COVID-19 has propelled the regulatory industry years ahead
Janssen’s Saskia De Haes, VP EMEA Regulatory Affairs, looks at how the pandemic response has shaped tomorrow’s regulatory landscape.
In my 26 years of industry experience, I’ve often found the regulatory process to have plenty of opportunities to become more efficient and faster.
When the COVID-19 pandemic hit, it lit a fire under the regulatory system. There was a genuine sense of alliance; regulatory bodies and pharma companies coming together to accelerate processes and find the most optimal model for collaboration.
This was part of a universal drive to prioritise and streamline, of course. The development of COVID-19 vaccines required levels of cooperation and pioneering science comparable to the Apollo space programme.
Within 12 months of the outbreak, vaccines were being deployed to prevent severe infections, hospitalisation, and death. And, as of September 2021, there are 22 COVID-19 vaccines in use globally , and over 2.3 billion people have been fully vaccinated.
It is a remarkable achievement, tempered only by the sheer numbers of people the vaccines have yet to reach.
So, while there is still so much to be done, the regulatory industry – along with many other areas of the healthcare ecosystem – has been propelled forward.
In the past, it’s taken many years to put meaningful changes in place. During the pandemic, the collective will to expedite regulatory steps was extraordinary.
It’s difficult to gauge how many of these advances can be retained and sustained outside of a global public health emergency scenario, but we must not let the fire burn out.
We have learned so much. We can help achieve a lasting improvement in patient access to potentially life-changing and life-saving therapies, by capturing ways to facilitate swifter review and approvals. And there are many.
Streamlining approval processes
In many parts of the world, the deployment of COVID-19 vaccines during the pandemic was based, at least in part, on their approval in other countries.
In ‘normal’ times, the model is usually individual and sequential. For some countries, it has taken many years to approve a drug that was already available elsewhere, and such delays clearly have a negative impact on patients in those countries.
Also worth exploring is the regulatory oversight and approval process for medical devices and in-vitro diagnostics (IVDs).
In contrast to the evaluation of medicines, the supervisory framework for medical devices and IVDs is fragmented and there is no single ‘go-to’ authority, which can be problematic for some products.
Where we can integrate or streamline processes, we should. Integrated pathways for evaluation and regulatory approval could help reduce delays, in addition to more adaptive regulatory systems that will encourage innovation. It’s encouraging to see this as one of the goals in the EMA’s 2025 Regulatory Sciences Strategy.
We really must do more to reduce access delays and advance health equity, and put better health within reach of everyone, no matter where they live.
Integrating real-world data and real-world evidence
Real-world data (RWD) and real-world evidence (RWE) can be used by a variety of stakeholders to better understand patients’ experience of care and health outcomes, and to help improve the overall efficiency of the healthcare system.
In addition to data obtained in a clinical setting, information from outside of the clinic can contribute to understanding how a medicine works in daily life..
And it’s easier than ever to gather these data. There are new technologies at our disposal, and we have the opportunity to develop patient-centric digital tools that collect data in more real-world settings..
Federated data networks, such as EHDEN or HONEUR, can help unlock the potential of all this information, by harmonising and pseudonymising data to a common model, while ensuring the highest levels of privacy and security..
The key will be in agreeing what’s acceptable. We need to establish appropriate standards for different contexts. RWD and RWE must be scientifically rigorous in addressing the specific evidence needs of each decision-maker at a given decision point.
If we can formalise ways to accept RWD/RWE into the marketing authorisation process, alongside and complementary to clinical trial data, it could support faster approvals of a new treatment. Great strides have been made in this area, but there’s much more to be done.
Accepting digital measures and digital endpoints
The use of digital technologies in clinical research has significantly increased over recent years, and they have the potential to transform the way this research is designed and conducted.
In medicines development, these tools could enable more precisely targeted and differentiated products to come to market. So, a broader acceptance of digital measures and digital endpoints for evidence generation is crucial, as is the continuous collection of data in more real-world settings.
But many questions remain on how best to implement such technologies in clinical trials (particularly when used with the intent to create novel endpoints), and on how to meet the expectations of regulators and HTA bodies.
Development of digital endpoints, and the wider sharing thereof, could be supported by infrastructures such as those set up by the Digital Endpoints Ecosystem & Protocols (DEEP) initiative, an accelerator for the digital measurement ecosystem.
What remains very clear is that collaboration between sponsor companies, health authorities and other stakeholders will be required to move this forward.
Real-time sharing of data
Tackling COVID-19 has reinforced the value of efficient data exchange. Regulators and pharmaceutical companies are investigating cloud-based platforms to enable the real-time sharing of data.
