firms, policymakers can promote a more efficient and inclusive pharmaceutical industry. At the same time, rigorous oversight and sustained evaluations are important to maximize societal benefits. Applying these lessons can spur innovation, improve public health, and ensure timely access to critical treatments.