Therapeutic area

Menstruation and dysmenorrhea

  • Provision of regulatory support for use of the Pictorial Blood Loss Assessment Chart (PBAC) in women’s health
  • Development of an ePRO for adult and adolescent dysmenorrhea
  • Regulatory consultation on dysmenorrhea assessments for an FDA type C meeting
  • Endpoint strategy in dysmenorrhea


  • Local market adaption of economic models for 2nd line mesothelioma treatment
  • Consultancy and training on a shortness of breath claim in mesothelioma

Metachromatic leukodystrophy

  • Understanding and documenting the disease burden of metachromatic leukodystrophy
  • Support of patient survey study development among patients with metachromatic leukodystrophy
  • Web-based survey implementation and analytic consulting support in metachromatic leukodystrophy
  • Clinical outcome assessment strategy development and validation in metachromatic leukodystrophy

Metastatic bone disease

  • Pre-screen interviews for the relative resource utilisation of intravenous and subcutaneous drug administration in metastatic bone disease
  • Research to validate and investigate the relative resource utilisation of intravenous and subcutaneous drug administration in metastatic bone disease


  • Literature review of cardiovascular disease in migraine patients
  • Systematic literature review in migraine
  • Development of a PRO measure in chronic adolescent migraine
  • Statistical analysis plan for in-trial psychometric validation of the assessment of chronic migraine symptoms and impacts measures
  • Gap analysis and assessment of PROs in migraine
  • Development of a PRO measure in postdrome migraine
  • Development of an FDA briefing document and regulatory support in chronic migraine
  • Qualitative interviews in chronic migraine headaches
  • Gap analysis of PRO measures in paediatric migraine
  • Generation of evidence for the content validity of instruments assessing motor weakness in hemiplegic migraine
  • Review of concussion PROs relating to migraine


  • Meta-analysis of a treatment for recurrent miscarriage

Mitochondrial disease

  • PRO questionnaire development in mitochondrial myopathy
  • Cognitive debriefing interviews for the Primary Mitochondrial Disease (PMD) Symptom Assessment Questionnaire


  • Understanding the experience of the mucopolysaccharidosis I (MPS I) patient to improve outcome assessments: evidence from the literature and treatment experts
  • Cognitive debriefing interviews on the Mucopolysaccharidosis Health Assessment Questionnaire (MPS-HAQ)
  • Evaluating the MPS-HAQ for use in patient registries to support EMA regulatory commitments

Multinodal goiter

  • PRO endpoint review in multinodal goiter
  • Cognitive debriefing on quality of life in multinodal goiter
  • Qualitative data analysis and FDA support in multinodal goiter

Multiple myeloma

  • Development of a manuscript on the burden of relapse in multiple myeloma
  • Value dossier for treatment in multiple myeloma
  • Development of a conference poster on multiple myeloma
  • Research strategy and publication plan to support the burden of relapse in multiple myeloma
  • Communication of the findings from patient and physician interviews on the burden of relapse in multiple myeloma
  • Conference poster on the burden of relapse in multiple myeloma
  • Meta-analysis, mixed treatment comparison and multiparameter evidence synthesis of the efficacy of a treatment for multiple myeloma
  • Quality of life analysis of clinical trial data in multiple myeloma
  • Burden of disease deck, payer value dossier deck and frequently asked questions deck in multiple myeloma
  • European survey quantifying the impact of novel treatments on quality of life in multiple myeloma
  • Mixed treatment comparison of a treatment for multiple myeloma
  • Standalone budget impact models in multiple myeloma
  • Analysis of a treatment in multiple myeloma
  • Analysis of PROs in randomised controlled clinical trial in multiple myeloma
  • Statistical analysis of the minimally important differences and responder thresholds in myeloma
  • Sensitivity analysis in myeloma
  • PRO analysis in multiple myeloma
  • PRO analysis of clinical trials on a treatment for multiple myeloma
  • Patient interviews in multiple myeloma
  • Manuscript and publication support on the use of minimal residual disease testing by US physicians who treat patients with multiple myeloma
  • Manuscript on multiple myeloma quality of life trial results
  • Conference presentation of PRO study results in multiple myeloma
  • Poster reporting results from multiple myeloma patient interviews
  • FDA regulatory review on a treatment for multiple myeloma
  • Clinical trial analyses to support HRQoL in multiple melanoma
  • Value dossier and e-v@luate platform for a drug used in the treatment of relapsed/refractory multiple myeloma (RRMM)
  • Global value dossier and e-v@luate platform for a treatment in RRMM
  • Quality of life analysis for treatment of RRMM
  • Quality of life analysis, SAP and reporting for a treatment in RRMM
  • Full analysis and QoL endpoints in RRMM
  • German PRO analysis to support submission of a study on a biological therapy drug used in RRMM

