Putting patients at the heart of cell and gene therapy
Nareda Mills, Global President Patient Solutions, EmerGENE
Ben Beckley, Global Lead, EmerGENE
EmerGENE, is an end-to-end cell and gene therapy network which promises to support small and midsize biotechs with the commercialization of their discoveries. The first of its kind, EmerGENE is a multidisciplinary team of Ashfield experts focused on delivering expert-led guidance to biotechs from clinical to commercial and beyond. Find out more: https://oneashfield.com/emergene-cell-gene-therapy
Cell and gene therapies (C>s) present opportunities to not only treat disease, but to restore function and improve disease management. Although the opportunities with C>s are continuing to expand, there is a widening gap between the potential of treatment advances and the patient experience. The industry is beginning to acknowledge the problems faced in one of healthcare’s most complex areas for patient experience.
While the initial modern concepts of cell and gene therapy were pushed forward in the 1970s, novel advances such as clustered regularly interspaced short palindromic repeats (CRISPR) genome editing tools have propelled this new modality of disease treatment into breakthrough therapies. Over the past decade, novel C>s have been developed to treat chronic conditions, cancer and inherited disease. The nature of these therapies is in their personalization. Patients are often the source of the raw materials needed to create the treatment. Their ‘personal’ genetic information has been sequenced and their code may be ‘redesigned’ to treat the disease. Patients are now central to the research and development process, not merely receiving the end product.
The fact that every single patient’s healthcare journey is completely unique is exemplified in cell and gene therapy. This journey impacts not only the patient but also the patient’s caregiver and family. Patients have to undergo lengthy treatments, that often include a conditioning regimen, cell collection, and long wait times in between treatment. This can be an extremely draining and isolating experience, particularly during patient conditioning where long periods of time in isolation are essential to avoid infection. Removing a patient from their support system at this vulnerable time can also exacerbate social and cultural factors that all contribute to an often-negative perception of the C> healthcare journey. There is also a large financial burden on families, due to the high cost of treatment and the loss of ability to return to work for both the patient and often family members acting as caregivers. Most therapies are given through centers of excellence that may be far from patients’ homes. This results in long-distance travel and due to the length of treatment and monitoring, monthly stays in rented accommodation close to the centers. Both lead to an increase in the financial burden upon C> patients.
The premise of C>s enlists the patient into the supply chain, in cell collection and sequencing procedures. As with all components of a supply chain, the experience needs to be fully comprehended. For C>s to advance, the patient experience needs to be understood and valued, with true support offered in all areas from social to financial.
Valuing the patient experience
The industry needs to think differently about strategies to communicate effectively with C> patients. Companies need to address pricing, reimbursement and support systems with the patient experience in mind. To advance effectively towards a patient-centric C> journey, information delivery and experience analysis need to be conducted with a focus on a deeper level of empathetic and personalized interactions. Interactive patient journey maps are needed to record and analyze the experiences patients have through early onboarding, cell collection processes, treatment and beyond. For C> to reach its market potential and treat every patient effectively, communication needs to be prioritized. Patients need to understand the treatment, the support and options from which they can choose and how they can tailor their own healthcare journey. This can only be achieved through effective dialogue between patients, HCPs and the pharmaceutical industry.
The COVID-19 pandemic paired with this era of digitalization has accelerated the creation and importance of education and communication platforms for all HCPs and patients. As the healthcare industry has had to ensure that healthcare information is communicated rapidly and efficiently, novel solutions and streamlined processes are emerging. For C> patients this translates to more understandable and engaging content, on-demand healthcare access and support, as well as virtual appointments. Although these advances are aiding in positively impacting the patient experience, better patient engagement is still needed. Meeting with patients and families in the early stages enables the creation of patient support solutions that are as personalized as their therapy.
Solutions also need to be tailored individually by therapy area. Gene therapy, for example, might offer a cure for sickle-cell disease, and clinical trials have already demonstrated positive results. The therapy adds the beta globin gene, which is defective in patients suffering from sickle-cell disease to hematopoietic stem cells that have been removed from the patient. These are then returned to the patient, facilitating the production of anti-sickling hemoglobin. The experiences of patients with sickle-cell disease have been the subject of many influential reports, such as their inclusion in a series of FDA reports on The Voice of the Patient: Sickle cell report . The continuation and implementation of key results from these reports will be crucial for the advancement of C> into this area.
Before and beyond the doctor’s office
As with many new treatments there is an element of mistrust that must be overcome for C>s to reach their potential. Patients are suffering due to lack of information and misunderstanding that can often be propagated through HCPs. The industry needs to encourage and educate HCPs, who may be uncomfortable “losing” patients to a different provider and treatment pathway. HCPs may also be apprehensive in managing patients as they go through their C> healthcare journey. There is a gap that the industry understands can be filled through extensively educating and preparing providers with product information prior and post treatment.
Medical communications have an important role in positively impacting the HCP and patient experience. Providers and patients may be comfortable with their current healthcare management plan and uncertain if C> is right for them. This apprehension and delay in referral for curative treatment can lead to a negative patient experience with C> treatment viewed as a last resort. The industry alongside the centers of excellence, need to prioritize communication strategies to bridge the knowledge gap, enabling early life-changing intervention for patients.
For a true relationship to be built, realistic expectations must be set with patients and their families. Honest dialogue regarding treatment uncertainties and the translation of clinical benefits with dynamic, real-world data are needed as integral parts of patient onboarding. Novel communication solutions between community healthcare providers and the C> specialists can facilitate this stronger collaboration. For example, transparent communications within the supply chain that improves handoffs; shared real-time data to manage and improve the specifics of patients’ treatment; and forums for easier specialist-provider and provider-patient communications.
“Misinformation costs lives” – WHO Report 2020
Today patients want to understand as much as possible before making a decision regarding their healthcare. The pandemic has demonstrated the necessity for dynamic, scientifically accurate information to be easily accessible to all. The World Health Organization reported the importance of this in September 2020 regarding the COVID-19 pandemic, stating that information must be accessible and responsibly managed.
There is a clear challenge in increasing the accessibility and reducing the perceived complexity of C> lexicon. To ensure the optimal patient experience, the industry must use clear and consistent language to allow public education, where communication can flow between the industry and the individual. A reciprocal relationship of communication is crucial in creating a more positive healthcare experience.
The future for C> patients
The experience of a patient undergoing C> is a mixture of weighing the risks and benefits of each decision made. Education and the long-term tracking and evaluation of patients is central to the patient experience as was clear in the trials of Lentiglobin in sickle-cell disease. A case of acute myeloid leukemia (AML) in a patient dosed 5 years earlier led to an FDA-mandated clinical hold. The case was proven unrelated to the therapy but demonstrates the importance of long-term patient experience monitoring.
Companies are developing solid framework to overcome challenges, such as scattered data, giving the industry a unique advantage in understanding the complexity of the C> patient lifecycle. Novel data technology and software can enable a seamless experience for both providers and patients. In creating detailed maps of each patient’s clinical, behavioral and emotional journey companies can design a long-term support services program for the individual.
C> treatments are changing the healthcare landscape. Human connection is now viewed as critical for treatment success. Emerging technology and clear communication are driving the relationship between industry and the individual like never before. There is now a real opportunity to redesign the C> patient experience. Although there will be many hurdles to overcome, one thing is clear- patient experience is central to C> success.
 Wirth T, Parker N, Ylä-Herttuala S. History of gene therapy. Gene. 2013 Aug 10;525(2):162-9. doi: 10.1016/j.gene.2013.03.137. Epub 2013 Apr 23. PMID: 23618815.