UK to introduce first-of-its-kind framework to make it easier to manufacture innovative medicines at the point of care

In 2021, the MHRA held a public consultation seeking views on a legislative proposal to introduce a new regulatory framework for the manufacture and supply of Point of Care (POC) products. This post is about the formal government response to that consultation.

In a nutshell, the UK will introduce a first-of-its-kind framework to make it easier to manufacture innovative medicines at the point of care

The current human medicines legislation

The current human medicines legislation is geared for products which can be manufactured at a very small number of sites and supplied globally. It is quite likely that this will not be sufficient as POC products may only have a shelf-life of minutes or need to be highly personalised, so have to be manufactured on demand when a patient is present. This could necessitate having to manufacturing a POC product at hundreds of sites across the UK alone.

The three key areas of the consultation proposal

The consultation proposals included three key areas:

  • The requirement for a new regulatory framework: a description of the regulatory impediments hindering or preventing products that are required to be manufactured and supplied at or near the POC.
  • A proposed framework that is tailored for products manufactured at POC: a regulatory framework that allows for manufacture and supply at or near the POC and which has proportionate controls across the different stages of development and manufacture to retain equivalent levels of safety, quality and efficacy currently in place for more conventional medicinal products
  • The scope of manufacturing and supply scenarios: a range of scenarios were presented, ranging from those similar to current factory‑based manufacture termed modular manufacture, through mobile manufacture, POC manufacture and extending to home‑based manufacture.

Comments/views on the consultation

Comments and views were expressed from a variety of stakeholders from across the UK and internationally.

  • There was significant support for the creation of a new UK regulatory framework for manufacture and supply at the POC.
  • Responders also supported a wider scope of manufacturing and supply, for the framework to also include modular manufacture and home‑based manufacture. Based on this feedback, we will prepare a statutory instrument to introduce the new framework. Future guidance will be developed alongside the statutory instrument to provide the necessary interpretation and procedural support.

Next steps based on the consultation feedback

In order to introduce and implement the new regulatory framework, new legislation is being developed to amend the UK’s Human Medicines and Clinical Trials legislation and will be brought to Parliament later this year. The MHRA will also begin to develop guidance, in conjunction with stakeholders, to accompany the new framework, which will be issued in due course.

The UK will be the first country to introduce a tailored framework for the regulation of innovative products manufactured at the point where a patient receives care, as announced by the MHRA today.

What is the objective of the proposed new regulatory framework?

The objective is to provide an enabling framework to support increased manufacturing and supply of innovative medicines that can only be manufactured at the point of care.

An enabling framework is particularly important in this area where there are rapid and continued technological developments. This will, in turn, improve the availability of novel products to patients, benefiting patients where there may currently be no or few treatment options.

The proposed POC framework will deliver on commitments made in the UK Life Sciences Vision to deliver a progressive UK regulatory offer that supports early patient access to novel treatments by providing regulatory clarity for POC manufacturing of advanced therapies and products.

More about the proposed new regulatory framework

The new regulatory framework:

  • will be based on and link into the current regulatory systems for medicines approvals, clinical trials, evaluation of regulatory compliance at manufacturing sites and safety monitoring. The safety, quality and efficacy of POC products will remain paramount.
  • is similar in concept to that established for control of blood products, which delivers regulatory control of quality while delivering the necessary flexibilities for the product type.
  • will support increased manufacture and supply of new products manufactured at POC whilst ensuring that these products retain equivalent levels of safety, quality and efficacy currently in place for medicinal products manufactured in factory-based locations.

Providing a new framework, tailored to these innovative products, will improve the availability of novel products, benefiting patients across the UK.

More detail on the proposed new regulatory framework

The proposed new framework:

  • sets out a model of regulatory requirements to provide the necessary regulatory oversight to assure that POC products have appropriate quality, safety, and efficacy attributes, whilst allowing the increasing numbers of patients to benefit from these innovative products in clinics and hospitals around the country.
  • is centred on a Control Site, which will be the only location named on the manufacturing authorisation. The Control Site:
    • will be required to maintain a POC Master File which amongst other information will name all of the individual POC manufacturing sites and this will be the mechanism to authorise those sites of manufacture.
    • will oversee all aspects of the point of care manufacturing system, including the individual manufacturing locations and their activities and be named on clinical trial and marketing authorisation applications. For example, the control site would be responsible for ensuring quality of the product and notifying the MHRA of any issues.
  • will link into current regulatory systems for medicines approvals, clinical trials, evaluation of regulatory compliance at manufacturing sites and safety monitoring. Some of the key regulatory foundations are based on those that have successfully been in use for many years in other areas of medicines and related regulations.

What are the expected benefits of a regulatory framework for POC manufacturing?

It is expected that the establishment of a regulatory framework for POC manufacturing would bring a range of benefits to:

  • Patients and carers – by providing access to new and more personalised treatments in a timely and more convenient manner with the potential for less travel and time spent in hospitals.
  • Healthcare professionals – by providing a greater range and more effective treatment options and improve patients’ adherence to those treatments.
  • Innovators – by providing clear regulatory expectations and enabling easier product development.

Which products will the new regulatory framework apply to?

Once implemented, this innovative framework will apply to all POC products manufactured in the UK, including a range of Advanced Therapy Medicinal Products (ATMPs) , such as cell therapy, gene therapy and tissue engineered products; 3D printed products, blood products, and medicinal gasses.

Source: MHRA; MHRA