Commodore Paroxysmal Nocturnal Hemoglobinuria (PNH)

• Research type

  Research Study

• Full title

  COMMODORE Burden of Illness: Quantifying the socio-economic burden of Paroxysmal Nocturnal Hemoglobinuria (PNH).

• IRAS ID

  301271

• Contact name

  Alan Finnegan

• Contact email

  a.finnegan@chester.ac.uk

• Sponsor organisation

  University of Chester

• Clinicaltrials.gov Identifier

  n/a, n/a

• Duration of Study in the UK

  1 years, 4 months, 2 days

• Research summary

  In this study, the UoChester will work alongside HCD, which is a health economics agency specializing in real-world burden of illness studies in rare diseases. Burden of Illness studies involve estimating the economic impact of a particular disease (in this case Paroxysmal Nocturnal Haemoglobinuria (PNH)) on a particular society (usually a nation) in monetary terms. PNH is an ultra-rare, acquired, cloal, hematopoietic stem cell disorder caused by mutations in the phosphatidylinositol glycan class A (PIGA) gene.
  Inhibition of the complement C5 has been proven to be a successful therapeutic intervention in patients with PNH. However, access restrictions due to price remain a significant challenge in many countries. Despite these significant improvements in the treatment of PNH, there remains a high unmet medical need.
  New therapies have the potential to change how PNH is clinically treated in the future, but robust data on the ‘real life’ burden and cost of PNH is needed to understand the impact of these new therapy approaches, as well as the impact of those available currently used. Minimal published evidence exists quantifying current treatment burden and cost of illness in PNH. Therefore, there is an unmet need for examining all aspects of the PNH disease burden. To gain a patient- and caregiver-level understanding of the PNH burden, this study will conduct a cross-sectional burden of illness (BOI) study across the UK.
  The proposed PNH burden of illness study has multiple aims:
  1. Quantify the direct/indirect costs of PNH (drug and non-drug) from a healthcare system and patient perspective
  2. Demonstrate the real-life HRQoL impact of PNH for patients and highlight any unmet needs the current standard of care may not address
  3. Leverage the study to validate the QLQ-AA/PNH questionnaire

• REC name

  South Central - Berkshire Research Ethics Committee

• REC reference

  21/SC/0268

• Date of REC Opinion

  14 Sep 2021

• REC opinion

  Favourable Opinion