Guest Column | March 27, 2024

The Role Of Behavioral Science In Improving Cell & Gene Therapy Adoption

By William Hind, founder, Alpharmaxim; and Lisa Campbell, Ph.D., SSI Strategy

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Despite 10+ years of familiarity to regulators, cell and gene therapies are still nascent, with the result that there is still a lot of work for therapy developers to do to promote their uptake. For these important novel treatments to have the desired impact for patients, the whole health ecosystem needs to be confident in their potential benefits over current accepted therapies. It’s here that behavioral science has an important role to play in signaling where any barriers to adoption lie for physicians and their patients, so that biopharma companies can tailor their messaging.

Challenging entrenched patterns of decision-making is important so that patients don’t miss out on a real chance of a cure or a better quality of life and so that healthcare providers (HCPs) keep up with the fast-moving science and the expectations of the patient community. Indeed, the U.K.’s Medical Research Council (MRC) is now advocating the use of behavioral science in the design of interventions such as marketing campaigns, to ensure that important new biopharma innovation fulfils its potential for patients. A realization has dawned that even when a new therapy has been approved by regulators as being safe for use, that does not guarantee its take-up.

Barriers To Breakthrough Treatment Adoption May Not Be Obvious

In November 2023, the U.K. approved the gene therapy Casgevy, which aims to cure two blood disorders, sickle cell disease and beta thalassaemia: painful, lifelong conditions that can be fatal, for which up to now a bone marrow transplant from a closely matched donor has been the only permanent treatment option.

The availability of new solutions is tremendously exciting, but too often that potential remains latent. The barriers to novel therapy take-up may not be obvious. Education — a lack of data, of information – is rarely the issue, although broadening awareness is important to ensure that new therapies are duly considered as an option. But there are likely to be other subtler and more involved barriers to adoption that must be overcome.

In the case of rare and debilitating diseases, patients, their caregivers, and their physicians may have inadvertently arrived at a comfort zone (patients treated with Casgevy have talked about their amazement at what they tolerated, in retrospect,1 having learned to manage the disease as well as they could, even if their quality of life has been suboptimal.

In many cases, this is because a change of approach can feel frightening - especially if the proposed new therapy is so groundbreaking that there are very few comparative patient cases to draw on to demonstrate success. Encouraging such a profound shift in approach is likely to require a destabilizing of that false sense of comfort.

How Might Behavioral Science Help?

In life sciences, the opportunity associated with behavioral science is to lower any identified resistance to supporting and offering novel therapies over entrenched drug prescription pathways.

While it’s possible to make an educated guess about healthcare providers’ reasons for sticking with tried and tested treatment choices (including officially recommended first-line treatments, budget restrictions, and/or a lack of familiarity with the emerging options), the reality is usually more complex. The established COM-B model for behavioral science sets out 93 different techniques and how they can be successfully combined to address barriers to change, based on the relative roles of Capability, Opportunity, and Motivation (M) as determinants of current behavior (B).

Drug approval and associated education/data represents only one-third (the Capability element) of the combined factors that may be needed to effect a change in behavior. The other factors at play can be harder to predict. It could be that the HCPs don’t have access to the right resources to administer a new treatment plan. Or perhaps prescribers are not being encouraged to try something new by the system they are in. (It doesn’t help that the global healthcare and life sciences ecosystem is still fairly siloed, so that details of successful instances of similar treatments being applied in other countries are not publicly available.)

Pinpointing How & When To ‘Nudge’ The Target

Distilling the particular combination of “blockers” to behavior change is the first step in designing an effective medical communications campaign. Ideally, this needs to be guided by a structure, framework, or methodology to ensure that campaigns are evidence rather than assumption based and have maximum impact across the target blend of channels.

In February 2022 the NHS rolled out a lifesaving treatment for babies and young children with a rare and fatal genetic disease, offering the prospect of a normal life. The revolutionary gene therapy, Libmeldy, is used to treat metachromatic leukodystrophy (MLD), a disease that causes severe damage to the nervous system and organs and results in a life expectancy of just 5 to 8 years.

The therapy has made the headlines, not only for its breakthrough properties but also for its high price-tag (it has been dubbed the world’s most expensive drug). But that price has to be put in context: this isn’t an intervention that has to be administered repeatedly, as with existing treatment regimes. And powerful data continues to emerge to support Libmeldy’s potential: the gene-edited cell therapy has continued to show benefit in updated data from an integrated analysis of 39 treated patients, with follow-up now reaching up to 12 years.2

In other words, challenging the long-term cost/benefit considerations is among the barriers to be overcome. And it’s here that an evidence-based marketing communications campaign offers to help in first identifying and then targeting objections as they emerge.

Behavior changing frameworks contain a set of instructions to guide the development of a behavior change strategy. Drawing on the COM-B model described above, medical communications teams can start to determine what kind of campaigns might work best to drive change. That could involve a combination of targeted education and messaging that focuses on the relative quality of life and also the overall value (cost/reward). That’s when that quality of patient experience is added to the lower lifetime cost of treatment, for instance (of one-time cell or gene therapy versus long-term drug administration and symptom management).

Although behavioral science-based strategies have yet to take shape in promoting cell and gene therapies, broader health examples illustrate what is possible.

In a public health context, the COVID-19 pandemic required a series of new behaviors, starting with fastidious handwashing, face covering, and keeping a distance from others, and it was very targeted, nuanced communications that elicited the desired changes. Getting the public on board required improving their knowledge of what to do and how (Capability); ensuring they had access to soap/hand gel or face coverings (Opportunity); and developing a clear plan of when they should do these things and align this with why they needed to do it/what they would get from it (Motivation, e.g., reduce the risk of negative outcomes/protect themselves and others).

With strong evidence-based insights into the changes that are needed, and the specific barriers that need to be overcome, it becomes easier to identify the right type of message and content, as well as the most effective combination of modes of delivery and precise targeting. Other more significant shifts will be needed, too — such as the scientific, medical, and regulatory landscapes doing more to share experiences and expertise (regulators could do more to communicate the benefits of the newly approved approaches, e.g., that they do not require costly long-term intervention, like a drug regime) to the payors and HCPs.

But understanding where prescriber or patient inertia or reticence is strongest will go a long way in helping to identify what drug companies need to do differently in their market communications, hand in hand with other considerations such as greater attention to the patient voice.


  2. “Orchard Therapeutics announces presentation of data comprising the clinical package for the OTL-200 BLA in MLD at the SSIEM Annual Symposium 2023.” News release. Orchard Therapeutics. August 31, 2023. Accessed September 1, 2023. (as reported by CGT live, September 2023:

About The Authors:

William Hind founded Alpharmaxim in 2001, after more than 35 years working in sales, marketing and business development in the pharmaceutical industry, including roles at Merck, CMC International, and Oxford Pharmagenesis. Alpharmaxim helps cross-functional biopharma teams communicate effectively with clinicians about new medicines or alternative treatment regimens and their potential to improve the patient experience, supported by relevant scientific evidence. Hind can be reached at

Lisa Campbell, senior director at SSI Strategy Global Life Sciences Consultancy, served as a medic (working in obstetrics and gynecology in the U.K. NHS for 13 years), and then for the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) for a decade in its clinical trials unit. SSI Strategy is a boutique management consulting company with particular expertise in advanced therapeutic medicinal products (ATMPs).