What does it mean to improve outcomes for patients with chronic conditions?

Improving health outcomes for patients is the guiding principle of most drug development programmes. But who decides which outcomes need to be improved?

Through our work across chronic and rare diseases, Sprout researchers know that what constitutes a positive outcome for patients does not always align with the outcomes of interest to researchers. Improving health outcomes for people with chronic conditions needs to start at the very beginning of a drug development programme, with the selection of which outcomes to measure. After all, if a clinical trial successfully demonstrates improvement in an outcome that isn’t meaningful to patients, can we really say that the drug offers an improvement to patients’ lives?

What outcomes are important to people with chronic conditions?

In cases of acute health crises, the outcomes of importance may be quite similar for patients and researchers: treat the infection; reduce the pain; do whatever it takes for the patient to recover and get back to normal life as soon as possible. With chronic conditions, the goals and definitions of a successful outcome can be quite different. There is no returning to ‘normal life’, but instead learning how to create a new normal as someone living with a health condition.
For a clinician or researcher, achieving a good outcome for individuals often centres entirely around addressing the signs and symptoms of their health condition. For patients with chronic health conditions, their diagnosis is just one piece of who they are, another aspect of life that will need to be managed and accommodated. In this context, an improved health outcome may not centre around improving specific signs and symptoms, but instead might be oriented toward improving the ability to live a meaningful life despite the signs and symptoms of their condition.

Haemophilia: bleeds aren’t everything

An example of this comes from Sprout’s work with a global pharmaceutical company developing a novel prophylactic treatment for haemophilia, a rare genetic disorder in which blood does not clot properly, leading to spontaneous and potentially prolonged bleeding. Improved health outcomes for this condition may seem straightforward – reduce the number and severity of bleeds and patients’ health will improve. However, in our interviews with haemophilia patients we learned that having fewer bleeds was not always the most important indicator of an improved outcome.
The adult haemophilia patients we spoke with learned to live with bleeds – they knew how to treat them when they occurred and, given that there is currently no cure for haemophilia, they expected that breakthrough bleeds were an inevitable part of life. Some patients told us that a marker of good health for them was whether they could continue participating in physical activities that brought joy and meaning to their lives – even if it meant that these activities would cause additional bleeds. For these patients, being able to do what they loved and manage bleeds that would likely occur was a much better outcome than having fewer bleeds but being less physically active.

Centring the patient perspective in drug development

Ensuring that patients’ health outcomes have improved in meaningful ways requires incorporating patient voices and experiences into the beginning stages of drug development. It may seem efficient to skip this step, but at Sprout we have seen how early investment in the patient perspective saves enormous resources down the line by helping to avoid regulatory hurdles, marketing challenges or problems with patient adherence. In this case, a prophylactic medication that reduces the number of bleeds but interferes with daily life in other ways – for instance, with bothersome side effects or a time-consuming administration process – may not be worth taking from the patients’ perspective, even if it leads to better outcomes clinically.
Centring patients’ perspectives does not mean letting go of clinically meaningful outcomes but thinking carefully and critically about which outcomes should be measured and why. The qualitative clinical outcome assessment research that we do at Sprout helps sponsors grapple with these decisions, ensuring that clinical trial goals and endpoints are aligned with patients’ own definitions of what improved health looks like.