Patient involvement and engagement sits at the heart of the HCD Economics commitment to leadership in health economic research and analysis. This commitment is applied to addressing the needs and interests of patients, specifically in terms of supporting negotiation for access to innovative medicines and healthcare. The expression of patient needs and interests is sometimes described as ‘the patient voice’. The challenge for health economists is to adapt and employ statistically meaningful and measurable techniques, to quantify what is expressed by the patient voice into robust and comparable data, that stands as real-world evidence that market access and patient advocacy negotiators can draw upon.
The technique adopted by HCD Economics is within the discipline referred to as ‘preference research’. Preference research is a research discipline that has attracted the intense engagement of those organisations leading healthcare innovation. One of those leading organisations is the Innovative Medicines Initiative (IMI). IMI aims to boost biopharmaceutical innovation in Europe and to speed up the development of better and safer medicines for patients. Through its unique and innovative funding scheme, IMI supports research projects in the areas of safety and efficacy, knowledge management and education and training.
IMI is leading a project entitled IMI-PREFER. This project is running patient preference studies in both academic and industry settings. IMI-PREFER is a public-private collaborative research project. This means that in addition to academia and the biopharmaceutical companies, PREFER also includes patient organisations, Health Technology Assessment bodies and small and medium-sized enterprises. The project has been structured with equal leadership and participation from academia and industry in all areas of the project, exemplifying the collaborative and inclusive approach advocated by IMI.
Experience gained is intended to provide a better understanding of what will be a recommended best-practice approach to patient-preference studies. PREFER will establish recommendations to support development of guidelines for industry, Regulatory Authorities and HTA bodies on how and when to include patient perspectives on benefits and risks of medicinal products. IMI-PREFER will also show how patient preference studies can give valuable information to support decision making for regulators and HTA bodies.
One such body, the UK’s National Institute for Health and Care Excellence (NICE) has recently published a paper entitled ‘Use of Patient Preference Studies in HTA Decision Making: A NICE Perspective’.( The Patient - Patient-Centered Outcomes Research (2020) 13:145–149 https://doi.org/10.1007/s40271-019-00408-4). The authors confirm that patient preference studies could provide valuable insights to a National Institute for Health and Care Excellence committee into the preferences patients have for different treatment options, especially if the study sample is representative of the broader patient population. The authors confirm they see a role for patient preference studies to be submitted alongside other types of evidence. Examples described by the authors where patient preference studies might add value in health technology assessments include cases where two distinctly different treatment options are being compared, when patients have to decide between multiple treatment options, when technologies have important non-health benefits or when a treatment is indicated for a heterogenous population.
However, the authors do qualify that in their view, in the context of the National Institute for Health and Care Excellence’s methods and processes, they do not see a role for quantitative patient preference data to be directly incorporated into health economic modelling. However, novel methods are being explored in this area via the use of quality-of-life instruments in DCE’s to be able to incorporate this data source.
Recent research by HCD Economics has sought to employ a tri-phasic approach that results in the generation of:
- Qualitative interpretation data to elicit what is expressed by the patient voice and
- Quantitative patient preference data that is legitimate real-world evidence and informs the definition of health states applicable for HE models
The HCD Economics tri-phasic approach begins with the exploration phase. This involves the organisation of patient panels and advisory boards drawing upon appropriate academic, medical and PAG (Patient Advocacy Group) experts. This phase is a structured approach, dependant on health economic techniques, including coding of responses to generate comparative qualitative data.
The tri-phasic quantitative phase has many options in terms of study design. Discrete Choice Experiments (DCE) are one such method where analysis of DCE data can be employed to quantify the patient voice. In parallel or as an individual piece of research Time Trade Off (TTO) analysis can also be undertaken to elicit health state utility valuations.
For more information on the innovative research undertaken by HCD Economics, in the quantification of the patient voice – please contact Alison Rose (firstname.lastname@example.org)
For a one-to-one discussion on the recent patient preference research undertaken by HCD Economics, please contact patient preference lead – George Morgan (email@example.com) additionally, a video detailing what we can do with patient preference research is here