Gene therapy: Getting ready for the next medical frontier

Gene therapy has been talked about for years as the next medical frontier but is only now becoming a reality. How can we be ready for these new innovative therapies?

There have been significant advances in gene therapy in recent years, with increased clinical and scientific understanding and improved manufacturing capabilities. The UK is recognised as a world leader in genomics, accounting for over 12% of global cell and gene therapy clinical trials.

Whilst progress has been made, there are still many hurdles that companies face in securing patient access to their innovations, both worldwide and in the UK. These challenges are broad and cross all stages of drug development and access.

  • Clinical data

Although our understanding of gene therapies has come a long way, long-term efficacy data for many therapies are yet to be established. The recent FDA rejection of BioMarin’s gene therapy application signals potential concerns over the durability of these therapies; companies and regulators will need to work together closely to agree on what evidence is needed for approval.

  • Pricing and reimbursement

One-time therapies disrupt current healthcare economics as, while the expense is high; they don’t require repeat administration. Companies are now starting to explore innovative cost-based solutions to combat this, such as performance-based regulation in which the payer only pays a portion of the cost upfront, with the remainder based on meeting pre-specified outcomes.

  • Complex supply chains and logistics

Gene therapies and other advanced therapy medicinal products (ATMPs) have a more complex supply chain than most other medicines, complicating the logistics of getting gene therapies to the people who need them. Whilst this is specific to each therapy, this can include complex quality checks, short shelf-lives, temperature requirements and patient scheduling.

  • Clinical practice and service re-design

Re-design of services and systems for routine delivery of a new class of medicines, including NHS adaptation, will also be needed. Currently, due to the use of gene therapies for the most part in rare diseases, experience of administration is lacking. Education and training for healthcare professionals to understand and administer these treatments will be vital once approved.

The added challenge of the global coronavirus pandemic

The global coronavirus pandemic will likely have both short and long-term impacts on the entire healthcare system, from approval and reimbursement of new therapies, through to supply and delivery of these medicines to patients.

With an increased pressure on the healthcare system, COVID has required adaptations across the board. For example, NICE was originally prioritising its work but has now begun to publish planned guidance again now that things are starting to return to normal. Not only this, but there has been a shift in how patients receive their care and treatment, with testing before procedures, social distancing in medical centres and increased virtual consultations.

It will be important to bear in mind that this increased pressure on the system will be an additional hurdle for companies trying to launch new therapies, which can only add challenges in an already complex therapy area.

Collaboration is key

These challenges cannot be addressed by one organisation alone. They will need a collaborative effort to help patients get the therapies they need. To effect genuine change, industry, patients, payer bodies and many others will need to work together.

As a strategic communications agency with years of experience launching complex therapies, we are keen to play our role. We need to demonstrate the benefits that gene therapies may bring to patients, and support the clinical and non-clinical community with education and information that enables integration into the care pathway.

Whilst communications alone can’t solve these issues, there is an opportunity for strategic and relevant communications to elevate the gene therapy effort.

We are happy to discuss our experience working on the most innovative medicines and collaborate with organisations working in gene therapy, if you would like to know more please get in touch with

Related Blog posts

Corporate counsel and implementation

‘What can’t we afford to treat anymore?’

“The pharma industry in this country… could be an engine to modernise, improve and grow the economy, and we’ll all benefit from that” – Sir David Nicholson On Monday evening,…

Collaboration is key
Corporate counsel and implementation

Alone we can do so little; together we can do so much

“Caught between the critics who say NICE is being ‘ridiculous’ for not listening to the experts, and those that say we are being overly influenced by those with a vested interest…”…

Patient involvement and engagement

A glimpse into the patient world during COVID-19

Stories of anxiety, struggles with accessing medication and difficulties finding information relevant to specific diseases. These are just some of the things patients experienced during the early stages of the…

Post a comment

Your email address will not be published. Required fields are marked *


This site uses Akismet to reduce spam. Learn how your comment data is processed.

Get in touch

The quickest way to get in contact with us is to give a us call or ping us an email.

Sign up to our newsletter

Keep up to date with the great things we’re doing