Experience

Growth hormone deficiency

• Strategic literature review to select PRO for a paediatric growth hormone deficiency trial
• Establishing support of content validity for the modified Injection Pen Assessment Questionnaire (IPAQ) in growth hormone deficiency
• Injection Pen Assessment Questionnaire (IPAQ) field study in growth hormone deficiency

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

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

Head and neck cancer

• Adaptation of a multi-indication value-based pricing model in head and neck cancer to China
• Health economic advisory board on a treatment for head and neck cancer
• Payer tools implementation guide in head & neck cancer
• Payer value proposition deck in 2nd line head & neck cancer
• Payer communications strategy and tools in head and neck cancer
• Payer communication tool and summary in SCCHN
• Burden of disease deck, payer value dossier deck and frequently asked questions deck in 2nd line head and neck cancer
• One-pager on HPV in head and neck cancer
• Market access and pricing tools to support a value proposition in squamous cell carcinoma of the head and neck (SCCHN)
• Market access payer value tool in head and neck cancer
• Economic value story in SCCHN
• Standardization of economic models in head and neck cancer
• Value-based model in head and neck cancer
• Multi-indication budget impact model in head and neck cancer
• Cost-effectiveness model in head and neck cancer
• Global model update and country adaptations for a multi-indication value-based pricing (VBP) model on head and neck cancer
• Dossier for a chemotherapy drug for head and neck cancer
• Reimbursement dossiers in subgroups of head and neck cancer
• Mini literature review in SCCHN
• Literature review and payer communication tools in SCCHN
• Development of an evidence compendium for HPV-related head and neck cancers
• One-pager on HPV-related head and neck cancer
• Update to a Belgian value-based pricing model in head and neck cancer
• Updates to a Health Impact Projection (HIP) model in recurrent or metastatic head and neck cancer
• Update of market access tools in SCCHN
• Updates to a global value dossier from a targeted literature review in HPV-related head and neck cancer
• Clinical trial analyses to support health-related quality of life in head and neck cancer
• Qualitative interviews to assess equivalence of paper and electronically administered questionnaires in head and neck cancer
• SMC submission of a chemotherapy drug in head and neck cancer
• Two manuscripts and one mini report for the head and neck cancer models for the UK
• US costing for a head and neck cancer model
• Health-related quality of life analyses to support a treatment for head and neck cancer: a Q-TWiST analysis
• AMNOG and ad hoc analyses to support health-related quality of life in SCCHN

Heart disease

• Global value dossier on cardiovascular disease and dyslipidaemia
• Literature review of cardiovascular disease in migraine patients
• Update of a report on the impact of changing total cholesterol targets in the General Medical Services (GMS) contract for heart disease
• NHS and public health impact of revising General Medical Services (GMS) targets for cholesterol in coronary heart disease
• Manuscript on the cost-effectiveness and budget impact of a statin treatment for coronary heart disease
• Targeted literature review and value messages for cardiovascular disease
• Investigator training and analysis of QoL data in heart disease
• Analysis of the prevalence of bradycardia and co-morbidities in coronary artery disease
• Data management and reporting of an outcomes project on a statin drug for the treatment of heart disease
• Analysis of the SF-ESTQ in the JEWEL studies in heart disease
• PRO questionnaire development for statin intolerance
• Demonstrating the value of a transcatheter heart valve in aortic stenosis
• Report on the cost of diagnosing vascular diseases with an MRA as the primary imaging modality
• Consultation on an epidemiological quality of life study in cardiovascular disease
• Development of a PRO best practices document in cardiovascular diseases – interactions with international regulatory and HTA agencies

Heart failure and transthyretin amyloid cardiomyopathy (ATTR-CM)

• Payer value proposition and communication for a new remote patient monitoring platform in congestive heart failure
• Objection handlers in transthyretin amyloid cardiomyopathy (ATTR-CM)
• Objection handler and value story updates in ATTR-CM
• Payer research/interviews and value proposition testing in ATTR-CM
• Disease area review, burden of illness and unmet needs in heart failure (and associated renal dysfunction)
• Development of a PRO dossier for the Minnesota Living with Heart Failure Questionnaire and selection of a validated dyspnoea questionnaire
• Content validity of a symptom endpoint and dossier in heart failure with preserved ejection fraction
• Consultation on the validation of a primary clinical endpoint in heart failure
• Chart review of heart failure in China
• Meetings for a heart failure study
• Heart failure chart review in China
• Caregiver burden in patients with chronic heart failure
• Psychometric validation of the chronic heart failure (CHF) caregiver burden questionnaire
• Support for additional translations of the Heart Failure Compliance Questionnaire (HFCQ) for chronic heart failure
• Network meta-analysis of pharmacological treatment of heart failure
• Selection of a caregiver burden questionnaire for acute decompensated heart failure

