Gene therapy and advanced cell-based therapies offer patients hope and the potential to make them ‘chronically well’. Long-term monitoring of patients to collect outcome data after treatment with these therapies will be standard practice for the foreseeable future; it will therefore be necessary for bio-pharma and health system stakeholders to find effective ways to develop sustainable data capture programmes that meet regulatory requirements and demonstrate value. In this article, we explore several possible ways of supporting the unprecedented need for high-quality and sustained data capture.
Bio-pharma are required to adhere to strict regulations set by the FDA and EMA on monitoring long-term outcomes when launching advanced therapeutic medicinal products (ATMPs). They request that large cohorts of patients are followed up for many years to assess long-term safety and efficacy. In addition, any novel pricing mechanism to support the reimbursement of these ‘one- or few-time’ treatments will require that processes to ensure that robust generation of outcome data are achievable in the long term. These requirements are truly unprecedented, with existing registry models and methodologies unlikely to offer sustainable and cost-effective solutions to these challenges.
Ergo, new models need to be identified. However, to truly meet the needs of all stakeholders involved, it is important to first recognise some of the specific challenges that may be encountered when generating long-term outcomes data.
From a patient perspective, and if ATMPs fulfil their promise, post-treatment disease symptoms may be considerably reduced or even non-existent. Therefore, ongoing contact between patients and their healthcare team may become limited, and this could be a key factor that leads to patients being lost to follow up. Add to this the likely scenario of changes in the healthcare setting (e.g. from a treatment initiation centre back to the referring physician) and significant life changes (e.g. from adolescence into adulthood), monitoring patient outcomes may become increasingly more difficult.
From a health system perspective, it is likely that ATMPs will continue to be introduced in a small number of specialised treatment centres. Connectivity between healthcare professionals (HCPs) across the referral and treatment pathway could be complex, responsibilities for data input may not be clear, and governance arrangements for transferring data between settings are also likely to be complicated. This means that it will be challenging to work with all HCPs and centres involved in a patient’s ongoing care from a logistical and cost perspective.
So, there are multiple challenges involved in monitoring long-term outcomes where there is little precedent. Solutions need to be well-thought-out, multi-functional and innovative to have any chance of success. We recommend using the following strategic framework to build an effective solution: