How can we fulfill the promise of Cell and Gene Therapy?

11 mins read

Ben Beckley, Global Lead at EmerGENE explores the market challenges holding back Cell and Gene Therapy (C&GT) from taking its place as an established treatment area. He explains how we can navigate obstacles to ensure C&GT can achieve its full potential.

As a treatment area, C&GT has shown robust growth in recent years. Some 3,366 treatments are currently in preclinical and clinical development[1]. The global market is projected to reach $13.8 billion by 2026 from $7.7 billion in 2021, at a CAGR of 12.4% throughout the forecast period[2].

There are several reasons for this growth, including high global cancer rates, combined with investment in research for treatments for cancer and rare diseases – 80% of which are genetic, making them a particular challenge to treat[3]. A new interest among pharmaceutical companies in collaborating with partners is also helping to advance research and development[4], contributing to C&GT diversification and market expansion.

However, there are challenges facing the C&GT sector, which have the potential to hinder growth. From our experience across EmerGENE working with clients who are developing, launching, or already marketing C&GTs, it is crucial to think differently about marketing strategy and communications for this treatment area.

Beyond matters of pricing, reimbursement, manufacturing, supply chain, and logistics, there are other obstacles facing C&GT developers that must be addressed to prepare and maintain a marketplace that will embrace this life-changing treatment. Understanding them is the first step to navigating them successfully.

Challenge #1: Patient and HCP skepticism

As a new treatment area, C&GT often elicits questions, apprehension and uncertainties, not just among patients, but their families and their healthcare providers (HCPs) as well.

A review of recent studies found that patients generally show broad acceptance of C&GTs. However, there is some variation between geographies, age and educational attainment, with older patients – with a higher level of education – most likely to feel positive about C&GT[5].

The length of C&GT treatment has been cited as a reason for skepticism among patients, as well as the loneliness of the treatment journey. The financial burden is another issue contributing to negative patient perceptions[6].

For HCPs, incorporating C&GT into their patient recommendations can be an enormous undertaking, and often means breaking familiar treatment habits. Our experience suggests that preapproval enthusiasm may also wane once a treatment becomes a reality, with some HCPs taking a more cautious approach to prescribing C&GT until they see their peers’ experience[7].

Clear communication with both patients and HCPs is key to overcoming these issues. Providing plenty of information, using plain, strategic language, aligned with existing conventions, is crucial to successfully engaging all stakeholder audiences to overcome any hesitation.

Challenge #2: Streamlining the patient journey

C&GT treatment often follows a complex patient journey. This is especially the case for rare diseases, due to the time it can take for a definitive diagnosis to be made. In these cases especially, our experience suggests patients and HCPS often need support from pharmaceutical companies to pursue a rare disease diagnosis and explore C&GT as a treatment option[8].

Complicating the issue is the fact that there may be geographical, motivational, financial, and educational barriers preventing patients from reaching one of the few available treatment centers.

With this in mind, it is important for pharmaceutical companies to provide support in overcoming logistical and knowledge challenges. They can also help encourage potential referring physicians, who may be uncomfortable “losing” a patient with whom they’ve built a long relationship to a different treatment pathway and team of providers.

Challenge #3: Managing expectations

The value of a C&GT is not always obvious to patients. Setting expectations is one of the most important ways providers can help patients and families make treatment decisions. Often this means being honest about uncertainties and translating clinical benefits to functional outcomes without overpromising.

The same applies to HCPs and payers. In order for them to be confident in supporting patients along a C&GT journey, they need to understand the duration of therapeutic effect, whether re-treatment is needed, the target patient and the point on their journey they need to be treated. This means they both need to see the available data and experience, which is often in short supply for new treatments.

The importance of such information to the acceptance of C&GT as a viable treatment is highlighted by bluebird bio’s trial of LentiGlobin in treating sickle cell disease. A case of acute myeloid leukemia (AML) in a patient dosed five years before, as well as an unconfirmed, then disproven, case of myelodysplastic syndrome, led to bluebird pausing its trial, a hold that was later mandated by the US Food & Drug Administration (FDA). During the hold, bluebird’s research demonstrated that the AML case was unrelated to LentiGlobin, but the case illustrates how vigilant companies must be in tracking and analyzing adverse events in the long term[9]. Failure to do so could undermine confidence among stakeholder audiences in any C&GT treatment.

Frequent data updates, real-world evidence collection, and communications from experienced prescribers and peers are vital to address data issues and provide both HCPs and payers with the information they need to make informed decisions. Simple, clear communication from pharmaceutical companies to patients, meanwhile, can provide them with the transparency and reassurance they need to be confident with the C&GT pathway.

Challenge #4: Putting patients first

The patient and their family should always be at the center of the treatment ecosystem. They are the ones experiencing the upheaval of serious illness and undergoing treatment.

Providing a patient-centric experience, however, is easier said than done. The logistical, clinical, and psychological path from prescription to treatment and follow-up can be time-consuming, frightening and overwhelming for patients. Making the actual C&GT treatment pathway as seamless and effortless as possible can help address this issue for both patients and their families.

Creating a detailed map of the patient journey for a new treatment – incorporating clinical, behavioral and emotional milestones – can help identify the critical points where additional patient support may be needed. Doing so as early as possible during the development phase can allow the creation of a long-term, high-touch support program that can help patients and families through the treatment.

Time to develop the journey as well as the therapy

C&GTs have remarkable potential to transform the health and wellbeing of patients with a wide range of diseases, in some cases, providing them with effective treatment for the very first time.

However, they can’t live up to this promise if we don’t address the patient and HCP journey and take steps to overcome their lack of knowledge about C&GTs, manage their expectations and enhance their experience of the treatment.

It is vital that drug developers invest in their research, education, and marketing efforts alongside the therapy itself long before the treatment reaches the regulatory approval stage. Every disease brings its own unique challenges, meaning that every treatment must have its own tailored journey in order to meet the needs of target patients.

By doing this work early, pharmaceutical companies can ensure they have the tools in place to overcome commercialization challenges so that their treatments are able to reach the patients who need them.

To find out more about how to address these C&GT challenges, read our whitepaper: Fulfilling the promise: Navigating the market challenges in gene and cell therapies

[1] American Society of Gene and Cell Therapy (ASGCT), Gene, Cell, & RNA Therapy Landscape, Q1 2021 Quarterly Data Report


[3] Mind+Matter, Fulfilling the promise. Navigating the market challenges in gene and cell therapies, May 2021


[5] Olalekan. L. A. et al. Patient and public perspectives on cell and gene therapies: a systematic review 2020

[6] Wang, J. H. et al. Public attitudes toward gene therapy in China. Mol. Ther. Methods Clin. Dev. 6, 40–42 (2017)

[7] Mind+Matter, Fulfilling the promise. Navigating the market challenges in gene and cell therapies, May 2021

[8] Mind+Matter, Fulfilling the promise. Navigating the market challenges in gene and cell therapies, May 2021

[9] Mind+Matter, Fulfilling the promise. Navigating the market challenges in gene and cell therapies, May 2021

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