As the medicines development environment becomes progressively patient-centred, the model of co-creation and collaborative working with patients and the public (referred to as Patient and Public Involvement, or PPI) is ever more understood as an essential approach for shaping health solutions that deliver better value.
This is especially the case when it comes to clinical trial design, where inclusion of the patient voice can support clinical teams to deliver not just patient-centred but also cost-effective trials. For example, we know that new medicines are up to 19% more likely to launch when trials have been co-designed with patients, and there is now evidence to suggest that return on investment associated with involving patients in clinical development could be significant, even up to 500-fold in some cases.
Patient-reported outcomes (PROs) are an important part of this picture. Defined by the FDA, a PRO is simply “any report of the status of a patient’s health condition that comes directly from the patient, without interpretation”. While a broad definition, PROs generally provide reports from patients about their own health, quality of life or functional status associated with the healthcare or treatment they have received. PRO measures (PROMs) are the tools or instruments used to collect and report PROs, typically within a clinical trial setting.
PRO data can provide vital insights into both the patient experience and the impact of a therapeutic intervention. They help the developers of medicines and regulators to determine the benefit-risk balance through perspectives that are complimentary to (and not available in) the clinical data.
PROs and PROMs are not automatically patient-centred
However, we must consider that PROs and PROMs aren’t necessarily always patient-centred by design or synonymous with PPI in research. If PROs/PROMs are not agreed or developed in collaboration with the people that live with (or are impacted by) the condition, then they run the risk of being burdensome for patients to engage with, potentially being misrepresentative and, crucially, failing to assess the outcomes that matter most to patients.
Ensuring that PROMs are relevant and accurately capture the patient experience is critical, especially in cases where PRO data are pivotal during the analysis, review and approval of new therapeutic interventions.
For us here at Ashfield MedComms, the optimal way to achieve this, is to collaborate with patients to review, select or even co-create a meaningful PROM.
Collaborate to ensure PROs and PROMs are patient-centred
When considering the utility of PRO endpoints in a clinical trial, our suggestion is to engage with representatives of patient organisations as your advisors as early and as authentically as possible.
Patient organisations are valuable strategic partners for clinical teams; offering expertise, advice and insights that can support early decision-making. Ongoing dialogue on how to shape clinical development programmes to optimise patient experiences and outcomes is mutually beneficial ground that can be part of a constructive partnership. For PROs specifically, starting discussions before a PROM has even been considered as a good approach. Part of your discussions can be to ratify research objectives or a PRO hypothesis to help understand if your research questions are meaningful and relevant.
Once objectives have been discussed, you can begin to talk about how PROs might be measured in your trial setting in a patient-centred and respectful way, while of course remaining clinically meaningful and robust for your own drug development or licensing application purposes.
The areas to talk about with your patient advisors in order to define an appropriate PROM are broadly around relevance, suitability and practicality:
- Relevance – are the questions we are asking relevant to the patients’ health condition, and reflective of their lived experience, needs and concerns? Are they covering topics or outcomes that matter to patients themselves? Will the findings help support patient decision-making?
- Suitability – are the questions suitable for patients in the context of a clinical trial setting? Is the language and format appropriate?
- Practicality – what is the burden vs benefit of completing and using the PROM and answering the questions? What is the time requirement, and is it a realistic and respectful request?
These are all questions you can work with patient advisors to answer. And by doing so, you can assess if an existing PROM might be suitable, or if you should consider starting discussions about co-creating and validating a new one that is meaningful to patients in your particular setting. In either scenario, you can leverage many benefits. For patients, the benefits not only include improved in-trial experiences but will also will ultimately help ensure PRO data are more meaningful to others when published. For industry, further to the financial savings and practical efficiencies in running the clinical trial, there are also benefits to be found in terms of reputation and delivering clinical trials that are more reflective of patients’ needs, concerns and perspectives.
And while the co-creation of PRO endpoints and measures offers benefits, it is also becoming a requirement. The FDA has issued guidance around the use of PROs in labelling claims that also supports a co-creation approach, stating “because the purpose of a PRO measure is to capture the patient’s experience, an instrument will not be a credible measure without evidence of its usefulness from the target population of patients”.
If you want to understand more about how to shape your strategy for working with patient organisations to develop PRO endpoints or broader clinical development planning, then the Patient Engagement team at Ashfield MedComms would be delighted to speak with you to explore your needs.