The scenario is that the regulators are all ramping up pressure when it comes to the biopharma industry in 2024. Pharma companies not only happen to need to jump through new hoops when it comes to an increasingly intricate suite of therapeutics such as cell and gene therapies, but they also happen to be navigating technological shifts and a growing demand so as to take a more patient-centric approach in terms of drug development, said the vice president of regulatory science, strategy, and innovation at Worldwide Clinical Trials, Aman Khera.
The federal Inflation Reduction Act, which happens to be aimed at reducing prescription drug costs for Medicare patients as well as trimming federal drug spending, is going to most likely add to those pressures. The regulation could go on to potentially lessen access to Medicare Part B and Part D-covered drugs as well as, at the same time, discourage the development of specific types of drugs, like small molecules.
Khera added that some companies already happen to be rethinking their approach when it comes to R&D, cutting projects, and reallocating resources. There are certain others who are reconsidering whether to give medicines in the U.S., as they may never be able to recoup the costs.
It is well to be noted that these pain points may be in particular acute for smaller and emerging biopharmaceutical companies that happen to have a growing footprint in the sector. The fact is that the number of emerging biopharma companies has gone on to see a jump of 4% every year for the last five years, and these companies, apparently, went on to produce two-thirds of all new drugs in 2022, as per IQVIA. Studies have also suggested that by 2039, IRA provisions could very well result in a 31% depletion in profits and potentially lead to 135 fewer new drug approvals within the same timeline.
Apparently, now, companies are putting their thrust to develop effective medications on an accelerated timeline.
As per Khera, while these two objectives have often appeared at face value to be pretty contradictory, this has happened to be the case in recent times.
It is worth noting that the modality landscape has expanded prominently to not only include traditional small molecules along with proteins but also peptides, a variety of nucleotide-based therapies like antisense oligonucleotides, antibody-drug conjugates, small interfering ribonucleic acids, messenger RNAs, as well as cell and gene therapies, said Khera. This new crop of therapeutics goes on to bring new preclinical as well as drug development requirements, along with regulatory complexities.
She added that such a rise in patient input into all elements of drug development, such as regulatory review, has also gone on to affect medicinal product regulation. Regulators’ anticipation of patient involvement when it comes to drug development is growing, even when the new tools are helping to eliminate practical barriers when it comes to working with patients.
To stay in sync with the regulatory requirements, Khera advises organizations to take a strategic pathway to compliance this year. Let us look at the key trends that are most likely to shape drug development regulations in 2024:
An elevated focus on digital technology
Many regulatory changes that are all set to take effect in 2024 look to clarify rules around technology as well as data collection, such as an update by the International Council for Harmonisation- ICH to its Good Clinical Practice- GCP E6(R3) draft guideline that looks to better define best practices that happen to be related to technology, software, as well as remote elements, Khera opined. The FDA has also taken steps to give organizations better guidance on how to acquire remote data by way of using digital health technologies in its final guidance, Digital Health Technologies when it comes to Remote Data Acquisition in Clinical Investigations.
Notably, the regulators continue to hammer out regulations governing decentralized trials. DCT recommendations by the FDA as well as the EMA sparked a worldwide trend, spurring similar steps 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 gone on to emerge as a prominent player when it comes to clinical development, embracing new digital approaches like electronic signatures as well as electronic informed consent. But challenges still happen to remain around data residency as well as privacy.
It is worth noting that in January 2023, the NIH went ahead and issued an updated policy mandating that companies go on to adopt a Data Management and Sharing- DMS framework for research that’s linked to various initiatives.
Hence, one should be on the lookout for further steps that happen to be related to DMS framework development in the coming months, Khera said.
It is well to be noted that there have also been prominent developments when it comes to digital health regulation, such as the Digital Health Technologies for Remote Data Acquisition in Clinical Investigations final guidance and an acceptance that’s growing when it comes to self-reported outcomes in terms of primary endpoints.
As per Khera, the FDA’s embrace of digital health happens to be changing its clinical trial oversight so as to advance novel methodologies that enhance access and convenience.
Enhancing trial efficiency
There are many regulators who happen to be considering steps so as to enhance trial speed and efficiency. The FDA, for instance, has moved to get clinical research regulations in line with the HHS’ Common Rule in order to streamline research processes. One proposed step looks to lessen the administrative burden in terms of clinical investigators and Institutional Review Boards- IRBs by requiring a single IRB in terms of clinical studies with many institutions, potentially speeding up the pace of research sans compromising patient safety, remarked Khera.
There is another proposed rule by the FDA that looks to elevate the informed consent process for people who happen to be considering participation in clinical trials.
It is well to be noted that in the EU, the Clinical Trials Regulation- CTR went into effect in 2022 and looked to harmonize the submission, assessment, as well as supervision processes when it came to clinical trials. It came up with the Clinical Trials Information System- CTIS so as to offer a single-entry point for sponsors as well as regulators of clinical trials in order to submit and evaluate clinical trial data.
According to Khera, with CTIS, sponsors can go on to apply for authorizations in almost 30 EU as well as European Economic Area countries at the same time and with the same documentation, which may strengthen Europe’s position as a hub location in terms of clinical research.
In October last year, EMA also happened to make a move to enhance the transparency of information that happens to be submitted by way of the CTIS system.
As per Khera, one of the major changes happens to be the removal of the deferral mechanism, which enables the sponsors to postpone the publication of certain data along with documents for almost seven years after the end of a trial in order to safeguard personal data as well as commercially confidential information. The fact is that the revised transparency rules will be applied after their technical execution in CTIS, and the EMA will most likely finalize them in the second quarter of 2024.
Khera adds that in Europe, one should keep a close watch on the ongoing regulatory challenge, which is focused around Accelerating Clinical Trials in the EU multi-year work plan, going through 2026.
When it comes to the U.S., one must watch out for follow-up on an upgraded policy issued by NIH in January 2023, which asked for the adoption of a DMS framework when it comes to the research linked to numerous initiatives.
A consistent push for diverse as well as patient-centric trials
In 2024, regulators will go ahead with the push for trials that have more patient input and go on to represent a broad spectrum of populations, and there will be a push on the FDA’s guidance when it comes to diversity action plans within clinical trials.
Khera finally adds that it is indeed worth noting that the draft guidance invited many feedback and comments. This year, the FDA will have to publish an aggregate report of the action plans yearly along with the reasons why any trials fell short of the goals. One can expect regulatory agencies to take into account the FDA’s guidance in 2024 and go ahead with issuing reflections to make sure of patient diversity as well as inclusion.
