From complexity to clarity in cell and gene therapy
Breakthrough CGT therapies are coming thick and fast, now pharma must work on multiple fronts to educate non-specialist stakeholders about their exciting possibilities
One of the most exciting frontiers in medicine, cell and gene therapies are already offering breakthrough treatments and potential cures in severe genetic diseases and cancer. The innovations continue to advance rapidly, with press releases announcing major breakthroughs on a seemingly monthly basis.
It’s undoubtedly a good news story but these advances bring with them the challenge of explaining all the exciting, but complex possibilities to patients and caregivers.
The groups that have historically been expected to help keep doctors up to speed cannot be expected to do so in this dynamic environment, says Kelly Hosmer, Director, Global Marketing for bluebird bio.
“The science is moving quickly, and this is a challenge for the traditional education bodies to keep up, whether medical schools or the educational arms of major physician organisations. They are in a fast-changing world. Just look at the last six months.”
Explaining the science
The novelty of CGT therapies and their speed of progress is not the only challenge. These are by any measure advanced therapies that draw on decades of deep and complex research. Within the CGT umbrella therapies also vary widely and explaining this all to non-specialists isn’t easy, says Nicolas Garnier, Director of Patient Advocacy, Rare Disease, Global Product Development at Pfizer.
“Gene therapy is the fruition of a century of innovation and discovery in molecular biology, genetics and precision medicine. That comes with many challenges. Gene Therapy is an umbrella term and it is a daunting task to define what it is and is not. It encompasses different technologies that have been very recently developed.
“It is one thing to discuss gene therapy with experts and doctors who are experienced in clinical research and trials but another with practitioners and HCPs who don’t specialise. Reports show GPs are not 100% comfortable with what gene therapy is and how it works. That means it is a definite area of opportunity for education.”
Understanding the science is just the start. Non-specialist HCPs may simply not know where to start helping their patients on the path to a gene therapy, says Hosmer. “They may be managing a large population of patients and CGT therapies may be available for a small number of those but they may not know who is eligible, who to refer them to, or how to refer them.”
Helping patients understand the science and the implications of choosing these treatments is an even greater challenge. “We have to make sure we don’t assume everyone knows the basics of molecular biology. That is even more applicable with the general public, patients and caregivers,” says Garnier.
Making informed decisions
“It is likely that new therapies will be developed for rare genetic disorders, which means that oftentimes we will be looking at a paediatric patient, which means that the parents will have to weigh the pros and cons and make a decision.
“How do we address that gap to enable someone to make an informed decision about whether or not gene therapy is right for you or your child or family member if they don’t know what a gene is, what DNA is, what a protein is or a viral vector? These all form building blocks of a proper understanding of what a gene therapy is and what it can mean for your body, health and life.”
So how does pharma help stakeholders through this steep and complex learning curve?
The first aim should be providing clarity. It is understandable that therapy developers will want to talk about their amazing breakthrough therapies but a product-first approach may not be the best place to start, says Hosmer.
Different terminology used by companies eager to showcase new technologies and approaches, sometimes used to describe the same processes such as gene addition or gene replacement, is a source of potential confusion for patients, adds Hosmer.
“They hear about gene editing and gene replacement. They hear a lot of words. The challenge is distilling the difference in those words. As an industry we can do better about being transparent with the community, about providing the context, about using accessible terminology, making gene therapy human and not over promising.”
Complexity and variety
Offering a big-picture overview of the state of the science and some biological basics is vital in building understanding and dispelling myths, confusion and misconceptions. Bluebird is seeking to do this via its educational site thegenehome.com in the US and is working on a similar initiative in Europe due to launch in the next 12 months.
“As a leader in the space, we have a unique opportunity to educate and address misconceptions. Education and transparency will be important in helping communities – HCPs, payers, patients and lay audiences - understand all the opportunities and the options as well as the history behind the technology,” says Hosmer.
A further challenge is explaining the complexity and variety of the treatments and the practical differences this entails when it comes to treatment.
Collecting patient stem cells and adding functional copies of a gene in a manufacturing facility before returning the cells to the patient is quite different and has unique challenges compared to an in vivo, single administration of a different therapy for example, says Hosmer. “Every disease area will be different yet the general audience hearing about gene therapy in the news may think it seems so simple, that you get an injection and you are done.
“Many therapies are distributed at select highly specialized treatment centres. You may need to go to an academic centre or centre of excellence. It could different for every therapy so that adds a level of complexity when it comes to getting a referral for treatment.”
Bluebird is working with hospital teams to develop resources to explain the processes involved.
Partnering with patients
Working with patient groups in building an understanding of CGT is also a vital educational strategy but the nature of this work will depend on the capabilities of the individual organisations, says Garnier. “There’s no one answer. For some therapies there are well organised patient organisations that work well with pharma.
“That will be very different in a scenario where the advocacy community is more recent and does not have the resources that are required to put together education awareness campaigns. You have to try to adapt to the needs of the specific community.”
Hosmer agrees on the value of close co-operation with patient groups. “Across the board our approach is to work closely to support the patient organisations,” says Hosmer.
The fact that these therapies are in many cases non-reversible raises the stakes further in ensuring HCPs, payers, patients and carers understand how these therapies work and what is at stake, she adds. “CGT as we know it today is not reversible. In many therapies these are significant, life altering decisions that have amazing upside. They are potentially curative but they are also major decisions for physicians, along with patients and their families.”