Patient-reported outcomes captured electronically to improve health

My Topol fellowship problem / project:

Clinical trials often provide data only for surrogate outcomes, which have shorter timeframes than clinically meaningful endpoints. On the other hand, patient-reported outcomes are reported directly from patients about how they feel or function in relation to a health condition and its therapy (often via self-reported questionnaires). They represent what is most important to patients about their condition and treatment.

Following approval of gene therapies, long-term questions may remain unanswered and robust long-term data collection would be helpful. This is particularly pertinent for gene therapy as most therapies aim to achieve long-lasting effects and may be associated with delayed adverse effects.

Regulators may request newly- licensed gene therapy manufacturers to monitor patients for up to fifteen years. While collecting data on safety and the patient experience during this period can help address many uncertainties, the data collection infrastructure is not currently available to support this on a broad scale.

Collection of patient-reported outcomes may have the following short- and long -term benefits:

  • Supplemental efficacy and safety data to inform the risk-benefit profile over longer periods than feasible in clinical trials
  • Useful for gene therapies which are novel treatments often for rare diseases for which there may be little evidence to inform or support use
  • Improved communication between patients and clinicians, helping shared decision-making
  • Improved quality of life and reduction in hospital admissions as side effects or loss of efficacy may be picked up earlier

Over the few past years I have worked locally and regionally as a Formulary Pharmacist within North Central London, where I led on the introduction of new medicines onto the formulary. With the pandemic expediting digital transformations within healthcare and building momentum for further digital progress, I began to consider how I could build on the successes the NHS has achieved and continue to transform services. I started to look for ways I could harness my knowledge in healthcare and research with my interest in digital projects, whilst still maintaining a patient-centred approach. I spoke with clinicians within my organisation about pain points that would benefit from a digital solution and arrived on a project that fitted many different areas of interest – genomics, research and digital transformation.

I hope this fellowship will give me the opportunity to develop my experience in sustainable system improvement within digital health and explore the theory of change.