While patient engagement from early development through market access to patient access is becoming more and more a standard in the pharmaceutical industry, it is even more important in cell-, gene, and organ replacement therapies (so-called ATMPs).
Why is this the case?
Early Patient Preference Information
First and foremost, we need to currently understand what patients (and caregivers) are truly expecting from a therapy for a given disease and what their preferences are in terms of new therapies. Otherwise, we are at risk of missing the point when designing a development program. So, insightful patient-preference-information should be obtained right at the beginning of the development journey. In the case of ATMPs, this can and should also include the potential promises but also hurdles and issues that can be expected.
Patient-relevant and acceptable clinical trial design
Patient engagement is crucial in designing the clinical development program for changing paradigms that have governed traditional pharma development for decades such as large sample sizes, parallel designs, and well-established endpoints. If we want patients to benefit from ATMPs in yet poorly served diseases, we need to be prepared for new endpoints including surrogates, adaptive designs, truly meaningful and validated patient-reported outcome measures, and post-approval enhancement of the evidence base on long-term follow-ups and insightful disease-specific registries.
Patient to site recruitment approaches
Clinical trials in ATMPs often need referrals of patients from hospitals not participating in a given trial to the few well-qualified study sites. Relying here only on the ability and the preparedness of those referring sites is a risky endeavor and may end up in significant delays and increased costs. We need to reverse the traditional paradigm of “site to patient” to “patient to site” by empowering patients to find a clinical trial site and assisting them in joining the eligibility screening. However, at the same time, ATMP clinical trial sites must be protected from requests of potential trial participants that will never meet the eligibility criteria of the study. Only a combination of digital and highly personalized solutions accompanying patients from first contact to the eligibility screening appointment with the investigator will help to overcome this challenge.
Newborn screening and widely available genetic testing
When ATMPs for genetic diseases are getting closer to marketing authorization, genetic screening including newborn screening for those diseases that manifest during childhood will be needed to ensure a truly patient-focused and inclusive healthcare service. However, introducing such screening offerings in a healthcare system requires multi-stakeholder approaches and very timely planning ahead of the potential availability of a new ATMP.
Elucidating the “mysteries” of ATMPs
The mode of action and the particularities of the production of ATMPs make it necessary to address questions from patients appropriately, in full compliance with the law and industry codices, but also as empathetic as possible. That effort requires timely preparation ahead of launch. It must include educational aspects (such as videos or infographics) and should also offer the opportunity for true dialogue, e.g., by training HCPs and their staff in regard to motivational interviewing and communication. So, aspects like eligibility criteria (e.g., based on age or gene characteristics), waiting and processing times and the need for follow-up data should be addressed as well as risk-management programs and the anticipation of safety concerns.
Putting pricing into perspective
Undoubtedly, the pricing of ATMPs may be of particular concern to healthcare systems but also sometimes to patient representatives and HCPs. Many stakeholders may find it unbalanced in comparison to the resource use in the healthcare system elsewhere, e.g., medical services, hospitals, and non-pharmacological treatments such as physiotherapy. Of course, patient engagement in the Health Technology Assessment of ATMPs must be diligently prepared to ensure an unbiased, fair and patient-focused appraisal of the new technology. Putting the pricing of a one-off ATMP treatment into perspective as compared to the standard of care is also quite needed. “Creative” contracting with elements such as “pay for performance” and/or “risk-sharing agreements” can also be the subject of a dialogue with patient representatives to ensure broad societal acceptance.
ATMPs may be associated with high and sometimes unrealistic expectations of patients and their loved ones. Expectations must be managed appropriately and all those responsible for communication with the outside should be aware of that fact. If, for instance, an investor press release may leave patients under the impression that a given ATMP may be available to them personally soon, this can in turn become the basis for bitter disappointments. Such communication – if necessary for investor-relation reasons – should therefore always be accompanied by appropriate patient engagement activities that put such communication directed to a non-patient and non-medical audience into perspective.
So, in a nutshell, ATMPs require particular efforts in patient engagement to maximize the benefit to patients, the healthcare system and the manufacturers, but also to minimize the risk of avoidable delays or even failures of appropriate patient access.