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Bridging the innovation-regulation gap

Two scientists in protective gear, masks, goggles, and green gloves, examine a pink substance in a petri dish, conveying focus and teamwork.

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We're in the year 2038. The European Union’s General Pharmaceutical Legislation entered into force almost 10 years ago. For the 3rd consecutive year, 7 of the 10 most innovative products in BioWired’s BioHealth Impact ranking* emerged from companies tapping into Europe’s scientific and data science excellence.

* Illustrative only

Europe is still not the largest single biopharma market but it leads the way in the development, testing and embedding of healthcare innovation into clinical practice. The innovation ecosystem in Europe has become adept at fostering the development of quantum leap products, balancing an openness to significant innovations on the one hand with the regulatory obligation to ensure product quality, safety, and efficacy on the other.

It was different back in 2023. At the time, Europe was losing ground to other large mature and developing markets. For established companies, the fragmented nature of its regulatory system was complex to navigate, and daunting for SMEs and start-ups.  It also inhibited vibrant innovation. This resulted in a deterioration of Europe's attractiveness for pioneering science and clinical trials. Companies chose other locations for the translation of research advances into clinical studies and approved, applied solutions for patients. This, despite the priority of public health to European values and the strategic importance of a robust ecosystem and health union to society at large.

How did the European regulatory system turn around its laggardly reputation for the regulation of medicines and related healthcare products? As in science, so also in regulation. Through experimentation. By having the foresight to adopt a regulatory sandbox approach to figure out how best to regulate the truly novel.

There were multiple drivers to the needed change: Fast advancing, new and disruptive technologies as well as the blurring of boundaries between interdependent dimensions of care.

Multiple triggers leading to the adoption of the sandbox approach:

  • Regulatory challenges at the interplay of drug, medical devices and in vitro diagnostics due to healthcare solutions drawing on multiple disciplines

  • Fewer clinical trials conducted in Europe with risks to the knowhow of the investigator community and consequently to the access of patients to innovations addressing unmet needs

  • Artificial Intelligence (AI), accelerating at an unprecedented pace, affecting not only R&D processes but also the nature of medical solutions offered to healthcare professionals and patients

  • The EMA’s own horizon-scanning report identified upcoming trends in innovations that would challenge the regulatory system1

Decision makers in Europe realised that the modernisation of the regulatory framework called  for it to be inherently able to anticipate future shifts in technology, in order to enable innovation uptake, while ensuring the safety and efficacy of the future’s incoming novel, medical technologies. The regulatory sandbox was the missing mechanism in the toolbox of regulators

A regulatory sandbox provides the means to clarify and calibrate


As of 2038, the sandbox approach is used more systematically, in conjunction with formalised horizon scanning and consultation mechanisms, to define which innovation requires the setting up of such a sandbox and which ones can be assessed via other means. The sandbox mechanism helps to clarify the pathway where none exists and to expedite regulatory decision making in the face of multidisciplinary complexity. 

By initiating a sandbox approach for truly novel innovations Europe worked around the  "pacing problem", the lag between the advent of those innovations and clarity on how to regulate them in a timely manner and fit-for-purpose way.  Through sandbox experimentation, it reinforced both its regulatory science and fostered the uptake of those innovations as they arose. 

The framework provided a means of reaching certainty, and with that certainty, developers were enabled to advance investment and progress their plans with confidence.  The accommodation within a regulatory sandbox of the weave of multiple disciplines and technologies has been decisive and a boost to the dynamism of healthcare startups in Europe. It has added credence to the sense of an openness in Europe to healthcare innovation leadership. 

The sandbox mechanism helps to clarify the pathway where none exists and to expedite regulatory decision making in the face of multidisciplinary complexity.

What if the sandbox mechanism had not been introduced or been of too narrow a scope? Europe may have closed out a great deal of innovation potential, and healthcare systems and patients might have missed from their armamentarium the eventual products/ solutions from the following blue sky case opportunities: 

Illustrative examples of the regulatory challenges ahead

A regulatory framework during which it is possible to develop, validate and test in a controlled environment innovative or adapted regulatory solutions that facilitate the development and authorisation of innovative medicinal solutions, pursuant to a specific plan and for a limited time under regulatory supervision. The approach has been implemented in other geographies3 in fintech, energy and education sectors among others. The concept is getting traction globally (OECD4, World Economic Forum, United Nations Organization) as an enabler to foster innovation uptake.

Nothing defines a bottleneck as clearly as the past years’ experience with implementation of IVDR (In-Vitro Diagnostics Regulation). That this has been unduly challenging is widely understood and even acknowledged by companies and regulators alike. There have been many pain points emerging with its implementation, ranging from a lack of coordination across the various supervising regulators to difficulties for Notified Bodies5 in absorbing the workload that came with further requirements6. It has caused erosion in the attractivity of Europe as a clinical trial destination and has delayed the start of several studies. 

What if a regulatory sandbox had already been in place in the EU? A sandbox approach could have been useful to anticipate the concrete implications of the emergent changes in the field of clinical trials with drug and devices/IVDs, and take measures to inform and adjust the regulatory provisions, as needed, based on evidence. 

Future proofing the regulatory system means equipping it with mechanisms to better anticipate, adapt and accompany the timely uptake of innovation.

Looking ahead, technology will drive the rise of more and more complex solutions, drawing on several frameworks (including new ones such as AI, SOHO) and will face further complexity at interfaces. It will take multi-disciplinary regulatory support and oversight, and mechanisms that can anticipate such developments, to resolve the open questions on how best to regulate such future products and solutions. 

As illustrated above, innovation is increasingly arising from inter-disciplinary approaches. It can take a very long time to formalize into regulation the changes brought about by advances in science and technology. And such formalization is an essential condition of a dynamic environment that stimulates and enables the uptake of related innovations. On one hand developers need regulatory certainty on the path to follow to secure authorisation. On the other hand Regulators need to calibrate what’s right in terms of regulatory oversight of emerging, novel clinical products. And it’s important to get it right, so that even for the most experimental medicinal solution, the principles of quality, safety, and efficacy, as well as of benefit risk assessments, continue to guide regulatory decisions. 

The essential multidisciplinary nature of incoming waves of innovation makes it imperative that the regulatory sandbox mechanism extends beyond pharmaceuticals.

If we peer only a little into the future, we can see several trends that may require regulatory adaptation, including gene editing platform medicine, multi-intended computational tools, the rise of synthetic data, AI/ML, and the Internet of Medical Things (IoMT). Chat GPT has surprised society and AI applications are exploding. What will surprise us 10 years from now? Synthetic biology? Implantable drug factories? Digital twins?

Future proofing the regulatory system means equipping it with mechanisms to better anticipate, adapt and accompany the timely uptake of innovation. Today the provisions allowing the use of regulatory sandboxes are being debated in the context of the EU’s revision of the General Pharmaceutical Legislation. The essential multidisciplinary nature of incoming waves of innovation makes it imperative that the regulatory sandbox mechanism extends beyond pharmaceuticals.

Decorative illustration
Health horizons: Frontiers in Medicine journal, December 2022.

References

  1. Health horizons:Frontiers in Medicine journal, December 2022.

  2. Pullen LC. Non-transplantable organs and tissues: a golden opportunity. Am. J. Transplant. 22(9), 2127–2128 (2022)

  3. Notified bodies are government accredited, mostly private companies, that take over sovereign tasks on behalf of national authorities. Related article: