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There are strong indications that patient acceptance of cell and gene therapies increases significantly following the provision of clear, easy-to-understand information. Here, we outline some opportunities to create more patient-centric research in this area.
Co-designing with patient communities
A key example of co-designing and approving therapies with patients was demonstrated during the review process for Casgevy, a breakthrough gene therapy Casgevy has been approved for the treatment of sickle cell disease (SCD) and transfusion dependent beta thalassemia (TDT) in patients aged 12 years and over in the UK. The Medicines and Healthcare products Regulatory Agency (MHRA) collaborated with the Sickle Cell Society to work closely with patients, ensuring that patient voices and lived experiences were shared and included as a significant factor in the benefit-risk review stage.
One patient who was part of the review process said: “It was so important to me that the voices of people who live with the condition were heard and understood.
“Making my contribution to a decision about such a life-changing therapy is really important to me. Seeing it approved gives me hope for the future.”
Integrating patient voices in the approval and development processes via collaborations with advocacy groups and organizations is a highly impactful way of building awareness and trust in gene and cell therapies. By making patient involvement the standard in therapy design and approval, patient understanding and acceptance is likely to improve significantly.
Clarity around future therapy options
Creating greater patient awareness of the impact of undergoing gene or cell therapy on future treatment options is also a key area of focus. In many cases, agreeing to undergo cell or gene therapy means that patients will not be eligible to receive a second one should a newer, more effective therapy be developed in the future. Additionally, in some cases of SCD, receiving a gene therapy means that the patient is no longer eligible to receive bone marrow transplant therapy.
Transparently sharing this knowledge is vital for empowering patients to give truly informed consent and for healthcare providers to establish what a successful outcome looks like for each individual based on their unique needs and situation.
Consent as a multi-stage process
The complexities of gene and cell therapies, including the risks and side effects of preconditioning and autoimmune response (which in some cases can lead to organ failure and the need for transplants) mean that informed consent is not a single one-size-fits-all approach. Patients need to be fully aware of what the treatment process involves and the risks at each and every stage (particularly in cases where cell or gene therapy is the only viable treatment option), so that they can continuously make an informed decision about if the therapy is right for them, as well as have full awareness of their right to withdraw and stop treatment at any time.
Expanding support systems during and after trials
Another key area for expanding patient-centric approaches is the development of effective support systems for patients and carers both during and after clinical trials. Again the provision of clear, easy to understand information from the outset will prove central to the process; from trial recruitment to patient retention and recontacting.
From-home capabilities
Decentralised, from-home capabilities will be key to diversifying patient participation and enabling access for patients who are based in remote geographical regions or for whom transport and mobility can be challenging. Being able to return DNA and blood samples from home, and complete eligibility surveys and e-consent forms using digital services, significantly reduces patient burden and the time required to participate.
From-home capabilities are particularly beneficial in the rare disease space and have the potential to significantly improve trial recruitment efforts. In rare and ultra rare diseases the limited number of individuals who fit study requirements can result in potential participants being geographically dispersed, making decentralized approaches fundamental to the success and efficiency of recruitment.
Concierge services
Offering support in the form of concierge services also has the potential to significantly improve patient experience. Assigning personal assistants to support patients and carers throughout the process via in-person visits or telemedicine increases understanding and reduces barriers to participation while improving engagement, outlook and aiding the process of continuous consent. Such approaches also enable dynamic and adaptive changes to trial design in order to respond to emerging patient needs, questions, and concerns.
Partnerships with advocacy groups
Partnerships with patient advocacy groups are not only key to understanding current patient perceptions of gene and cell therapies, but have a central role in providing support to patients and carers both during and after clinical trials. By collaborating with advocacy groups, drug developers can provide participants with access to a built-in support network that understands their specific needs and day-to-day challenges.
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