It is a challenge as old as lawmaking: how do legislators write rules today that can keep pace with the innovations of tomorrow?
The problem is particularly acute in fast-moving sectors where scientific advances arrive at breakneck speed. Artificial intelligence (AI) and big data, wearables and genomics — these are among the most disruptive and unpredictable fields in modern science. And they are among several cutting-edge trends that converge in medical research.
As the EU embarks on a once-in-a-generation overhaul of pharmaceutical legislation, it is vital that we design rules with the flexibility that the future will demand.
While this inspires opportunities to detect and treat illness in entirely new ways, managing the pace of scientific progress can be a headache for legislators, regulators and innovators.
As the EU embarks on a once-in-a-generation overhaul of pharmaceutical legislation, it is vital that we design rules with the flexibility that the future will demand. We must keep patient safety and quality at the heart of all that we do, while acknowledging the costs to patients that would arise if innovation were slowed or discouraged by regulatory rigidity.
This can be done. How? By legislating for innovative sectors with the same spirit of creativity required to solve the complex problems facing patients and health care systems.
When drugs meet digital
Before getting into the details of what flexibility looks like, we must be clear what we are talking about. Let’s consider the kinds of product that regulators will look at in the years ahead: combinations of drug, device and digital technologies.
Imagine a product comprising a matrix of cells on a scaffold mesh. The cells are designed to produce a protein that has a therapeutic effect. The cells and scaffold mesh are to be implanted into the patient’s body to treat an illness.
However, to ensure the patient receives the dose of protein that is right for them, the production of the protein from the cells is controlled by an AI system attached to the scaffold mesh. This AI tells the cells to produce more or less protein, depending on the patient’s need.
Let’s consider the kinds of product that regulators will look at in the years ahead: combinations of drug, device and digital technologies.
The question is, how this product is regulated and by whom? The answer now is complicated because multiple pieces of EU legislation would apply. The scaffold mesh would be considered a medical device and subject to the requirements of medical device legislation.
The cells would potentially be subject to EU legislation covering Substances of Human Origin and Genetically Modified Organisms and or the Advanced Therapy Medicinal Product Regulation. The protein produced by the cells would be a medicinal product and therefore overseen by the pharmaceutical legislation. And the AI component would be covered by both the medical device legislation and forthcoming AI legislation.
You can see the problem.
It should be stressed that while the example above is not on the market, it is not science fiction. These are the kinds of combination products innovative companies are exploring in their tireless efforts to improve lives. Any organization taking on this challenge faces multiple regulatory hurdles in the hope of reaching patients.
In addition, there are novel products and combinations that we cannot even conceive of today, which may rewrite medical textbooks in the coming decades.
Enter the regulatory sandbox
In the above example, several sets of strictly-defined requirements would apply to a single product. These rules have a common aim: to ensure that high quality, safe and efficacious products are made available to those who need them. Each piece of legislation brings different expectations of evidence, which can make it technically difficult and prohibitively expensive to develop a product.
Looking across various legislations and identifying what is needed, known as the regulatory sandbox, optimizes the chances of bringing true innovation to patients.
Flexibility is the answer. Medicines regulators need the option to look across the various legislations and, working with the developer, identify what evidentiary requirements must be met. In some instances, a more bespoke development approach could be taken.
This approach of looking across various legislations and identifying what is needed, known as the regulatory sandbox, optimizes the chances of bringing true innovation to patients in a timely manner, without compromising on quality, safety or efficacy.
The case for phased review
Legislators have an opportunity to get acquainted with new techniques and innovative solutions when innovators share data with regulators on products in the research pipeline. Today, a product’s benefit-risk profile is assessed at the end of the development process based on a dossier containing all the data generated during the years-long development process.
But why wait until the end to start the assessment if some data is ready for review earlier in the process?
Companies and regulators should engage early and often in the development process. This gives regulators a chance to become familiar with cutting-edge products and helps companies to ensure they are collecting the right data at every step.
This serves patients by making for a more efficient and faster assessment without any compromise on safety.
For regulators, this would give opportunities to ask questions or request data; for companies, less uncertainty about whether they are generating the kinds of evidence regulators expect. It also creates space for incorporating patient perspectives and feedback from member countries authorities well in advance of any decision to authorize a product.
Ultimately, this serves patients by making for a more efficient and faster assessment without any compromise on safety. This approach has already proven its worth in the phased review of COVID-19 vaccines and treatments. Now is the time to embrace such an effective regulatory tool in normal times — not merely at a moment of crisis.
The bigger picture
Unless this kind of agility is built into the new EU General Pharmaceutical Legislation, there is a real risk of the new framework not being adapted to the science to come and as a result, of not being able to ensure European patients benefit from the upcoming scientific, technological and digital advances. This would have a knock-on effect on Europe’s position in the fiercely-competitive global life sciences sector, too.
It cannot be forgotten that this conversation is taking place against the backdrop of declining research and development investment in the EU, with the U.S., China and others pulling ahead. Europe needs a cutting-edge regulatory system for today and tomorrow’s innovations, coupled with an ecosystem driven by science and a restless drive to fill therapeutic gaps.
We cannot know where tomorrow’s game-changing innovations will come from, but we can design rules that support the safety, quality and efficacy of medicines, and enable science and progress.