Hepatitis

Hepatitis A

• Update to payer value deck in Hepatitis A
• Payer material development for vaccines in hepatitis A
• e-v@luate platform on vaccines for hepatitis A
• Burden of disease literature review on hepatitis A in the UK and Ireland

Hepatitis B

• Systematic literature review of a treatment for chronic hepatitis B
• Preparation of a NICE submission for a hepatitis B drug
• SMC submission for a treatment for chronic hepatitis B
• Database analysis to calculate the economic burden of hepatitis B in the UK

Hepatitis C

• Therapeutic area strategy analysis and reporting in chronic hepatitis C virus
• Biomarkers in the treatment of hepatitis C virus infected patients – personalised medicine
• Maximizing the use of a treatment for hepatitis C-infected patients through personalised medicine – extending the tool to additional treatments
• Treatment algorithm for hepatitis C treatment
• One-pager value communication tool for Hepatitis C
• Comprehensive literature research in chronic hepatitis C in Asian countries
• Database development and analysis in hepatitis C
• Concept elicitation and cognitive debriefing interviews with hepatitis C patients in multiple countries
• Development of an EMA summary document for several instruments used in hepatitis C trials
• Development of a PRO outcomes summary document and communications support in hepatitis C trials
• Description, interpretation and analysis of pooled data related to the use of several questionnaires, scales and screening tests in three hepatitis C clinical trials
• Conceptual model for patients with hepatitis C
• Psychometric validation of a hepatitis C symptom checklist using phase III trial data
• Treatment patterns, healthcare utilization and costs associated with the hepatitis C virus in Japan
• Selection of PRO measures for hepatitis C trials

Hepatology

• Value dossier on a treatment for post liver transplant in paediatric patients
• Development of an economic model for treatment in post-liver transplant in paediatric patients
• Structured literature reviews on the burden of disease of nonalcoholic steatohepatitis (NASH)
• Strategic review and recommendations for paediatric endpoints to assess pruritis in colostatic liver disease
• Written critique of hepatology
• Measures for use in patients with autosomal dominant polycystic kidney and liver disease: development and validation of a symptom measure and validation of a health survey

Hereditary angioedema

• Development of the evidence base regarding treatment patterns, cost and burden of disease for patients with hereditary angioedema to support the use of a new treatment
• Communications package in hereditary angioedema
• Clinical endpoint review, recommendations and update of an FDA submission package in hereditary angioedema
• Hereditary angioedema clinical trial endpoint review, recommendations and FDA PRO submission package
• Pooled clinical trial validation analysis of a PRO primary endpoint in hereditary angioedema to include in FDA submission package
• FDA meeting support for a hereditary angioedema submission
• Evaluation of market research reports in hereditary angioedema to support PRO submission package
• Analysis of Visual Analogue Scale (VAS) summary scores with Last Observation Carried Forward (LOCF) in hereditary angioedema
• Psychometric validation analysis of VAS summary scores in hereditary angioedema
• Patient interviews for the face and content validation of the Patient Global Impression in hereditary angioedema
• Psychometric validation of the 6-item and 8-item composite severity scores in hereditary angioedema
• Consulting on the linguistic validation of a hereditary angioedema questionnaire into French, German and Portuguese
• Cognitive debriefing of a questionnaire in hereditary angioedema patients
• Validation and modifications to a questionnaire in hereditary angioedema patients
• Consultancy services for linguistic validation of a hereditary angioedema diary
• Publication strategy to present the results of studies in hereditary angioedema
• Patient interviews to support a PRO submission package in hereditary angioedema
• PRO briefing document in hereditary angioedema
• Strategic consulting and analysis in hereditary angioedema
• Re-analysis of patient composite score in hereditary angioedema
• Training materials for a PRO questionnaire in hereditary angioedema
• Publication strategy on hereditary angioedema

