Industry Insight

Fiona Maini from Medidata Solutions, speaks about pharmaceutical regulation and the opportunities and policy challenges posed by digitalisation and a rapidly evolving industry

The past few decades have seen science and technology accelerate at a rapid pace, bringing greater efficiency and quality across all aspects of our lives. The pharmaceutical industry is no different, benefiting massively from innovative approaches. But one area in which this rapid digitalisation has posed a problem is regulation, with policymakers struggling to keep up. Regulators are right to carefully consider and evaluate policy, but this has historically made it impossible to keep pace with such rapid change in the industry.

To put things into context, the pharmaceutical industry is still working to International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP) guidelines, which, while updated in 2016, were first introduced in 1996. Technological capabilities have taken off exponentially since then, but some of the benefits have been constrained – and some of the dangers have been exposed – by outdated regulation. This pacing problem is not unique to the life sciences industry, but recent moves by global regulators to address some of these discrepancies are most welcome; in terms of innovation, we are only as good as our regulatory framework. The US and EU, for instance, are bringing in cyber laws requiring companies to highlight IT vulnerabilities in medical devices before they go to market. To the policymakers of the 1990s, the idea that a medical device could be hacked would have sounded crazy, but this is now a potential problem in devices such as pacemakers. This is the technological labyrinth in which regulators are operating and having to react to.

In recent years there has been a flurry of regulatory updates in the US, EU and UK, in a bid to better reflect the realities and considerations of the modern pharmaceutical industry – and clinical trials in particular. Cyber-security, the use of artificial intelligence (AI) in the field and the importance of championing diversity and inclusion in medical development are all points absent from the regulation of the 1990s and which global regulators are now addressing.

Remote technology solutions for clinical trials, such as electronic patient diaries, wearables and sensors have existed for some time, but COVID-19 accelerated the move towards the wider adoption of decentralised clinical trials (DCTs). This is a positive development that supports the democratisation of trial access, enriching data sets and ultimately facilitating scientific discovery. DCTs are, however, an area with a particular need for regulatory revamp as the ICH was created when this technology didn’t even exist.

As a consequence, while COVID-19 helped to drive adoption of decentralisation, many pharmaceutical companies and CROs in the industry have found that the lack of guidance from regulators has prevented them from fully embracing the benefits of DCTs. Thankfully, regulation is modernising. While the details are yet to be confirmed, the EU’s GCP revision 3 will hopefully bring a more adaptive regulatory approach to DCTs later in the year. Similarly, the US Food and Drug Administration (FDA) has recently released guidance for ‘Decentralized Clinical Trials for Drugs, Biological Products, and Devices’. While there are some limitations, the supportive attitude from the agency to facilitate a more flexible approach to clinical trials can hopefully unlock the benefits for trial sites and patients alike.

The promise that AI offers the life sciences sector is colossal, the benefits of which are already being seen in clinical trials. For example, vast amounts of data are generated from clinical trials and AI can be used to forecast potential issues, optimise trial design or even create a synthetic control arm. While the industry is harnessing the power of AI, there are still some concerns around safety, ethics and privacy, which could be assuaged with robust regulatory frameworks.

The AI Act Europe will be the first global regulation of AI, heavily influenced by GDPR and covering all industries. The regulation takes a risk-based approach, with ethical considerations at the forefront of the industry. The life sciences industry is deemed to be high risk given the potential impact on people’s lives. This may be restrictive in some cases, however it is likely to provide some much-needed guidance and reassurance. Many companies will need to look at the act and consider what activities they conduct using AI before seeking certification that they are appropriately compliant.

Although the regulation should lay a good foundation from which to operate, there are some elements that will need modifying. For example, there are potential duplications between the EU Medical Device Regulation and the AI Act, both of which require companies to perform a conformity assessment. Life sciences companies using AI might need to undertake two conformity assessments illustrating the same thing, expending unnecessary time – although this is currently under discussion. Regulationwillalsoaddcoststolifesciencesbusinesses using AI, which could arguably deter investment in the technology. For the sake of quality control and ensuring companies know where they stand, the regulation is a big step in the right direction – but we must hope that it does not stifle innovation. Trying to regulate such a rapidly evolving technology is always going to be difficult – which is why policymakers must collaborate with industry to resolve these teething issues and ensure the regulatory frameworks encourage the use of AI. This technology has the capability to vastly change the life sciences industry as we know it and provide solutions to society’s toughest medical problems.

There is also massive potential to leverage the power of real-world data (RWD) in clinical trials, however this is not an easy task. Using RWD alongside clinical data requires a strong technology platform that can process the data and deliver it in a way that can have a meaningful impact. But in principle, global regulators seem to be supportive of this approach and are implementing frameworks to support the use of RWD in clinical trials, unlocking the potential of this groundbreaking technology.

Currently, the industry is seeing significant guidance emerge. For example, in October 2022 the FDA launched the Advancing Real-World Evidence Program, facilitating agency advice before protocol development and study initiation. Similarly, the European Medicines Agency (EMA) and the European Medicines Network are in the process of establishing a Data Analysis and Real World Interrogation Network, known as Darwin EU. The project supports regulation by looking to deliver real-world evidence from across Europe on diseases, populations and medicines. It is again through support from regulatory authorities that the industry can properly harness this technology; regulation and policy within the EU and US appear to be catching up with developments in the field.

Diversity and Inclusion (D&I) are at last being meaningfully considered in pharmaceutical regulation in the US and EU, with the FDA Reauthorization Act of 2017 requiring companies to have D&I plans for the development of their drugs and medical devices. The EMA is also placing increased emphasis on diversity. D&I is a cornerstone of patient engagement that will enrich the quality of data and research available. Looking forward, it is a matter that is likely to be further prioritised.

The revamped ICH guidance also points to patient engagement more widely and the importance of ensuring participants have positive views of their clinical trial experiences. The UK’s Medicines and Healthcare Products Regulatory Authority (MHRA) Patient Engagement Programme also underscores it. An important part of this is making sure that patients are engaged throughout the life cycle of a trial, from the planning phase until beyond the readout, meaning they feel involved and as though their participation made a real difference. Patient engagement can also be enhanced with the power of DCTs, which is especially helpful when attempting to access patients in traditionally hard-to-get geographies or in rare diseases where patients are disparate. While some of these aspects are not covered by regulation, we are seeing increased guidance from regulatory authorities as they appear to be increasingly recognising the need to engage with patients during clinical trials.

The continued digitisation of clinical trials is inevitable and regulation and policy must continue to evolve and keep pace to reflect this. Looking forward, training the next generation and upskilling the population is a must. As we become increasingly reliant on this technology we must ensure we have skilled data scientists who understand the algorithms and regulators who are willing to embrace change and evolve regulatory frameworks.

The speed at which technology advances will always outpace regulatory change, but while there will always be an element of playing catch up, the key is ensuring that this gap is not too wide. If regulators can continue to modernise and become more nimble, they will play a crucial role in guiding the pharmaceutical industry towards fully harnessing digitalisation.