BIA/MHRA Regulatory Innovation Conference
Hannah Lewis, Consultant at Scendea
Introduction
In July 2021, MHRA published their two-year roadmap: ‘Medicines and Healthcare products Regulatory Agency Delivery Plan 2021-2023; Putting patients first: A new era for our agency’, within which scientific innovation, healthcare access and patient safety were called out as top priorities.
Following the exit of the UK from the European Union, the passing of the Medicines and Medical Devices Act (2021), and the challenges and response to the COVID-19 pandemic over the last two years, the Agency has taken a proactive approach in establishing itself as a sovereign regulator and intends to maintain this momentum in delivering this vision for the life sciences.
The joint annual BIA/MHRA Regulatory Innovation Conference 2021 hosted a series of panels, drawing from a wealth of expertise and experience from regulators and industry and patient representatives. The themes, reflecting the Agency’s objectives as set out in the two-year roadmap, were as follows:
Transforming the regulatory environment to drive innovation and enable patient access to cutting-edge medicines
New innovative technology transforming product development
The first session focussed on the multifaceted approach taken by MHRA and affiliate bodies to address these priorities.
Transforming the regulatory environment to drive innovation and enable patient access to cutting‑edge medicines.
A key aspect of the MHRA roadmap is the overhaul of the clinical trials system to support innovation and reduce time to approval. CPRD SPRINT (Speedy Patient Recruitment INto Trials) and the decentralisation of clinical trials are two such methods which aim to accelerate recruitment and deliver clinical trials in a novel, more efficient, manner. SPRINT has been developed to provide an anonymised UK-wide database from GPs (currently covering 25% of the UK population), from which patients can be located through near real-time centralised searches of UK-wide electronic health records against protocol criteria for Phase 2-4 clinical trials. The service provides increased opportunities for patients to participate in trials regardless of location and enables GPs to approach patients that are most suitable to take part in a trial, and who they believe will benefit from participation. Risk-adapted approaches and decentralised clinical trial designs additionally offer the potential to facilitate trial conduct and improve patient engagement and experience.
The aims of the MHRA’s flagship scheme, the Innovative Licensing and Access Pathway (ILAP), are representative of the Agency’s overarching strategy, with the objective to provide a streamlined approach to regulation and patient access. Importantly, meaningful patient engagement is intended for every stage; patients are involved in the selection process and are also included in outcome discussions. Patient feedback during the conference emphasised the need for Sponsors to consider patient use early in development, and highlighted that patients should been seen as partners during the development process. To date, 67 applications have been received and 35 Innovation Passports have been awarded. Feedback from the BIA on the use of the TDP (target development profile) cited very high levels of engagement from the attending parties. The TDP assesses the potential challenges and can cover special populations, evidence generation, patient engagement (including future plans), the use of patient reported outcomes, and the product lifecycle (including future indications and repurposing). The discussion highlighted that Health Technology Assessment (HTA) reimbursement should consider how well prepared the healthcare system is for the proposed technology.
Thirdly, the transformation of the regulatory environment should consider the precedent set by the development of the COVID-19 vaccines, and in particular the opportunities for real-time delivery of pharmacovigilance at scale and pace. The pandemic highlighted the need for a tailored UK vigilance strategy for vaccinations, and increased the acceptability for data sharing, to identify signals in real time. The implementation of more responsive systems will include the initiation of the SafetyConnect system, which will be live from April 2022. Furthermore, as the role of genetics in adverse drug and vaccine reactions is not yet sufficiently understood, MHRA are scoping the potential for a YellowCard Biobank as a resource for patient safety. This would comprise the collection and sequencing of DNA, primarily focussing on a priority list of research topics to establish genetic predisposition of individuals to certain side effects.
New innovative technology transforming product development.
The second session focussed on the ongoing transformation of the development of medicinal products and devices, with a key focus of the impact of artificial intelligence (AI). It is expected that the coming years will see a profound increase in the use of AI and machine learning (ML) across the pharmaceutical, biotech and medical device arena. Anticipated applications will include AI for knowledge discovery (e.g., drug repurposing), clinical trials (in silico trials), the product package itself (pharmacogenomics, companion diagnostics, and tailoring products or doses for patients), and for pharmacovigilance. Case studies were presented on the use of data modelling to identify predictive factors for patient dropout in clinical trials for Alzheimer’s disease, and the potential use of AI in tailoring medications to treat hypertension.
Although the use of AI has the potential to have an extraordinary impact across product development, a cautionary approach is urged, particularly regarding the ethics of AI stratification. With this in mind, the MHRA, US FDA and Health Canada have jointly identified ten guiding principles to inform the development of Good Machine Learning Practice (GMLP), with the intention of promoting safe, effective, and high-quality medical devices that use AI/ML. Sponsors intending to incorporate AI into product development are advised to approach the Agency as early as feasible to initiate such discussions.
MHRA Key Messages.
In summary, the prospective focus for MHRA in the coming years will centre on:
The involvement of patients in every aspect of product development, building on the concept that ‘patients = partners’.
Driving innovation, with the role of MHRA to be not only of a ‘watchdog’, but an enabler to support product development.
Safe and early access to new innovative treatments, with a key role for ILAP.
Transformation of regulation and technologies. An increasingly important consideration across the industry is the integration and use of AI/ML in the product lifecycle, and MHRA have confirmed a proactive approach towards this change.