Haematology

Acquired thrombotic thrombocytopenic purpura (aTTP)

Expert consensus elicitation to support a product in acquired thrombotic thrombocytopenic purpura (aTTP)

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

Qualitative literature and instrument review in atypical Hemolytic Uremic Syndrome (aHUS)

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

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

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

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

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)

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

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

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

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