Herpes zoster (shingles)

• Payer communications on herpes zoster
• Payer communications on a herpes zoster vaccine
• A review of HTA guidance in nine countries to determine requirements for indirect costs in herpes zoster
• Review of cost-effectiveness studies in post-herpetic neuralgia (PHN)
• PRO literature review, instrumentation, gap analysis and recommendations in herpes zoster and post-herpetic neuralgia (PHN)
• Development of a conference abstract and poster on herpes zoster and post-herpetic neuralgia
• Development of two manuscripts on a herpes zoster study
• Literature review investigating the burden of disease of herpes zoster and post-herpetic neuralgia in Europe
• Systematic literature review on the caregiver burden in herpes zoster (shingles) and post-herpetic neuralgia
• Searches to update a critical review of evidence documenting the humanistic, economic and societal burden of herpes zoster in Europe
• Research study on the impact on quality of life in herpes zoster
• Systematic literature review report on herpes zoster
• Validation of the Numerical Rating Scale (NRS) in post-herpetic neuralgia
• Payer material development for vaccines in shingles
• e-v@luate platform on vaccines for shingles

Hidradenitis suppurativa

• Core value dossier to support new treatment in hidradenitis suppurativa
• Update to a value proposition deck with adolescent data in hidradentis suppurativa
• Core value dossier, payer value proposition and objection handler update in hidradenitis suppurativa
• Structured literature review and value dossier updates in a biosimilar for hidradenitis suppurativa
• Payer value deck for a biosimilar in hidradenitis suppurativa
• Development and validation of a PRO tool in hidradenitis suppurativa
• Measurement of pain in hidradenitis suppurativa
• Development of a PRO in hidradenitis suppurativa
• Development of an investigator training manual in hidradenitis suppurativa
• Development of FDA response on phase II hidradenitis suppurativa study protocol
• Poster for a PRO in hidradenitis suppurativa
• Mechanism of Action (MOA) Equivalency Testing in hidradenitis suppurativa
• Clinical endpoint guidance and observational study in hidradenitis suppurativa
• ClinRO inter-rater reliability study in hidradenitis suppurtiva
• Design, placement and placement of outcomes in registry for hidradenitis suppurativa
• HEOR strategic consulting in hidradenitis suppurativa
• Pre- and post-surgery research study in hidradenitis suppurativa
• Development of three measurement dossiers in hidradenitis suppurativa to support regulatory submissions
• Psychometric testing of the Hidradenitis Suppurativa Impact Assessment (HSIA) and Hidradenitis Suppurativa Symptom Assessment (HSSA)
• Summary report and publications for hidradenitis suppurativa pain and relevant tools
• Evaluating the clinical meaning of defining treatment response based on the Hidradenitis Suppurativa Clinical Response (HiSCR)
• Statistical analyses of registry data (UNITE) in hidradenitis suppurativa
• Literature review on hidradenitis suppurativa

Homocystinuria

• Health economic analysis for SMC resubmission in homocystinuria

HPV vaccines

• Value pack for a HPV vaccine
• Payer value deck update for HPV
• Literature review of epidemiology and economic data in HPV
• Systematic literature review on the indirect protection from the HPV vaccination
• HTA submission for a HPV vaccine
• Cost-effectiveness analysis of a HPV vaccine for Spain
• Manuscripts on the German and Spanish cost-effectiveness analysis of a HPV vaccine
• Brief adaptation of a Markov model for a HPV vaccine
• Update of a budget impact model for a HPV vaccine
• Analysis and reporting on the budget impact of a HPV vaccine
• Training sales reps on an interactive model for a HPV vaccine
• Update to European one-pager value communication tool for a nine-valent HPV vaccine
• One-pager communication tool on cross-protection in HPV
• Revision of one-pagers in HPV vaccines
• e-v@luate platform for HPV vaccines
• Evidence package (literature review and value messages) for HPV vaccination
• Development of a rationale for the need for a HPV vaccine
• Development of an evidence compendium for HPV-related head and neck cancers
• One-pager value communication on discounting approaches to HPV vaccination
• One-pager on HPV-related head and neck cancer
• Global value dossier modules for HPV vaccination cohorts
• Anthropological study on the impact of HPV-related diseases
• Manuscript on a HPV vaccine acceptance study in middle aged persons
• HPV vaccination impact model in infectious diseases
• HPV dossiers workshops and value message development
• Targeted literature review on a HPV vaccine to support development of a payer value deck and e-v@luate platform
• Value communications development and strategic consultancy for HPV communication strategy
• Value communications support and manuscript on the burden of HPV-related diseases in men globally
• Workshop on an alternative dosing schedule in HPV
• Global value dossier on an alternative dosing schedule in HPV

Human immunodeficiency virus (HIV) / Acquired immune deficiency syndrome (AIDS)

• Business planning toolkit in HIV
• Development of a payer value story in HIV
• Payer value story briefing document in HIV
• Review of an economic model for HIV and submission to the SMC
• Budget impact model in HIV
• Targeted literature review of adherence in HIV
• Targeted literature review in HIV and inflammation
• Targeted literature synthesis of viral blips and viremia in patients with HIV
• Literature review on inflammation in patients with HIV
• Consulting in a multinational case-control study: Impact of side effects on the quality of life (QoL) of HIV patients
• Advisory board consultation regarding product status and development in HIV
• General consultancy on PRO endpoints in HIV/AIDS trials
• Recommendations for the inclusion of PROs into HIV trials
• Development of an expert report and FDA meeting on tolerability in HIV
• Consulting on the development of a PRO strategy for international HIV trials with interviews in the US and Puerto Rico
• Consulting on study documents and development of SAP for psychometric validation of a PRO instrument of tolerability in patients with HIV
• Preparation and development of a manuscript on HIV Patient Symptom Profile (HIV PSP)
• Preliminary psychometric validation of the HIV PSP questionnaire using the UK SWITCH study data
• Analysis of HIV PSP data from the SWITCH study to compare treatment arms
• Confirming concept definitions of the HIV Treatment Tolerability Index for translation purposes
• An evidence synthesis on the efficacy and safety of two drugs and protease inhibitors in treatment‑naïve HIV patients
• Manuscript network meta-analysis of an antiretroviral drug for the treatment of HIV
• Budget impact tool for an antiretroviral drug for treatment-naive HIV
• Literature review of the epidemiology and burden of illness in HIV-related neuropathy
• Top-up literature review, revised manuscript and presentations on HIV and AIDS
• Development and psychometric validation of a PRO instrument of tolerability in patients with HIV
• Two abstracts, two posters and a manuscript on the development and psychometric validation of a PRO instrument of tolerability in patients with HIV
• Selection of PRO measures for HIV trials

Human papillomavirus (HPV)

• Value cards for human papillomavirus (HPV)
• Payer value deck and frequently asked questions for HPV and HPV-related diseases
• Value pack for a HPV vaccine
• Brief adaptation of a Markov model for a HPV vaccine
• Update of a budget impact model for a HPV vaccine
• Cost-effectiveness analysis of a HPV vaccine for Spain
• HPV vaccination impact model in infectious diseases
• HTA submission for a HPV vaccine
• Manuscript on the German cost-effectiveness analysis of a HPV vaccine
• Manuscript on the Spanish cost-effectiveness analysis of a HPV vaccine
• Systematic literature review on the indirect protection from the HPV vaccination
• Value demonstration and implementation plan for a new HPV test
• Training sales reps on an interactive model for a HPV vaccine
• Analysis and reporting on the budget impact of a HPV vaccine
• One-pager communication tool on cross-protection in HPV
• One-pager on HPV in head and neck cancer
• Revision of one-pagers in HPV vaccines
• Literature review of epidemiology and economic data in HPV
• Update to European one-pager value communication tools for HPV and for a nine-valent HPV vaccine
• e-v@luate platform for HPV vaccines
• Update to a payer value deck in HPV infection
• Evidence package (literature review and value messages) for HPV vaccination
• Adaptation of a Markov model on a HPV vaccine to Belgium
• Development of a rationale for the need for a HPV vaccine
• Development of an evidence compendium for HPV-related head and neck cancers
• One-pager value communication on discounting approaches to HPV vaccination
• Global value dossier modules for HPV vaccination cohorts
• Anthropological study on the impact of HPV-related diseases
• Manuscript on a HPV vaccine acceptance study in middle aged persons
• Value communications for HPV immunisation data systems
• Targeted literature review on a HPV vaccine to support development of a payer value deck and e-v@luate platform
• Value communications development and strategic consultancy for HPV communication strategy
• Value communications support and manuscript on the burden of HPV-related diseases in men globally
• Global value dossier on an alternative dosing schedule in HPV
• Workshop on an alternative dosing schedule in HPV
• HPV dossiers workshops and value message development
• One-pager on HPV-related head and neck cancer
• Development of a PRO strategy in cervical cancer and HPV
• Summary report, top-up literature review, meta-analysis, editorial board and manuscripts on HPV infection and cervical cancer

