In 2021 we published 22 manuscripts, across a range of therapeutic areas, including rare disease, neurology, allergy, mental health and oncology.
Sunday, 28th February
There are over 6000 rare diseases, with approximately 300 million people living with a rare disease worldwide. Many rare diseases are chronic, progressive, degenerative, disabling and life threatening. Due to the rarity of each individual disease, expertise and information is scarce. Most rare diseases have no cure and limited treatments are available.
Acaster Lloyd Consulting acknowledge the importance of research into the treatment of rare diseases and would like to help spread awareness this #rarediseaseday.
We are proud to have supported many successful submissions in rare diseases, as well as helping to find some solutions to the methodological challenges that working in rare diseases can bring. In 2020 alone, we undertook 20 projects in rare diseases. In recent years, we have conducted prospective research in a range of rare diseases, including:
- Fabry disease
- Spinal Muscular Atrophy
- X-linked hypophosphatemia
- RPE65 inherited retinal diseases
- Duchenne Muscular Dystrophy
- X-Linked Myotubular Myopathy
- Atypical Hemolytic Uremic Syndrome
- Paroxysmal nocturnal hemoglobinuria
- Metachromatic Leukodystrophy
- Familial Chylomicronemia Syndrome
- Neurofibromatosis Type 1 (NF-1)
- Short-Lasting Unilateral Neuralgiform Headache Attacks
- Hereditary Angioedema
- Acute Myeloid Leukemia
- Dravet syndrome
- Tuberous Sclerosis Complex (TSC)
- Lennox-Gastaut Syndrome
- Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
Take a look at our dissemination from 2020!
Acaster Lloyd Consulting researchers, Sarah Acaster and Katy Gallop, have been partnering with Aimmune Therapeutics psychologists, Audrey Dunn-Gavin and Rebecca Knibb, healthcare providers and patient advocacy groups across Europe for the past four years exploring the impact of Peanut Allergy on the health-related quality of life.
We are very pleased that a large-scale (> 100 interviews) pan-European qualitative study of the impact of peanut allergy on adults, teenagers, children and parent/caregivers across eight European countries was selected for a mention in the Editorial of Clinical and Experimental Allergy this month, by editor-in-chief Graham Roberts, along with a replication of the conceptual model. Furthermore, the cover of the journal was developed to reflect the content of the qualitative findings.
To compliment this qualitative work, two burden of illness surveys reporting child and caregiver health-related quality of life in the UK have also recently been published quantifying the impacts highlighted in the qualitative work with validated patient and observer-reported measures.
It’s great to see qualitative work in this field getting such recognition, and this high unmet need being recognised and addressed.
The awareness and recognition of PPD, and getting women and families access to treatment, remains critically important. Earlier this year Sarah Acaster co-authored a paper with Sage Therapeutics on the relationship between patient reported outcomes and clinician-reported outcomes used in PPD trials. This new paper applies some of these findings to support the interpretation of data from three clinical trials of brexanolone injection.
The aim was to present clinical trial data in a format more accessible or informative for real-world and shared decision making, including outcomes such as individual patient-level sustained response and number needed to treat. Similar outcomes, also exploring broader symptoms such as sleep and anxiety, have been presented throughout the year at various virtual conferences, based on the treatment’s oral formulation, so look out for more publications in the coming months.
Acaster Lloyd Consulting presentations at virtual ISPOR Europe 2020
Sarah Acaster recently co-authored a paper entitled ‘Associations between commonly used patient-reported outcome tools in postpartum depression clinical practice and the Hamilton Rating Scale for Depression’ in the Archives of Women’s Mental Health. The full text is available here.
Kate Williams and Andrew Lloyd recently published the paper ‘Health state utilities associated with caring for an individual with cutaneous T-cell lymphoma (CTCL)’ in the Journal of Medical Economics. The full text is available here.
On Monday 8th June, Associate Director Siu Hing Lo will be moderating a free ISPOR webinar entitled ‘Why and How to Use Qualitative Research Methods in Conjunction with Discrete Choice Experiments in Healthcare’. You can sign up here.
Earlier this week, Acaster Lloyd Consulting presented two posters at Virtual ISPOR 2020. These describe two preference studies in spinal muscular atrophy (SMA) led by Andrew Lloyd and Siu Hing Lo. One study examined patient and caregiver treatment preferences in SMA. The other study estimated disutilities for SMA using results from a general population discrete choice experiment. You can view copies of the posters here and here.