NEW YORK – The Association of the British Pharmaceutical Industry (ABPI) on Monday said it has issued a report on the UK's approach to advanced therapy medicinal products (ATMPs), including cell and gene therapies, and recommendations to prepare it as more of these medications are developed.
The trade group said the UK National Health Service must do more to get ready for new advanced therapies coming down the pike targeted at larger patient populations.
Most ATMPs currently available are for rare diseases such as hemophilia B and spinal muscular atrophy, according to the report. In the past five years, an average two ATMPs per year have been approved in the UK, but this is projected to grow up to 10 to 15 per year by 2030, the ABPI said in the report.
The ABPI said rare disease treatments, although expensive, are easier for NHS to budget for, given the low numbers of patients. However, advanced therapies are on the horizon that aim to treat conditions with larger patient populations, such as dementia and Parkinson's disease. For these, the NHS will need to adapt how it approaches paying for such treatments, the trade group added.
The ABPI offered recommendations to UK regulators, such as testing outcomes-based payment methods that allow for payments over time — rather than a single upfront cost — and that are tied to treatment outcomes, building up the UK's ability to manufacture advanced therapies within the country and making the UK a destination for commercial clinical research.
"Advanced therapies have the potential to transform patients' lives with just a single treatment, avoiding a lifetime of care," ABPI CEO Richard Torbett said in a statement. "We need to take a long-term view of the value and cost-effectiveness of medicines, looking not just at the upfront costs, but at the lasting outcomes we want over years and decades. Doing so will require innovative new payment models that can respond to real patient outcomes."
The ABPI also called on regulators to allow for more flexibility in the evidence used to approve advanced therapies, for example, including the use of real-world evidence. As part of this, the National Institute for Health and Care Excellence and NHS England should support consistency in how treatment effectiveness data is collected, ABPI said.
The organization added that UK nations could establish a coordinating group to share lessons related to use of advanced therapies and to support capacity planning.