Advanced therapy medicinal products (ATMPs) hold great promise for treating many different diseases and the industry is seeing great success with therapies moving from clinical trial to licensed products. Over the coming years patient treatment is expected to increase significantly rising, in approximately 5 years, to tens of thousands of therapies being delivered to patients in the UK each year. However, to achieve this scale and embed cell and gene therapies in the health system for routine use, many barriers still need to be addressed.
ATMPs are different from traditional medicines, as biological material they present manufacturing and transport challenges, the delivery may be challenging requiring a multi-disciplinary approach and high one-off costs also present new reimbursement challenges.
In this blog, we have summarised what we consider the main hurdles to patient access in the UK. This blog is just one of many covering this topic as part of Signals’ fourth annual blog carnival. Please click here to read what other bloggers think about this topic.
The manufacturing hurdle
Manufacturing an ATMP is often difficult and costly, with inherent variability of the starting material and the timing of manufacture dictated by the patient treatment regimen. Currently, manufacturing practices for ATMPs are still in early stages of development and today companies must focus on meeting immediate demand for clinical trials and early supply. At present few production processes or technologies have been standardised and, as a result, lack robustness and have a high cost-of-goods. Innovation is required to prepare for delivery at scale and further cost and productivity gains need to be realised.
As the industry grows and progresses, more new and unique skills are required. The number of appropriately skilled operators also places restrictions on the manufacture of these products. Recruitment and retention of these individuals is becoming increasingly challenging. The UK is proactively addressing this challenge with the development of an apprenticeship scheme specific to ATMPs.
The clinical supply hurdle
ATMPs are associated with complex supply chains, demanding storage requirements and high-cost traceability requirements. Between the initial collection of patient cells and the administration of the final product, there are unique logistical challenges, with complexity added by the short shelf life of many of these living products. Many shipments are temperature-critical and time-sensitive, requiring additional IT infrastructure for ongoing monitoring.
With the manufactured product arriving at the clinic there is a significant task in integrating these new therapies with current clinical practice, requiring specialist manufacturing centres to work in complete coordination with clinical centres. Hospital pharmacies and/or cell labs are a vital part of the supply chain, and will be responsible for the storage, handling, preparation and logistics of a therapy within the hospital. ATMPs, especially those which are cryopreserved or with a very short-shelf-life, may require specialist handling and expertise that may not be immediately available in pharmacy departments.
Clinical use can be more complex, with intense periods of clinical involvement and follow up. The understanding of the potential impact of these therapies by frontline delivery staff can also not be underestimated. Capacity is an issue for most treatment centres, which may require service re-modeling e.g. increasing apheresis capacity, pharmacy facility expansion etc.
The reimbursement hurdle
Existing reimbursement practices within the UK healthcare system do not easily accommodate the potential outstanding value and risk profile that the potentially curative and disease-modifying ATMPs can deliver. These therapies may result in a long period of patient benefit from a single treatment, a marked difference from a pharmaceutical that would be administered and paid for in small payments over the course of a patient’s life.
Current payment systems are not well developed for potential payment schemes, which could share the risk between manufacturer and healthcare system. Such innovative payment models are under discussion in the UK and the Health Economics and Market Access team at CGT Catapult are actively exploring how outcomes-based reimbursement could support the adoption of these therapies.
The right ecosystem for the adoption of ATMPs
The UK has a number of innovative programmes to improve patient access involving dedicated resources within the National Health Service (NHS) including the Advanced Therapy Treatment Centre (ATTC) Network and the Accelerated Access Collaborative (AAC). The AAC brings together industry and the NHS with the UK government to identify innovations that should be made available to patients in the shortest possible time. The ATTC network is a coordinated network of treatment coalitions (Northern Alliance ATTC, iMATCH and Midlands and Wales ATTC) that aims to accelerate the routine supply of these innovative, but disruptive, therapies by the establishment of national processes and procedures and also through collaboration between NHS and industry. Together these initiatives aim to remove barriers to uptake of innovations, to accelerate UK patients’ access to transformative therapies.
To embed advanced therapies in the healthcare system it is necessary for all the barriers detailed above to be considered in a holistic way if they are to be overcome. The schemes detailed above, together with the apprenticeship and NHS training programmes, work to develop the UK ecosystem to facilitate this.