Regulators are ramping up pressure on the biopharma industry in 2024. Pharma companies not only need to jump through new hoops for an increasingly complex suite of therapeutics like cell and gene therapies, but they’re also navigating technological changes and an increased demand to take a more patient-centric approach to drug development, said Aman Khera, vice president of regulatory science, strategy and innovation at Worldwide Clinical Trials.
The federal Inflation Reduction Act, aimed at reducing prescription drug costs for Medicare patients and trimming federal drug spending, will likely add to those pressures. The regulation could potentially reduce access to Medicare Part B and Part D-covered drugs and discourage development for certain types of drugs, such as small molecules.
“Some companies are already rethinking their approach to R&D, cutting projects and reallocating resources. Others are reconsidering whether to offer medicines in the U.S. because they may never be able to recoup costs,” she said.
These pain points may be particularly acute for smaller and emerging biopharmaceutical companies, which have a growing footprint in the industry. The number of emerging biopharma companies has increased by 4% each year for the past five years, and these companies produced two-thirds of all new drugs in 2022, according to IQVIA. Studies have suggested that by 2039, IRA provisions could result in a 31% reduction in profits and potentially lead to 135 fewer new drug approvals within the same timeframe.
Now, companies are pushing to develop effective medications on an accelerated timeline.
“While these two aims have often appeared at face value to be contradicting, this is especially the case in recent times,” Khera said.
“The FDA’s embrace of digital health is changing its clinical trial oversight to advance novel methodologies that improve access and convenience."
Vice president, regulatory science, strategy and innovation, Worldwide Clinical Trials
The modality landscape has expanded significantly to not only include traditional small molecules and proteins, but also peptides, antibody-drug conjugates, a variety of nucleotide-based therapies such as antisense oligonucleotides, small interfering ribonucleic acids, messenger RNAs, and cell and gene therapies, she said. This new crop of therapeutics brings new preclinical and drug development requirements and regulatory complexities.
“The rise in patient input into all aspects of drug development, including regulatory review, has also impacted medicinal product regulation. Regulators’ expectations of patient involvement in drug development are growing, even while new tools are helping to remove practical barriers to working with patients,” Khera said.
To stay abreast of regulatory requirements, Khera advises organizations to take a strategic approach to compliance in 2024. Here are the key trends she said will shape drug development regulations in the coming year.
An increased focus on digital technology
Several regulatory changes poised to take effect in 2024 aim to clarify rules around technology and data collection. This includes an update by the International Council for Harmonisation (ICH) to its Good Clinical Practice (GCP) E6(R3) draft guideline, which looks to better define best practices related to technology, software and remote elements, Khera said. The FDA also took steps to give organizations better guidance on how to collect remote data using digital health technologies in its final guidance, Digital Health Technologies for Remote Data Acquisition in Clinical Investigations.
Tips for regulatory success in 2024
Khera’s advice for staying on top of key changes this year.
Engage with regulatory bodies: Early and frequent engagement with regulatory bodies can help organizations understand their expectations and get guidance on complex issues.
Focus on patient involvement: Organizations should look to engage with patients and incorporate their input into the development process.
Increase diversity in clinical trials: Regulatory bodies want to see more diversity in clinical trials, so organizations should look to cast a wider net when recruiting.
Invest in digital capabilities: With the increasing digital disruption in drug development, investing in digital capabilities, such as data management, analytics and digital health technologies, can help organizations stay competitive.
Build robust data management systems: With the new NIH Data Management and Sharing Policy in the U.S., organizations should ensure they have robust data management systems in place.
Regulators continue to hammer out rules governing decentralized trials. DCT recommendations by the FDA and EMA sparked a global trend, spurring similar measures from China’s National Medical Products Administration (NMPA), Taiwan’s FDA and Argentina’s National Administration of Drugs, Food and Medical Devices (ANMAT). China has emerged as a significant player in clinical development, embracing new digital approaches such as electronic signatures and electronic informed consent. However, challenges remain around data residency and privacy, Khera said.
Last January, the NIH issued an updated policy mandating that companies adopt a Data Management and Sharing (DMS) framework for research linked to various initiatives.
“Therefore, we should be on the lookout for next steps related to DMS framework development over the coming months,” Khera said.
There have also been significant developments in digital health regulation, including the Digital Health Technologies for Remote Data Acquisition in Clinical Investigations final guidance and a growing acceptance of self-reported outcomes for primary endpoints.
“The FDA’s embrace of digital health is changing its clinical trial oversight to advance novel methodologies that improve access and convenience,” Khera said.
Improving trial efficiency
Several regulators are also considering measures to improve trial speed and efficiency. The FDA, for example, has moved to bring clinical research regulations in line with the HHS’ Common Rule to streamline research processes. One proposed measure seeks to reduce the administrative burden for clinical investigators and Institutional Review Boards (IRBs) by requiring a single IRB for clinical studies with multiple institutions, potentially accelerating the pace of research without compromising patient safety, Khera said.
Another proposed rule by the FDA aims to enhance the informed consent process for people considering participating in clinical trials.
In the EU, the Clinical Trials Regulation (CTR) went into effect in 2022 and aimed to harmonize the submission, assessment and supervision processes for clinical trials. It established a Clinical Trials Information System (CTIS) to provide a single entry point for sponsors and regulators of clinical trials to submit and assess clinical trial data.
“With CTIS, sponsors can apply for authorizations in up to 30 EU and European Economic Area countries simultaneously and with the same documentation, which may strengthen Europe’s position as an attractive location for clinical research,” Khera said.
In October 2023, EMA also made a move to improve the transparency of information submitted through the CTIS system.
“One of the key changes is the removal of the deferral mechanism, which allowed sponsors to delay the publication of certain data and documents for up to seven years after the end of a trial to protect personal data and commercially confidential information,” Khera said.
The revised transparency rules will apply after their technical implementation in CTIS and the EMA will likely finalize them in the second quarter of 2024.
“In Europe, we should keep our eyes on an ongoing regulatory challenge centered around the Accelerating Clinical Trials in the EU multi-year work plan, extending through 2026,” Khera said.
In the U.S. watch for follow-up on an updated policy issued by NIH last January, which called for the adoption of a DMS framework for research linked to various initiatives.
A continued push for diverse and patient-centric trials
In 2024, regulators will continue the push for trials that include more patient input and represent a broad spectrum of the population and there will be movement on the FDA’s guidance on diversity action plans in clinical trials.
“It’s worth noting that the draft guidance invited many comments,” Khera said. “In 2024, the FDA will have to publish an aggregate report of the action plans annually, along with the reasons any trials fell short of the goals. We expect regulatory agencies to acknowledge the FDA’s guidance in 2024 and issue reflections to ensure patient diversity and inclusion.”