EMA Wants Stakeholder Input On Regulatory Challenges To 2025
Executive Summary
The EMA has launched a six-month consultation on the challenges facing the agency and the wider EU regulatory network in the face of emerging scientific and technological advances, and has put forward a range of recommendations for action in the period to 2025 to ensure the regulatory system is fit for purpose.
The European Medicines Agency has launched a consultation on whether the EU regulatory network is prepared for emerging scientific and technological innovations such as big data, precision medicines, innovative clinical trial design, and novel manufacturing technologies.
According to the EMA, the consultation is “an opportunity for regulators and our stakeholders jointly to shape a vision for regulatory science that will in turn feed into the wider EU network strategy in the period 2020-25.”
The agency’s executive director, Guido Rasi, says that as well as dealing with the disruption brought by Brexit, the EMA has to prepare for the challenges facing the system over the next five to 10 years.
In a “Strategic Reflection” on EMA regulatory science to 2025, which is out for consultation until June 30, Rasi says there is a need for more collaboration to improve innovation and patient access to new medicines, and to keep on top of developments, identify gaps in regulation, and bring together all stakeholders to discuss how to bridge those gaps.
“The strategy includes developments and challenges in medicines development that we together with the [European] Commission and [national competent authority] experts have identified in a thorough process of mapping and selection,” Rasi declares. “Now we want to hear from our stakeholders whether they consider this strategy is ambitious enough.”
The process began in 2016 when the EMA decided it needed to look at how best to allocate limited regulatory network resources to those areas where the impact would be greatest. “This need was made even more acute as a result of the UK referendum on membership of the EU” in June 2016, it says.
A detailed baseline report was commissioned in 2017 looking at the key trends that would affect the ongoing operations of the EMA and the network, and this was followed up by outreach activities with all stakeholders. The results of these activities were incorporated into the reflection document, which also takes account of the outcome of a stakeholder workshop organized by the EMA in October last year.
Five Key Goals
The consultation paper focuses on five key goals, each of them with their own recommendations and supporting actions in various regulatory areas. The five areas are: better integration of science and technology into the development of new drugs; improving collaborative evidence generation and improving the scientific quality of evaluations; working on patient access to medicines in partnership with healthcare systems; addressing emerging health threats and meeting new therapeutic challenges; and better use of research and innovation in regulatory science.
Details relating to each of the areas follow below.
Better integration of science and technology into the development of new drugs. This includes supporting advanced therapy medicinal products (ATMPs), precision medicine, biomarkers and ‘omics, investing more in the PRIME (priority medicines) scheme, and developing the regulatory evaluation of nanotechnology and new materials in pharmaceuticals.
Actions proposed include greater early EMA engagement with developers of novel biomarkers, addressing the impact of emerging ‘omics methods and their use through the product life cycle, and looking at how biomarkers affect clinical outcomes.
The EMA also says that while PRIME has been “broadly successful in bringing forward proposals to speed the development and timely approval of medicines for conditions that have proved difficult, if not impossible, to treat,” it is resource intensive and requires timely access to appropriate expertise. The scheme therefore needs to be explained more clearly to outside stakeholders to allow it to be better understood and further developed, and the time between scientific advice, clinical trials and marketing authorization application (MAA) submission needs to be shortened.
On the advanced therapies front, the EMA says that the low number of applications received so far means more needs to be done to address current challenges and those that will arise from emerging technological advances. It suggests more effort to identify therapies that address unmet medical needs, help in planning and development of ATMPs, and supporting the generation of evidence that will be pertinent to HTA bodies and payers.
This field of action also includes plans to facilitate the use of novel manufacturing technologies such as continuous manufacturing as well as additive manufacturing, which is intended for the production of complex customized therapies. “These techniques offer an opportunity to reduce waste, produce medicines in more flexible and responsive ways and tailor production to specific, even individual, medical needs,” the EMA says. “Their implementation should therefore be facilitated by the regulatory system.”
Improving collaborative evidence generation and improving the scientific quality of evaluations. Key points here are better use of non-clinical models, innovation in clinical trials, improving the use of modeling, simulation and extrapolation, and exploiting digital technology and artificial intelligence (AI) in decision making.
One action here is modernizing regulatory oversight of good clinical practice to allow “decentralized models of clinical trials coupled with direct digital data accrual”, and developing the agency’s ability to assess complex datasets captured by technology such as wearables.
In order to reduce the gap between regulatory approval and HTA/payers’ decisions on new drugs, the EMA suggests expanding the benefit-risk assessment by incorporating patient preferences, and improving communications with HTAs and payers on the therapeutic context surrounding a medicine.
On the AI front, the EMA plans to set up a dedicated “test laboratory” explore the use of innovative digital technology in decision making.
Working on patient access to medicines in partnership with healthcare systems. This will involve working more closely with health technology assessment bodies and payers, greater use of high-quality real-world data in decision making, and promoting the availability and uptake of biosimilars by national healthcare systems.
Among proposals put forward in the paper are ensuring evidence needed by HTAs ad payers is incorporated early in drug development plans, exploring additional methodologies to gather and use patient data from the wider patient community during benefit-risk evaluation, increasing patients’ participation in the EMA’s scientific committees, and working with specialists on how big data should be gathered, presented and analyzed.
On the biosimilars front, the EMA says that a lack of understanding of the nature and regulation of biosimilars “has been identified as contributing to distrust in their use,” but that it has been working with healthcare professionals and patients to better explain the scientific and regulatory aspects. To continue this effort, it plans more communication campaigns and more training of non-EU regulators in the evaluation of biosimilars, and will also look at the regulatory challenges in manufacturing – for example aspects of the comparability exercise and the “evolution of multisource biologicals/biosimilars.”
Addressing emerging health threats and meeting new therapeutic challenges. Under this heading the EMA says it plans to ring-fence resources and for health threats, support the development of new antibacterial agents, tackle supply issues on a cooperative basis at the global level, and help to find new innovative approaches to developing, approving and monitoring the safety of vaccines.
Better use of research and innovation in regulatory science. The EMA proposes to develop network-led fundamental research partnerships with academia, and to identify and disseminate the best expertise across Europe and more widely. It says this is “the key to delivering the other strategic goals and recommendations laid out in the proposals.”
Among the ideas in this area are to work with academia to identify fundamental research topics in strategic regulatory areas like ‘omics-based diagnostics, drug-device combinations, modeling and simulation, big data and AI. The EMA will also ring-fence funding for rapidly emerging regulatory science research questions such as diagnostics, precision medicine, distributed manufacturing and drug repurposing.
Once the consultation is over, a “consolidation workshop” will be held in the fourth quarter of 2019, after which the regulatory science strategy will be published.
From the editors of Scrip Regulatory Affairs.