Siu Hing Lo, Natalia Piglowska and Andrew Lloyd recently co-authored the paper ‘Exploring the burden of X-linked hypophosphatemia: a European multi-country qualitative study’ in Quality of Life Research. The full text is available here.
This morning Andrew Lloyd is presenting a talk entitled ‘Measuring children’s health and HRQL with EQ-5D-Y: Values, scores and decisions’ at the 4th EuroQol Academy meeting in Prague. Please get in touch if you would like further information.
The SMC have recently approved Luxturna (a gene therapy for a rare type of inherited retinal dystrophy) and Crysvita (a treatment for X-linked hypophosphataemia which is a rare skeletal disorder). Both submissions highlight the challenges in understanding the impact of very rare conditions on patients’ health related quality of life (HRQL). Acaster Lloyd Consulting Ltd worked with both of these companies to capture data regarding the impact of the disease and treatment on HRQL; data which was used to inform cost effectiveness analyses. In their review of both treatments the SMC highlighted uncertainty regarding the HRQL data because the methods relied in part upon expert opinion. We are keen to highlight this in order to illustrate the challenges we face in capturing HRQL data in rare diseases. The treatments will be available for use in Scotland for 3 years while the companies collect more data on the impact of the treatments on patients and carers.
Decision making regarding the adoption of very high price, potentially very high value treatments is probably the most challenging area of Health Technology Assessment. Read more about the news from the SMC here.
Our director Andrew Lloyd recently wrote a commentary entitled “Cognitive decline may not be adequately captured in economic evaluations of multiple sclerosis: are new treatments being undervalued?” in Neurology. The full text is available here.
Our director Andrew Lloyd and senior research manager Katy Gallop recently co-authored the paper “Estimation of the quality of life benefits associated with treatment for spinal muscular atrophy” in ClinicoEconomics and Outcomes Research. The full text is available here.
Our director Sarah Acaster led a study team to develop the first cystic fibrosis disease specific preference-based scoring algorithm based on the Cystic Fibrosis Quality of Life – Revised Questionnaire (CFQ-R). If you’re at the North American Cystic Fibrosis Conference (NACFC) this week you can be the first to see the results of this work in Poster Session 1, tomorrow at 11:15 – 1:45: ‘Development of the CFQ-R preference-based scoring algorithm’.
Our director Andrew Lloyd and research associate Natalia Piglowska recently co-authored a paper entitled ‘Estimation of impact of RPE65-mediated inherited retinal disease on quality of life and the potential benefits of gene therapy’ in the British Journal of Ophthalmology.
We are pleased to announce that our director Andrew Lloyd was co-author on a poster entitled ‘Can the EQ-5D capture health outcomes in degenerative rare diseases? An international study’ which received the Lee B Lusted award for Patient and Stakeholder Preferences at the Society for Medical Decision Making last week. Please contact us if you would like a copy of the poster.
The Acaster Lloyd Consulting team will be at ISOQOL in San Diego from 20th to 23rd October. Please get in touch with Sarah Acaster (email@example.com) or Kate Williams (firstname.lastname@example.org) if you’d like to set up a meeting, or come to one of our presentations:
Sarah Acaster: ‘Health-related quality of life associated with Hamilton Rating Scale for Depression remission and response in postpartum depression’
Kate Williams: ‘The Rett Syndrome Burden of Illness Survey: Results from Cognitive Debriefing Interviews’
Kate Williams will be giving a talk entitled ‘Engagement with caregivers during the development and implementation of a survey to evaluate the impact of a rare neurodevelopmental disorder’ as part of Symposium 6 on Wednesday 23rd October at 8.30am-9.45am.
Our team, including Director Sarah Acaster and Senior Research Manager Katy Gallop recently conducted a large European study exploring the psychosocial impact of living with peanut allergy. The results relating to Germany and the UK will be presented in the following upcoming conferences: “APPEAL (Allergy to Peanuts ImPacting Emotions and Life) 1 and 2: Results on Peanut Allergy Impact on Allergic Individuals, Parents and Caregivers in Germany” will be presented as an eposter on Friday 27th September. The UK results will be presented in the poster session at BSACI on Friday 4th October.