Determining outcomes pertaining to healthy ageing trials
At the recent UK SPINE conference there were a series of workshops facilitating a diverse range of discussions. One of these was on the topic of the outcome measures suitable for trials in healthy ageing. This built on the initial findings of a review undertaken by academics from Birmingham Health Partner’s Centre for Regulatory Science & Innovation, Dr Muslim Abbas Syed and Eliot Marston, who summarise the discussion below.
Introduction
The aim of the workshop was to explore and capture diverse perspectives from participants across a number of areas, including: the role of stakeholder groups; outcomes considered most important; challenges associated with measuring outcomes; and strategies for consensus and development of a core outcomes set for healthy ageing trials testing drugs and devices interventions.
Role of stakeholder groups
The participants that attended the workshop had diverse professional backgrounds which ranged from investors, representatives of pharmaceutical industry, social scientists, biologists, patient/public involvement and policy makers. The experience of participants pertaining to healthy ageing trials ranged from 1 to 5 years. A central finding of the workshop was the acknowledgement that while academia and the pharma industry commonly take the lead in healthy ageing trials, a larger number of stakeholders should be involved.
Important outcomes to consider in trials
Various outcomes were identified as important while testing drugs and devices interventions for healthy ageing trials. These included: mobility; autonomy; quality of life measures; diagnostic (lab tests and biomarkers); and assessment of physiological indicators.
Challenges associated with measuring outcomes
Recruitment of participants and the confounding effects of multi-morbidity in older adults were identified as key challenges of measuring outcomes in healthy ageing trials. In particular, recruiting cohorts which are representative of the general population, including (but not limited to) ethnic minority groups at greater risk of age-related diseases. Statistical powering of studies was a major hurdle to address. Retention of participants and providing ongoing support were also critical factors for consideration. Since elderly patients generally have multiple morbidities, polypharmacy and associated side effects of existing drug prescriptions was also mentioned as an important issue while measuring outcomes while testing other specific drug interventions.
Suggested strategies for establishing core outcomes
Different strategies were suggested for establishing core outcomes set for testing drugs and devices interventions for healthy ageing trials. An evidence-based discussion based on previous trials and related literature among key stakeholders (including academics, industry, clinicians and PPI/E representatives) as a starting point in building consensus was highlighted by participants. It was emphasized that ‘subject matter experts’ (stakeholders with relevant and substantial experience in healthy ageing trials) should be included in these discussions, but that future design should be informed by wide consultation, particularly representative of the target population for healthy ageing trials.trials. At later stages of development, focus group discussions and Delphi approaches were suggested for refinement of a mutually-agreed core outcomes set.
Conclusion
The workshop provided an important platform to garner a range of perspectives on the considerations around the use of outcome measures in clinical trials for healthy ageing. It is critical that such discussions are had in order to progress this field and provide practical answers to how trials of this type are designed and structured.