Josie Godfrey hosted the webinar and introduced the presentations, all of which presented on the work undertaken by panellists, on economic research which places the patient at the heart of the research questions investigated. Josie commenced the presentations with an overview of ‘Project Hercules’ a Duchenne study undertaken collaboratively and led by a steering group of more than 60 experts, HCD Economics is delighted to be working with Project Hercules, which Josie confirms has achieved success, leveraging patient leadership, collaboration by research partners, good governance, communication and high standards. A large number of patient organisations participated in the webinar.
Juliet followed Josie, with an overview on the key modelling challenges in rare disease. Overcoming these challenges is driven by the primary goal of maximising probability of HTA recommendation. Challenges include defining health states, order of health states, the natural history of disease, health state costs, health state utilities and impact of treatment in rare disease. Solutions to these challenges include securing input from patients, caregivers and early investment in data collection and study supervision with expert involvement. Collaboration as was experienced in Juliet’s involvement in Project Hercules is the key to a successful approach. Juliet continued with a detailed explanation of the development of the disease-level economic model.
Keith covered developing natural history models (NHMs), drawing upon his involvement with Project Hercules. He explained that NHMs inform planning of services, understanding disease trajectory, design of trials. The importance of model structure, data sources and analysis as key stages was outlined and the Project Hercules model was shared in schematic form demonstrating the triangulation approach used in its development. Data sources included US based Critical Path Institute, an elicitation exercise and life expectancy as reported in studies (informed by systematic review and pooling from 14 registry studies).
Jill covered the stages for development of a Patient Reported Outcomes instrument (PRO), illustrated with the context of the PRO developed for use in Project Hercules. The first four development stages include ‘Evaluation’ (need for and relevance of existing PROs), Developing the draft PRO, ‘Refining the draft in discussion with a representative sample and ‘Validation’ employing survey techniques’. John followed to fully explain the fifth stage of PRO development ‘producing preference based scoring for the PRO. He explained the application of preference based scores to inform the credibility of HTA submissions and optimising HTA review.
Jamie commented on the privilege for HCD Economics of collaborating with Project Hercules. The HCD Economics collaboration with University of Chester was outlined, along with the experience the 47 strong team has gained in rare disease, and the critical aspect in our work of collaboration with the patient communities in those diseases researched. The study design and typical variables for HCD’s burden of illness (BOI) research was outlined, this design is now being utilised in nine countries for Project Hercules. Publications from earlier BOI studies (CHESS/Haemophilia and GAIN/NASH) were shared as examples of earlier peer review recognition of the HCD Economics methodology.
Mark talked about his organisations work in the area of digital patient registries. Earlier examples in rare disease were shared (Myasthenia gravis and Charcot-Marie Tooth disease) to explain the granularity of data captured. Best practice standards include patient-centricity, security and information governance, transparency/multi-stakeholder governance and the publication of results.
Discussion covered opportunities for access to Digital Patient Registry data, the relationship between assumptions and reported data in modelling, and its relevance for HTA decisions, now and in the future. Jamie confirmed modelling in rare diseases is inevitably pragmatic. Pragmatism also extends to the use and acceptance of ‘vignette’ studies in quality of life evidence, subject to limitations.
The panel confirmed the inestimable value that an engaged patient group has in strengthening the value of the study design and implementation. Jamie also described the importance for patient groups of utilising results to strengthen their advocacy work. Duchenne UK endorsed the importance of PPIE. In response to a question, the panel confirmed the time invested in collaboration is more than justified in terms of the outcome achieved given the data the programmes deliver for the rare disease community.. In conclusion ‘the patient must be embedded in this type of research’, dialogue, including feedback is vital and time must be invested to allow that dialogue.
The webinar was a valuable contribution to the wider understanding of socioeconomic research in rare disease. The participants represent the leadership in this field. The programme introduced a new approach in which multiple companies collaborate to develop a core economic model and common data set. This approach aims to optimise HTA and reimbursement outcomes for all treatments in a rare disease.
The webinar was designed to address the challenge of developing an economic model in rare diseases, recognising the difficulty of fully capturing the effects of the disease and the benefits of treatment whilst using sufficiently robust data to minimise uncertainty. A question on patient literacy (in context of possible bias in patient reported data on health status) was addressed by Jamie, including the role of recent paradox research in understanding patient self-assessment as reported in BOI studies, specifically under-estimation of impact upon impaired quality of life, https://onlinelibrary.wiley.com/doi/full/10.1111/hae.14278
HCD Economics has made a strategic commitment to invest its human and financial resources to improve the social and economic outcome of rare disease patients and the wider rare disease community. Partnerships have been established with a number of rare disease patient groups and at the heart of this strategic commitment is HCD Economics adherence to the principle of patient and public involvement and engagement (PPIE).
For more information on our participation in the webinar, our economic modelling experience and partnerships and our commitment to rare disease, please contact email@example.com