Regulators aren’t exactly known for their speed. While companies — and drugmakers, in particular, in recent years — quickly pivot to meet emerging needs, regulatory agencies are often seen as being perpetually stuck in a game of “catch up.” But that doesn’t mean that the landscape of regulations can’t quickly shift under the feet of an industry.
As the global head of regulatory strategy at Worldwide Clinical Trials, Aman Khera’s job is to stay on top of these changes — from gathering “hard intel” on new guidelines, to evaluating “soft intel” on the thought-leadership trends that could inform the next wave of regulations. And as agencies such as the FDA and EMA continue to respond to innovations in pharma, notable shifts in 2022 are on the way.
To help the industry prepare, Khera provides a look at five trends that will impact how regulatory agencies work to keep pace with pharma in the coming year.
Digital health: “There is a lot of talk about digital transformation and digital health,” Khera says. “That voice is only going to get louder.”
While digitization tools continue to permeate throughout the industry, Khera says that agencies have begun to offer more guidance on how they should best be used. In June, for example, the World Health Organization (WHO) released new guidance on the ethics and governance of AI in health care. According to the WHO, the guidance was the result of 18 months of deliberation between experts in digital technology, health care, law and human rights, and that it “contains a set of recommendations that can ensure the governance of artificial intelligence for health maximizes the promise of the technology and holds all stakeholders — in the public and private sector — accountable and responsive to the health care workers who will rely on these technologies.”
Another emerging issue relates to how health data is stored and used.
“If you’re asking people to download an app and monitor their health…[there are questions around] the ethics of what data you’re collecting and what constitutes data,” Khera says.
In December, the FDA released draft guidance for industry, investigators and other stakeholders related to the use of digital health technology (DHT) for remote data acquisition. The guidance, which is open for industry comment until March 23, provides a number of recommendations for selecting DHTs for trials, verifying and validating them, using them to collect data for endpoints, and identifying and managing their risks.
“The FDA is trying to react to [these technologies],” Khera says. “[When it comes to digital health], agencies are going to have to pivot.”
Trust and transparency: The pandemic thrust pharma into the public spotlight to a new degree. With more eyes on the industry, Khera says that improving trust and transparency are growing priorities for regulators. To that end, drugmakers are facing new requirements in the EU to publish information about clinical trials in a system that will be more accessible to the public.
“In Europe they’ve been thinking about transparency for a long time,” Khera explains.
In fact, the EMA recently took several steps to finish readying its new Clinical Trial Information System (CTIS), which is slated to launch in January 2022. The reporting system will help harmonize clinical trial information between EU states and provide a single entry-point for monitoring and assessing updates. But it will also boost transparency by making the updates publicly available (aside from information protected by EU regulations).
The FDA has yet to mandate this kind of reporting system in the U.S., and Khera says that the agency has only recently begun to enforce misdemeanors on its reporting website, clinicaltrials.gov. But she says the agency is taking a closer look at ways it can help boost public confidence.
“I know the FDA has been talking about transparency, but I feel there is still more work to do,” Khera says. “From a bird’s eye view, they realize the public is demanding more.”
Partnerships: Making strange bedfellows was a key hallmark of the pandemic as pharma competitors jumped into commercial partnerships to more quickly develop and manufacture COVID-19 vaccines and treatments. The spirit of collaboration is also impacting how regulators work with the industry.
“The whole idea of keeping to ourselves…I think that’s disappearing,” Khera says.
According to Khera, the FDA recognizes that it doesn’t have expertise in every area of pharma. To fill the gaps, the agency has been hooking up with more private partners to boost its regulatory know-how.
In late 2020, for example, the FDA launched a new Digital Health Center for Excellence (DHCoE), which is aimed at driving digital health innovations through industry partnerships and by connecting digital stakeholders. Going forward, the emphasis of the DHCoE will shift towards updating and implementing regulatory frameworks for digital health.
“Regulators are saying that they might not be the experts, but ‘Show us,’” Khera says. “So there are more partnerships than ever before.”
Patient experience: Although “patient-centricity” has been a buzzword in pharma for a few years, Khera says that the phrase “patient experience” is a better fit for the focus of regulatory agencies.
In particular, Khera says that agencies could consider adding more endpoints to clinical trials related to the patient’s experience. Rather than only measuring physical reactions to drugs, agencies might ask for feedback on how a patient felt on the medication, during a clinical trial or how well the pharma company communicated with the patient. In fact, Khera says that a draft of the “Cures 2.0” legislation — which builds on the 21st Century Cures Act — currently includes regulations to help the FDA “modernize” clinical trial design with language focused on improving the patient experience.
“They want to know what feedback [companies] got from the patients,” she explains. “For example, [a medication] might not be as effective as we want it to be…but they want to see what the patient thinks when weighing the risk/benefit of taking it.”
The real world: In addition to monitoring patient experience more closely, Khera says that regulators will be examining ways to measure how medications work in real-world settings.
“What is real-world evidence? What constitutes data that can be used to inform regulators that this product might be effective and safe?” Khera asks. “It’s a huge conversation right now and it’s going to be even bigger next year.”
Previously, Khera says that measuring real-world evidence typically happened in post-approval studies. Going forward, she says that regulators could ask for real-world data earlier in the development cycle.
“New requirements for real-world data could impact trial design and execution, because regulators could ask for that data sooner,” Khera explains.