Multiple sclerosis

  • Global value dossier update in multiple sclerosis (MS)
  • Multiple technology appraisal in MS for NICE
  • Research into UK data sources for MS
  • Delphi study (with KOL and payer interviews) to inform market access strategy in MS
  • Utility elicitation study on patients with MS
  • Validation of three cost-effectiveness and budget impact models in MS
  • Negotiation of a licensing agreement for the Multiple Sclerosis Impact Scale (MSIS-29)
  • Review and formatting of patient reported resource use, work productivity and caregiver burden in MS
  • Qualitative research to validate a screening tool for secondary progressive MS
  • PRO endpoint review in MS
  • Briefing document, advisory board and labelling strategy for the FDA on a treatment for MS
  • Cognitive debriefing UK study on walking impairment in MS
  • Psychometric testing of walking impairment in MS
  • Manuscript for the development and psychometric assessment of the Early Mobility Impairment Questionnaire (EMIQ) in MS
  • Rasch analysis of the cognitive debriefing survey data collected in a qualitative phase for MS
  • Rasch analysis and report of DSP data collected on the Full Scale Intelligence Quotient (FSIQ) measure in MS
  • PRO instrument reviews in relapsing MS and spinal cord injury
  • PRO review on MS
  • PRO statistical analysis plan for a clarity trial in MS
  • Selection of a PRO for depression in MS
  • Endpoint review of a treatment for MS
  • Concept confirmation and cognitive debriefing of spasticity PRO measures in MS patients, and development of spasticity screener
  • Development and validation of a clinical screening tool for overactive bladder in MS
  • Advisory board for a clinical screening tool for overactive bladder in MS
  • Development of a predictive calculator for a clinician-completed screening tool in secondary progressive multiple sclerosis (SPMS)
  • Psychometric validation of a clinician completed screening tool in SPMS
  • Exploratory analysis of phase II clinical trial data for acute optic neuritis

Relapsed-remitting multiple sclerosis

  • Development, content confirmation and validation of a fatigue measure in relapsing-remitting multiple sclerosis (RRMS)
  • Work on the equivalency of an e-PRO on the FSIQ in RRMS
  • Qualitative work in subgroups with RRMS
  • Development of a PRO measurement strategy in relapsing MS
  • Mode of administration equivalency and usability testing of the MSIS-29 in patients with relapsing MS
  • Development of a PRO dossier for the MSIS-29 in RRMS
  • Psychometric validation of a newly developed PRO for patients with RRMS
  • Poster development on the Fatigue Symptom and Impact Questionnaire for Relapsing Multiple Sclerosis (FSIQ-RMS)

Muscle wasting

  • COA review and development for muscle wasting associated with chronic illness
  • Exit interviews to explore patient disease burden, treatment satisfaction and clinical trial participation in muscle wasting associated with chronic obstructive pulmonary disease (COPD)
  • Qualitative research to support the identification of key impacts on physical functioning and activities of daily living in patients with muscle wasting

Musculoskeletal pain

  • Conference abstract and poster for early decision modelling in acute pain
  • Strategic literature review of chronic pain
  • Competitive assessment and burden of illness in chronic low back pain
  • Content confirmation and testing of the Roland Morris Disability Questionnaire (RMDQ) and Pain NRS Personal Digital Assistant in chronic low back pain
  • Development of a responder index in low back pain
  • Manuscript on the chronic low back pain responder index
  • Development of a PRO measure of stiffness in acute low back pain
  • Focus group interviews with patients with chronic low back pain
  • Gap analysis and strategic recommendation for PRO and market access studies in chronic low back pain and diabetic peripheral neuropathy
  • Mode of Administration (MOA) equivalency study in lower back pain
  • Consulting on PRO analysis and study design of trials in chronic low back pain
  • Burden, economics, epidemiology and treatment patterns in chronic low back pain
  • Qualitative exit interviews to assess the patient experience of treatment and clinical trial participation among patients with chronic low back pain
  • Gap analysis and review of PRO measures in low back pain and neck pain
  • Development of a briefing document on the modified Brief Pain Inventory – Short Form (mBPI-SF) in acute musculoskeletal pain
  • Critical review of PRO instruments in fatigue and pain in rheumatic disease
  • PRO literature review in pelvic pain
  • Patient perspective literature review for chronic pelvic pain in both men and women

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)


  • Global value dossier and e-v@luate platform for a treatment in myelofibrosis
  • Quality of life analysis, value proposition and value messages for a treatment in myelofibrosis
  • Modification of a web-based patient survey to assess QoL, economic impact and work productivity for patients with myeloproliferative neoplasms
  • Development and documentation of a PRO questionnaire for use among patients with myelofibrosis in second line treatment
  • Score interpretation guidance on the use of the Myelofibrosis Symptom Assessment Form (MF-SAF) diary in treatment resistant patients
  • Post-hoc analysis of PRO outcomes in a phase II clinical trial in myeloid metaplasia

Myocardial infarction

  • Literature review of the unmet need in patients with a history of myocardial infarction and high risk of developing atherothrombotic events
  • Literature review of coronary artery bypass grafting (CABG) treatment in acute myocardial infarction (AMI)
  • Validation of clinician and patient questionnaires for myocardial infarction imaging
  • Development and validation of patient and clinician tools for myocardial infarction imaging
  • Analysis and reporting of myocardial infarction imaging validation study data
  • Clinician and patient satisfaction in an observational study in myocardial infarction SPECT imaging

Nasal polyps

  • Quantitative validation of symptom scores in nasal polyposis
  • Characterisation of symptoms and impacts of severe nasal polyps

Neural tube defects

  • Manuscripts, abstracts and posters on neural tube defects
  • Top-up literature review and communications on the burden of neural tube defects to patients, carers and society
  • Review on the burden of neural tube defects to healthcare systems, patients, care-givers and society
  • Content validation of a neurogenic detrusor overactivity (NDO) diary in paediatric spina bifida patients

Neuroendocrine tumours

  • Payer value proposition, payer value dossier and cost-effectiveness model in neuroendocrine tumours (NETs)
  • Development of preference, tolerability and satisfaction PROs for patients with neuroendocrine tumours (NETs)

Neurogenic orthostatic hypotension

  • Defence of the use of the Orthostatic Hypotension Questionnaire (OHQ) to demonstrate treatment benefit and labelling claim in neurogenic orthostatic hypotension
  • Validation of the OHQ in neurogenic orthostatic hypotension using clinical trial data
  • Key opinion leader interviews on neurogenic orthostatic hypotension
  • Evaluation of the current measurement strategy in neurogenic orthostatic hypotension