Kate Hanman
UK Head of Rare Diseases, Costello Medical
Gene therapies are a promising treatment option for people with rare diseases living with genetic conditions. However, pricing remains an issue. Payment solutions must be identified to ensure gene therapies are available to those who need them.
Since the majority of rare diseases have genetic causes, it is encouraging that there are around 150 ongoing gene therapy clinical trials for rare conditions. We can therefore expect more gene therapies to become available in the coming years.
Those involved in pricing negotiations for gene therapies must continue engaging with each other, as well as the patient community, to ensure appropriate payment solutions are identified.
Gene therapy pricing challenges
Some gene therapies may be clinically effective for many years or decades. These long-term benefits are one of the reasons gene therapies are often considerably more expensive than other types of treatments.
It is not possible for the National Health Service (NHS) and gene therapy manufacturers to know exactly how long a gene therapy will be effective when a price needs to be agreed upon. This creates more uncertainty, making it harder to determine what a fair price is.
Gene therapy payment options
Traditionally, manufacturers of new treatments have offered the NHS discounts where there is any uncertainty surrounding how effective a therapy is. However, some have argued that gene therapies would still be too expensive, even if large discounts were used.
One alternative payment option called ‘outcome-based payments’ would mean the NHS only pays for a gene therapy for as long as it is beneficial to the person receiving it.
This option would require medical teams to collect data from people receiving gene therapies over a number of years. However, implementing outcome-based payments may be difficult due to the resulting administrative burden and high costs for the NHS, at a time when the NHS is already under increasing pressures.
Pricing challenges must not affect access
Those involved in pricing negotiations for gene therapies must continue engaging with each other, as well as the patient community, to ensure appropriate payment solutions are identified. These challenges must not prevent patients from accessing potentially life-changing gene therapies.
