Medicines and Medical Devices Bill

Written evidence submitted by Advanced Accelerator Applications (AAA) (MMDB11)

Medicines and Medical Devices Bill 2019-21

Written Evidence Submission to the Public Bill Committee

About AAA

By way of background, we are pioneers of an exciting pillar of oncology using precision nuclear medicine. Our mission is to transform the lives of people with cancer; using expertise in oncology to develop highly targeted radioligand therapies for the benefit of patients in the UK and beyond. Radioligand therapy (RLT) is an innovative type of cancer therapy that can offer life-enhancing treatment by delivering therapeutic radiation to cancer cells in a targeted and precise way, wherever they are in the body. Our targeted therapy and precision imaging products are mainly used in clinical oncology, cardiology, neurology and infectious/ inflammatory diseases. The NHS has already taken positive steps to ensure that patients can benefit from this treatment approach in a rare form of neuroendocrine tumours.

The Medicines and Medical Devices Bill represents an opportunity to ensure that the UK’s life sciences sector can flourish post-Brexit. The establishment of new legislative frameworks following the UK’s exit from the EU will enable patients, academia, Government and industry to work collaboratively to shape and ensure the optimum regulatory structure is in place so that patients can benefit from highly innovative treatments such as RLT. We share the Government’s commitment to encouraging innovation through an agile and flexible regulatory system so that the Government can respond to developments in science in a sophisticated way. We are also keen that any future regulatory framework provides the long term certainty and confidence required to support the development and availability of innovative medicines and that this primary legislation does not lead to regular and indefinite alterations, which would undermine that objective.

The Medicines and Medical Devices Bill 2019-21 provides an opportunity to develop a foundation to establish the UK as a transformative life sciences hub globally and we are keen to work with Parliament and Government in three specific areas:

1. The Government should provide further clarity of its objectives around section (2,1,a) ‘authorisations to manufacture human medicines’

The manufacturing and administration of treatments and molecular diagnostics in hospitals, as opposed to licensed and CE marked treatments and diagnostics, is often referred to as ‘home brew.’

We feel that the use of ‘home brew’ in indications where a licensed alternative is available undermines the established regulatory frameworks that are in place to protect patients’ safety and ensure healthcare professionals can prescribe with confidence. There are several important quality standards specific to the development and production of licensed medicines that are not guaranteed through the use of unlicensed medicines manufactured in hospitals, such as the correct sourcing of raw materials, iterative consistency, R&D collaborations and pharmacovigilance. As a result, the use of ‘home brew’ could result in patients being given medicines that do not adhere to the UK’s high quality and safety standards.

We are concerned that unless "authorisations to manufacture human medicines" (2,1,a) is qualified in the draft Bill, this line could unintentionally imply support for the inappropriate use of unlicensed Advanced Therapeutic Medicinal Products (ATMPs), such as RLT and other nuclear medicines. It will also be important that government works closely with companies that have expertise in the discovery, development and manufacturing of ATMPs before the introduction of secondary legislation to ensure that the resultant regulatory framework maintains confidence and avoids unintended consequences for the safety and availability of medicines.

Key points:

· The Government should clarify its intention in this area to ensure that it is not misinterpreted by hospitals, life sciences organisations or as part of any future secondary legislation.

· The Government and the NHS should partner with experts in developing and scaling up ATMPs and RLTs in order to develop a framework - for introduction in secondary legislation - that will support hospitals in developing and spinning out breakthrough treatments where other, licensed treatments do not exist.

· AAA calls on regulatory bodies, such as the Medicines and Healthcare products Regulatory Agency (MHRA), to rigorously uphold quality and safety standards and wishes to work with the Government to develop a regulatory framework that is supportive of developing innovations in hospitals, whilst maintaining the preference of licensed treatments where they are available.

· Government should work with manufacturers such as AAA and other organisations that work with RLTs at the earliest possible opportunity to generate a consensus on appropriate quality standards, and to ensure consistency for patients no matter where they might receive treatment. This includes ensuring that ‘home brew’ is only utilised in specific and defined cases, as opposed to being used as a generic measure to generate cost savings that could jeopardise the quality of the medicine and patient safety.

2. The Government should work with the MHRA to publish any proposals for secondary legislation that amend medicines regulation to provide consistency of manufacturing quality and maintain a world-leading framework, both now and for future breakthrough medicines.

Through the Medicines and Medical Devices Bill 2019-21, the Government is creating a powerful mechanism that allows it to use secondary legislation in the future to make far reaching changes as to how medicines are regulated in the UK. Secondary legislation traditionally receives less parliamentary scrutiny. It is therefore very important that the Government works closely in partnership with life sciences companies and regulatory experts, both during the passage of the Bill and in the development of subsequent secondary legislation that may be laid before Parliament to shape future regulatory requirements. This will be particularly important in the development of secondary legislation concerning the manufacturing and use in patients of unlicensed ATMPs where a licensed alternative is available.

It will also be important for the Government to clearly set out its proposed regulatory framework for public consultation at the earliest opportunity so that Parliament has adequate opportunity to thoroughly understand the implications and consequences that any changes might result in. 

The Government should understand the importance of building confidence in UK regulatory bodies, particularly as it exits the European Union (EU) and the EMA, which currently provides licencing authorisation for medicines used in the NHS. The MHRA will need to establish itself as a standalone national regulatory authority that is equally committed to both high standards and consistent quality of medicines manufacturing if it is to retain its reputation as a world leader.

