Haematology

Other haematology experience

  • Preparation of three core European value dossiers (for use by affiliates and targeted to regional/local decision makers in key markets) and three value stories in haematology
  • Manuscript on a budget impact model for a biosimilar in haematology
  • Revisions to the Lymphoedema Quality of Life Questionnaire (LYMQOL)
  • EMEA training on PROs in haematology

Myelodysplastic syndrome

  • Value dossier and e-v@luate platform in a treatment for low-risk myelodysplastic syndrome
  • Value dossier and e-v@luate platform in a treatment for high-risk myelodysplastic syndrome
  • Resource utilisation in myelodysplastic syndromes
  • Quality of life analysis for a treatment in myelodysplastic syndrome
  • Patient survey of the indirect costs and quality of life impacts in myelodysplastic syndromes
  • Qualitative interviews with patients with myelodysplastic syndrome
  • In-trial analyses of treatments in myelodysplastic syndrome (TASTE-PRO)
  • Manuscript support in myelodysplastic syndrome (TASTE-PRO)

Severe aplastic anaemia

  • Caregiver interviews to support content validity of an ObsRO in a paediatric population with severe aplastic anemia
  • Palatability and acceptability of a treatment in paediatric populations with severe aplastic anemia
  • Development of a scoring algorithm for an acceptability and palatability questionnaire in severe aplastic anemia

Von Willebrand disease

  • Development and validation of a PRO measure in von Willebrand disease and haematology
  • Pre-validation work to inform a clinical trial in von Willebrand disease
  • In-trial validation of a PRO instrument in von Willebrand disease

Sickle cell disease

  • Development of a PRO questionnaire in sickle cell disease
  • Exit interviews in sickle cell disease

Immune thrombocytopenic purpura

  • Analysis and reporting on immune thrombocytopenic purpura
  • Caregiver interviews to support content validity of an ObsRO in a paediatric population with chronic immune thrombocytopenic purpura
  • Palatability and acceptability of a treatment in paediatric populations with chronic immune thrombocytopenic purpura
  • Development of a scoring algorithm for an acceptability and palatability questionnaire in chronic immune thrombocytopenic purpura
  • Literature review and qualitative research to support the content validation of the immune thrombocytopenia quality of life index

Haemochromatosis (Iron overload)

  • Consulting on a manuscript on a hereditary haemochromatosis and phlebotomy PRO and market research
  • FDA memo for the development and validation of a screening tool to identify patients with haemochromatosis
  • Utility study for treating for iron overload
  • Submission to the All Wales Medicines Strategy Group (AWMSG) for a treatment for chronic iron overload
  • Submission to SMC for a treatment for chronic iron overload
  • Manuscript on the utility of oral versus subcutaneous iron chelation therapy
  • High level consulting: PRO strategy for reimbursement packages in Europe for iron overload
  • Clinician and patient interviews for a PRO in iron overload
  • Abstacts, posters, analyses, report and manuscript on iron overload
  • High level consulting: PRO strategy for reimbursement packages in iron overload for Italy and France, using satisfaction and self-esteem data
  • PRO analyses and validation manuscript in iron overload
  • Development of a PRO augmentation and manuscript in iron overload
  • Analysis and communications of PRO data from a clinical trial in iron-overload patients receiving iron chelation therapy
  • Communication of PRO data from a clinical trial in iron-overload patients receiving iron chelation therapy

Thalassemia

  • Conference poster on the utility of oral versus subcutaneous iron chelation therapy for beta thalassaemia
  • In-trial analyses of two treatments in thalassemia (TASTE-PRO)
  • Manuscript support in thalassemia (TASTE-PRO)
  • Modification of a satisfaction questionnaire for patients with thalassaemia receiving phlebotomy or oral therapy
  • Clinical Trial analysis of PRO questionnaires in thalassaemia patients

Eosinophilic gastroenteritis

  • Evaluation of COA tools in eosinophilic gastroenteritis (EGE) and eosinophilic gastritis (EG) with additional longitudinal data collection

Anaemia

  • NICE consultancy for a treatment for chemotherapy-induced anaemia
  • Statistical pharmacoeconomic analysis of patient level data for a treatment for anaemia following chemotherapy
  • Linking a cost utility assessment model with a budget impact model for treatment for anaemia in cancer
  • Phase II cognitive interviews and regulatory support in inflammatory anaemia in cancer
  • Evaluation of the PRO landscape using a systematic literature review on chemo-induced anaemia in oncology
  • Patient consultations on anaemia
  • General consulting agreement for iron treatment in anaemia

Haemophilia

  • Literature review and report on haemophilia B
  • Literature review in haemophilia A
  • Value messages and payer testing for treatment of haemophilia A
  • Budget impact model for haemophilia in Japan
  • Consultancy support for a PRO strategy in haemophilia
  • Critical review of PROs to inform a PRO Strategy for anti–tissue factor pathway inhibitor (Anti-TFPI) in haemophilia A
  • Exit interviews to assess the impact of infusion frequency in haemophilia A
  • Consulting work in haemophilia
  • Systematic review of haemophilia treatments
  • IRB submission for haemophilia patient interviews
  • Development of a compliance questionnaire in haemophilia
  • Development of a novel tool to evaluate patient preference for a reconstitution device for haemophilia A in a real-world setting
  • Testing of a web-based tool to evaluate patient preference for a reconstitution device for haemophilia A in a real-world setting
  • Development of a content-valid life interference questionnaire for haemophilia
  • Patient expert advice meetings – mode of action (MOA) equivalency for hemophilia bleed and infusion diary
  • Patient expert advice meetings – usability for hemophilia bleed and infusion diary