Hunter’s syndrome

• Exploration of IQ as a relevant endpoint in Hunter’s syndrome
• Endpoint review in Hunter’s syndrome
• PRO dossier on the Disease Activity Score (DAS), Scales of Independent Behaviour-Revised (SIB-R), Behaviour Rating Inventory of Executive Function (BRIEF) and Vineland Adaptive Behaviour Scales (VABS) for Hunter’s syndrome
• Strategic PRO and ObsRO analysis of a Hunter’s Syndrome clinical trial database
• Regulatory support for questionnaires in Hunter’s syndrome

Hyperparathyroidism and hypoparathyroidism

• Payer objection handlers to support treatment in secondary hyperparathyroidism (SHPT)
• Literature review to assess the burden of illness of hypoparathyroidism

Hypertension and Hypotension

Hypertension

• Strategic treatment pathways for treatment in pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH)
• Update to payer value decks in PAH and CTEPH
• Framework for real world evidence generation in PAH and CTEPH
• Strategic plan and real-world evidence toolkit in PAH and CTEPH
• Consultancy on interpretation of phase III trial endpoints in PAH and CTEPH
• Meta-analysis, systematic literature review, data extraction and report on an oral combination treatment of PAH
• Systematic literature review, data extraction & report on sensitivity analyses and a meta-analysis of an oral combination treatment of PAH
• Meta-analysis of an oral combination treatment of PAH
• Meta-analysis of an oral treatment of PAH – exploratory analyses on the effect of administering additional PAH-specific therapy upon two additional endpoints
• Literature review of pulmonary hypertension secondary to chronic obstructive pulmonary disease (COPD)
• Utility assessment in PAH
• Literature review of PAH secondary to chronic obstructive pulmonary disease (COPD)
• Interpretation of clinical trial results in PAH
• Cognitive debriefing interviews with patients with PAH
• Network meta-analysis of combination therapies in PAH
• Psychometric validation of the Living with Pulmonary Hypertension (LPH) questionnaire in PAH
• PRO claims for PAH – endpoint review and regulatory support for the FDA and EMEA
• PRO claims for PAH: endpoint review, regulatory support and qualitative patient interviews
• Qualitative research to explore patient preference for modes of administration in PAH treatments
• Budget impact model for an anti-hypertensive treatment
• UK cost offset model of two treatments for hypertension
• Preparation of an interactive report on a cost-offset model for a treatment for hypertension
• Asian value dossier for hypertension
• Heart study economic model in hypertension
• Advisory board on hypertension
• Cost model for a direct renin inhibitor medication for the treatment of hypertension
• Validation of an adherence questionnaire for hypertension patients utilizing administrative data
• Hypertension database analysis in Japan
• Data management and reporting on a cardiovascular health outcomes project switching study in hypertension
• Development of a compliance and persistence screener in hypertensive patients at risk for cardiovascular events
• Validation of data analysis with an FDA response in orthostatic hypertension
• Adaptation of a heart study economic model in hypertension to Korea
• Validation of the hypertension / dyslipidaemia treatment satisfaction survey

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

Hypogonadism

• Utility evaluation for administration frequency for a drug for hypogonadism
• Targeted literature review, value proposition and budget impact model in hypogonadism
• Consultation on an FDA submission hypogonadism
• Development of a hypogonadism questionnaire
• Psychometric validation of the hypogonadism questionnaire
• Consultancy on FDA-related issues on a hypogonadism questionnaire

Hyponatremia

• Qualitative study to support the content validity of the trail making test and MOS-Cognition in patients with hyponatremia
• Psychometric validation study of the TMT-B and MOS-Cognition tests in patients with hyponatremia
• Regulatory support for cognition endpoints of a treatment for hyponatremia
• FDA advisory committee meeting support for HRQoL endpoints in hyponatraemia