Life sciences companies currently have some concerns about the administrative and cost implications of having to file for marketing authorisation with a new, separate national licensing authority after the UK leaves the EU. It will be important to work closely with the industry - both industry groups, but also individual companies that have specific expertise in highly technical areas such as RLT – to ensure that the future regulatory regime is robust, internationally competitive, and fit for a future of significant scientific breakthrough.

Key points:

· Government should immediately meet with experts in the manufacture of RLT and other ATMPs to understand the complexities of development, manufacturing, upscaling, clinical trials development and licensing of targeted treatments with a short shelf life.

· The MHRA should consult with experts in the development and manufacture of RLT and other ATMPs, at the earliest possible opportunity, to support it in developing a regulatory framework that provides consistency of manufacturing quality and maintains a world-leading regime for both current and future breakthrough medicines.

· Government should work with the MHRA to ensure that any secondary legislation to amend the regulation of the use of medicines should not unintentionally:

o Reduce the consistency of quality that patients receive in different parts of the country as a result of different manufacturing processes of targeted medicines.

o Undermine confidence in the MHRA as a world-leading regulatory authority further to exiting the EU and leaving the EMA.

o Degrade competitiveness of the UK as a place for inward investment into R&D and medicines manufacturing by undermining the confidence of the life sciences industry in their high regard of product licenses and innovator/intellectual property rights that has made the UK traditionally so attractive.

3. The Government needs to plan and build greater system readiness within the NHS so that it can continue to benefit from RLT and fully realise its ambitions to enable hospital level innovation in the discovery, development and manufacture of ATMPs.

RLT represents a pillar of oncology treatment that, in common with other ATMPs, requires thoughtful long-term planning, as well as enhancements to infrastructure and workforce, to ensure it can be provided to all patients who qualify for it. Currently, there is little system recognition of the future potential of RLT. AAA believes that increased understanding amongst system leaders will support discussions around the importance of infrastructure and workforce planning.

Work between AAA and NHS England to introduce RLT to the health service and patients with gastroenteropancreatic neuroendocrine tumours (GEP-NETs) has, to date, been successful. We believe, however, that further attention is needed to ensure that RLT can be effectively integrated into clinical practice, particularly as the use of this treatment expands into additional indications, to avoid further delays and variation in accessibility and uptake. This will also support the UK in maintaining its leading position in the use of RLT.

If the intent of the Government is to support the NHS in discovering and scaling up scientific breakthroughs, including in areas such as RLT, they need to be aware that NHS capacity to meet current patient need is at its maximum. To be able to do more – both in the treatment of patients in licensed indications as well as enabling the NHS to discover breakthroughs – the issue of improving capacity and system readiness will need to be recognised and addressed.

Key points:

· Government should work with AAA to plan future NHS requirements for the delivery of RLT, an important pillar of oncology treatment, to ensure that patients can fully benefit from the Government’s intention to enhance NHS ability to discover, develop and manufacture ATMPs.

4. Summary

AAA welcomes the Government’s ambition to encourage the discovery, development and manufacture of innovative personalised treatments within NHS hospitals where no licensed alternative exists. The Medicines and Medical Devices Bill 2019-21 provides an opportunity to create a framework that can safely allow this to happen without undermining confidence in existing regulatory authorities and maintaining the attractiveness of the UK to life sciences investors.

We share the Government’s commitment to encouraging innovation through an agile and flexible regulatory system that can respond to developments in science in a sophisticated way.

The establishment of new legislative frameworks following the UK’s exit from the EU will enable patients, academia, Government and industry to work collaboratively to shape and ensure the optimum regulatory structure is in place so that patients can benefit from highly innovative treatments such as RLT.

As a leading manufacturer of RLT, AAA proposes working with the Government at the earliest possible opportunity in the following three areas:

1. The Government should provide further clarity of its objectives around section (2,1,a) ‘authorisations to manufacture human medicines’ and work in partnership with MHRA and manufacturers of ATMPs, including RLT, to generate consensus on appropriate quality standards and ensure consistency for patients no matter where they might receive treatment. This includes ensuring that ‘home brew’ is only utilised in specific and defined cases, as opposed to being used as a generic measure to generate cost savings that could jeopardise the quality of the medicine and patient safety.

2. The Government should work with the MHRA to publish any proposals for secondary legislation that amend the regulation of the use of medicines in a way that provides consistency of manufacturing quality and maintains a world-leading regime, both now and for future breakthrough medicines. The MHRA and Government should work with experts in the discovery, development and manufacture of RLTs and other ATMPs to support it in developing a regulatory framework that provides consistency of manufacturing quality and maintains a world-leading regime both now and for future breakthrough medicines.

3. The Government needs to plan and build greater system readiness within the NHS so that it can continue to benefit from RLT and fully realise its ambitions to enable hospital-level innovation in the discovery, development and manufacture of ATMPs. Government should work with AAA to plan future NHS requirements for the delivery of RLT to ensure that capacity requirements can be planned so that all eligible patients can fully benefit from access to fully licensed treatments.

AAA will be delighted to work collaboratively with Parliament and Government to ensure patient safety is maintained and support the competitiveness of the UK life sciences sector.

May 2020

 

Prepared 10th June 2020