And advances in digitalisation could facilitate a move from document-based submissions to data-based submissions, through a cloud platform like the one being developed by Accumulus Synergy.
Such a platform would allow for real-time data submissions and enable health authorities around the world to interact with innovators and review the data simultaneously.
It would also improve efficiency, allowing pharmaceutical companies to make one submission (rather than redoing the work separately for each regulator), and make information sharing between a sponsor and health authorities more rapid, transparent and collaborative.
Robust data privacy and cyber security measures are, of course, essential.
But with those in place, this novel way of working should lead to improving outcomes for patients by bringing innovative medicines to them more quickly and more efficiently.
We can see glimpses of a different regulatory world. A vision of a more streamlined and fit-for-purpose regulatory system that enables swifter access to new treatments, and – in so doing – helps to profoundly improve the health of people everywhere.
Let’s work together to keep the momentum going and make that world a reality.
This article was originally published in pharmaphorum in October 2021.
The Economist. Ten million reasons to vaccinate the world. Available from: https://www.economist.com/leaders/2021/05/15/ten-million-reasons-to-vacc.... Last accessed: October 2021.
 AJMC. A Timeline of COVID-19 Developments in 2020. Available from: https://www.ajmc.com/view/a-timeline-of-covid19-developments-in-2020. Last accessed: October 2021.
Regulatory Affairs and Professional Society. COVID-19 vaccine tracker. Available from: https://www.raps.org/news-and-articles/news-articles/2020/3/covid-19-vac.... Last accessed: October 2021.
Our World In data. Coronavirus (COVID-19) Vaccinations. Available from: https://ourworldindata.org/covid-vaccinations?country=OWID_WRL. Last accessed: October 2021.
Wileman H, Mishra A. Drug lag and key regulatory barriers in the emerging markets. Perspect Clin Res. 2010;1(2):51-56.
Durán CE, Cañás M, Urtasun MA, et al. Regulatory reliance to approve new medicinal products in Latin American and Caribbean countries. Rev Panam Salud Publica. 2021;45:e10. Published 2021 Apr 9.
Petra Doerr, Marie Valentin, Nobumasa Nakashima, Nick Orphanos, Gustavo Santos, Georgios Balkamos & Agnes Saint-Raymond (2021) Reliance: a smarter way of regulating medical products - The IPRP survey, Expert Review of Clinical Pharmacology, 14:2, 173-177.
EMA. Regulatory science strategy. Available from: https://www.ema.europa.eu/en/about-us/how-we-work/regulatory-science-str.... Last accessed: October 2021.
Beaulieu-Jones BK, Finlayson SG, Yuan W, et al. Examining the Use of Real-World Evidence in the Regulatory Process. Clin Pharmacol Ther. 2020;107(4):843-852.
Khosla, S., Tepie, M.F., Nagy, M.J. et al. The Alignment of Real-World Evidence and Digital Health: Realising the Opportunity. Ther Innov Regul Sci 55, 889–898 (2021).
 EHDEN. Welcome to the European Health Data & Evidence Network (EHDEN), an IMI 2 consortium with 22 partners operating in Europe. Available from: https://www.ehden.eu/. Last accessed: October 2021.
Li M, Chen S, Lai Y, et al. Integrating Real-World Evidence in the Regulatory Decision-Making Process: A Systematic Analysis of Experiences in the US, EU, and China Using a Logic Model. Front Med (Lausanne). 2021;8:669509. Published 2021 May 31.
Inan, O.T., Tenaerts, P., Prindiville, S.A. et al. Digitizing clinical trials. npj Digit. Med. 3, 101 (2020).
Cerreta et al., Digital technologies for medicines: shaping a framework for success, Nature Reviews Drug Discovery 19, 573-574 (2020).
Marra, C., Chen, J.L., Coravos, A. et al. Quantifying the use of connected digital products in clinical research. npj Digit. Med. 3, 50 (2020).
EMA. Questions and answers: Qualification of digital technology-based methodologies to support approval of medicinal products. Available from: https://www.ema.europa.eu/en/documents/other/questions-answers-qualifica.... Last accessed: October 2021.
ISPOR. The Digital Endpoints Ecosystem and Protocols (DEEP) Initiative. Available from: https://www.ispor.org/publications/journals/value-outcomes-spotlight/vos...(deep)-initiative-a-collaborative-multi-stakeholder-approach-to-defining-and-developing-standards-for-digital-endpoints. Last accessed: October 2021.
Accumulus Synergy. Available from: https://www.accumulus.org/wp-content/uploads/2021/06/Accumulus_Synergy_W.... Last accessed: October 2021.