The pharmaceutical industry is undergoing rapid transformation as the FDA (U.S. Food and Drug Administration) modernizes its processes and introduces new accelerated approval pathways. These pathways allow for faster approval of drugs based on early evidence of effectiveness, particularly when they treat serious conditions with unmet medical needs.
For professionals in regulatory affairs, clinical development, and related disciplines, these changes are not just about faster market access—they represent an urgent need to adapt learning strategies, training programs, and internal processes to stay compliant with evolving standards. This means integrating new guidelines into role-specific learning, updating Standard Operating Procedures (SOPs), and building training that supports ethical decision-making in real-time. The implications of these changes extend across the entire drug development lifecycle, making continuous regulatory training a critical component for maintaining compliance, safeguarding patient safety, and upholding ethical standards.
A new era for accelerated approvals
With these shifts in regulatory frameworks, we are entering a new era for accelerated approvals. Recent FDA modernization efforts, such as the FDA Modernization Act 2.0 and new guidance on advanced manufacturing technologies, have introduced novel regulatory expectations and tools, including the use of microphysiological systems (e.g., Organs-on-Chips) and real-world evidence (RWE).¹˒²
A significant example of RWE playing a role in accelerating approvals is the FDA's approval of KYMRIAH® (tisagenlecleucel, Novartis), a CAR T-cell therapy for leukemia, based on a combination of clinical trial data and RWE. This demonstrates the FDA's evolving approach to integrating personalized medicine into the approval process. For internal training leaders, this means that teams must not only understand new tools—such as RWE and micro-physiological systems—but also know how to implement them appropriately within FDA-regulated development and approval processes.³
To support these evolving regulatory expectations, the FDA is introducing more specific guidance in emerging areas of drug development. The FDA now provides explicit guidance on advanced cell culture systems, such as the use of 3D cell cultures to simulate human biology more effectively than traditional 2D methods. Similarly, batch uniformity guidelines have become critical in ensuring that gene therapies like ZOLGENSMA® (onasemnogene abeparvovec-xioi, Novartis) maintain consistency across production. These shifts reinforce the need for focused, up-to-date training that keeps pace with emerging science and risk-based quality expectations.⁴˒⁵
Increased scrutiny on confirmatory trials
Accelerated approvals based on surrogate endpoints now come with more stringent expectations for confirmatory trials. The FDA requires sponsors to meet pre-established milestones and submit robust data on long-term safety and efficacy.⁶
A prominent example is ADCETRIS® (brentuximab vedotin, Seagen), which received accelerated approval for treating Hodgkin lymphoma based on a surrogate endpoint (progression-free survival) but was required to demonstrate overall survival in subsequent trials.⁷
Similarly, KEYTRUDA® (pembrolizumab, Merck), approved for multiple cancer indications including melanoma and non-small cell lung cancer, was subject to stringent milestone tracking and data transparency requirements as part of its post-marketing commitments.
While training programs have always addressed milestone management, there’s now an increased focus on it, with an added emphasis on the need for cross-functional coordination to meet FDA timelines. To meet these milestone requirements and ensure cross-functional alignment, teams must also understand how to incorporate and validate RWE—from sources such as electronic health records (EHRs), insurance claims, and observational studies—into their regulatory submissions. The FDA increasingly expects RWE to support both initial accelerated approvals and subsequent confirmatory trials.
The growing role of RWE in regulatory decisions—highlighted by the approval of LEQVIO® (inclisiran, Novartis), cholesterol-lowering drug for hyperlipidemia—underscores the need for teams to adapt their training programs to effectively integrate and validate this data in regulatory processes.⁸
Evolving responsibilities for pharmaceutical teams
As organizations update their training programs to address evolving FDA requirements, it’s equally important that these programs clarify the shifting responsibilities across key pharmaceutical functions. In addition to understanding new submission protocols and data expectations, training must also ensure that each role understands its new or expanded responsibilities within the regulatory landscape.
Regulatory Affairs: Must monitor regulatory updates/new guidance, such as the FDA’s Digital Health Innovation Action Plan. For example, the approval of IDx-DR (Digital Diagnostics), the first FDA-approved AI diagnostic tool for diabetic retinopathy, highlighted the need for regulatory professionals to stay up to date on new guidance for software and digital therapeutics.⁹
Medical Affairs: Need to translate complex clinical data and RWE into compliant communications. For example, the Medicare coverage of CAR-T therapies was influenced by medical affairs teams effectively communicating clinical data and RWE to demonstrate the potential impact on patient outcomes, ensuring regulatory compliance while pushing forward innovation.¹⁰
R&D and Clinical Development: Face increased pressure to design adaptive trials that meet accelerated approval criteria and anticipate confirmatory study requirements. For example, an adaptive trial design was employed in Bristol-Myers Squibb's OPDIVO® (nivolumab) program, where early-phase results from smaller patient populations were leveraged to accelerate trial timelines and move towards FDA approval.¹¹
Commercial Teams: Must be acutely aware of the boundaries between scientific exchange and promotion, especially pre-approval. A key example is the FDA’s scrutiny of pre-approval promotion for drugs like KYMRIAH® (Novartis), where concerns were raised about promoting the drug before full approval, potentially influencing public and clinical expectations.
The role of organizational culture in compliance
As regulatory frameworks evolve, the importance of a strong compliance culture cannot be overstated. A culture of compliance goes beyond just following regulatory updates; it is about fostering an environment where ethical practices and personal responsibility are deeply ingrained across all levels of the organization.
Employees, particularly in regulatory, clinical, and commercial roles, must feel accountable for the integrity of the data and decisions they contribute to. A culture that emphasizes individual accountability—where every team member understands their role in upholding standards—can mitigate regulatory risks and reinforce a commitment to patient safety.
Compliance training plays a crucial role in shaping this culture. Training must not only address the technical aspects of new FDA guidelines but also emphasize ethical decision-making, recognizing gray areas, and the personal responsibility each employee has in ensuring compliance. Tailoring training for specific roles (e.g., regulatory affairs, clinical trials, commercial) allows for a nuanced approach that acknowledges the varied responsibilities and challenges each department faces.¹²
Conclusion: Prioritizing regulatory training in a changing environment
By embedding regulatory training into every stage of the drug development process—starting from preclinical testing through to post-marketing surveillance—organizations can ensure their teams are prepared to navigate the complexities of modern FDA regulation. This includes staying ahead of evolving requirements around accelerated approvals, RWE integration, confirmatory trials, and post-marketing commitments. As seen with the approval of drugs like KYMRIAH® (Novartis), and ADCETRIS® (Seagen), RWE and confirmatory trials are becoming standard practice in the approval process.
To maintain compliance and safeguard patient safety, all departments must align their efforts, embracing continuous learning and collaboration to meet these regulatory challenges head-on. At the same time, these evolving regulations present valuable opportunities to improve and expedite patient care, ensuring that innovation can move forward responsibly, with both safety and efficiency in mind.
References
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U.S. Food and Drug Administration. Cellular & gene therapy guidelines. Updated 2024. Accessed June 2025.
ECA Academy. New FDA guidelines: Batch uniformity and drug product integrity / advanced manufacturing technologies. Updated 2025. Accessed June 2025.
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U.S. Food and Drug Administration. FDA approves brentuximab vedotin with lenalidomide and rituximab for relapsed or refractory large B-cell lymphoma. Updated 2025. Accessed June 2025.
U.S. Food and Drug Administration. Accelerate approval program. Updated 2024. Accessed June 2025.
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