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4. A Discrete Choice Experiment to Derive Health Utilities for Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency in France

Author

Smith AB, Hanbury A, Whitty JA, Beitia Ortiz de Zarate I, Hammes F, de Pouvourville G, and Buesch K

Subjects
aromatic l-amino acid decarboxylase deficiency, discrete choice experiment, health state utilities, health utilities, Medicine (General), R5-920
Abstract

Adam B Smith,1 Andria Hanbury,1 Jennifer A Whitty,2 Igor Beitia Ortiz de Zarate,3 Florence Hammes,3 Gérard de Pouvourville,4 Katharina Buesch5 1York Health Economics Consortium, University of York, York, UK; 2Norwich Medical School, University of East Anglia, Norwich, UK; 3PTC Therapeutics, Paris, France; 4Department of Economics, ESSEC, Paris, France; 5PTC Therapeutics, Zug, 6300, SwitzerlandCorrespondence: Adam B SmithYork Health Economics Consortium, Enterprise House, Innovation Way, University of York, York, YO10 5NQ, UKTel +44 1904 323620Fax +44 1904 323628Email adam.smith@York.ac.ukPurpose: Cost-effectiveness evaluations of interventions require health utility data. However, in medical conditions, such as aromatic L-amino acid decarboxylase (AADC) deficiency, this presents problems due to the rarity of the disease. The study aim therefore was to employ a discrete choice experiment (DCE) to generate health utilities for AADC deficiency.Methods: A previous literature review, clinician and parent interviews had identified six key AADC deficiency attributes: mobility, muscle weakness, oculogyric crises (OCG), feeding ability, cognitive impairment and screaming. A representative sample of the French general population was recruited. Participants rated 5 health state vignettes describing AADC deficiency using time-trade-off (TTO) and standard gamble (SG). Additionally, participants rated the worst health state using the Health Utility Index version 3 (HUI3). Subsequently, participants completed DCE 11 choice sets. Indirect DCE part-worth utilities were converted to health utilities using the anchors from the TTO, SG and HUI3.Results: The DCE was completed online by 1001 participants (50.9% female; mean age 45.7 years). Most participants (596, 59.5%) provided consistent responses to the repeated choice task. Five models were evaluated, and one preference reversal (“head control”/“sitting unaided”) was identified in all models. The rescaled utilities ranged from 0.3891 to 0.5577 (difference of 0.17 utilities) for TTO anchors corresponding to the worst (633233) and best (111111) health states. Health utilities ranged from 0.5534 to 0.7093 for the SG anchors. The disutility associated with a transition from “no problems walking” to “bedridden” was − 0.0533, whereas disutility of moving from “constant screaming” relative to “no screaming” was − 0.0248. The disutility associated with daily OCG was − 0.0167. Disutilities for the other attributes were small although there were exceptions.Conclusion: A DCE was used to derive health utilities for AADC deficiency. These health utilities will subsequently be used in an economic model evaluating an AADC deficiency intervention.Keywords: aromatic L-amino acid decarboxylase deficiency, discrete choice experiment, health state utilities, health utilities

Published
2022

6. Eliciting Health State Utilities for Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency: A Vignette Study in France

Author

Smith AB, Hanbury A, Beitia Ortiz de Zarate I, Hammes F, de Pouvourville G, and Buesch K

Subjects
aadc deficiency, vignettes, time trade-off, standard gamble, Medicine (General), R5-920
Abstract

Adam B Smith,1 Andria Hanbury,1 Igor Beitia Ortiz de Zarate,2 Florence Hammes,2 Gerard de Pouvourville,3 Katharina Buesch4 1York Health Economics Consortium, University of York, York, UK; 2PTC Therapeutics France, Paris, France; 3Department of Economics, ESSEC, Paris, France; 4PTC Therapeutics GmbH, Zug, SwitzerlandCorrespondence: Adam B SmithYork Health Economics Consortium, University of York, Enterprise House, Innovation Way, YO10 5NQ, UKTel +44 1904 323620Fax +44 1904 323628Email adam.smith@york.ac.ukPurpose: Health-related quality of life (HRQoL) is difficult to measure in rare diseases, especially in paediatric populations, yet capturing HRQoL is critical to evaluating treatment, including the cost-effectiveness of treatments. Given the ultra-rare nature of AADC deficiency indirect elicitation of HRQoL data through proxy caregiver/parent ratings is not feasible. In these circumstances, HRQoL data may be derived through vignette studies using the general population. The aim of the study was to generate health utility values specific for France for AADC deficiency using vignettes.Methods: The study was completed online by panel participants from a French representative sample. Five health state vignettes, reflecting key milestones in the eladocagene exuparvovec clinical trials and economic model, were presented to the participants: “bedridden”, “head control”, “sitting unsupported”, “standing with assistance” and “walking with assistance”. The vignettes had been previously developed with input from parents of patients with AADC deficiency, patients and expert opinion. Participants also completed the Health Utilities Index-3 for the “bedridden” health state.Results: A total of 1001 participants (51% females; mean age 46 years) completed the vignettes. Utilities increased linearly as the health state improved for both the time trade-off (TTO): 0.47 (standard deviation, SD 0.36) to 0.54 (SD 0.36) and standard gamble (SG): 0.61 (SD 0.29) to 0.67 (SD 0.27). A significant minority had incongruent responses (high utilities for the bedridden compared to walking health states) for the vignette (27%). When these were removed, the TTO health utilities (N=729) ranged from 0.39 (SD 0.36) to 0.56 (SD 0.38) and 0.61 (SD 0.30) to 0.69 (SD 0.27) for the SG.Conclusion: Health utilities were derived for AADC deficiency which will be used for a cost-effectiveness model of an AADC deficiency treatment.Keywords: AADC deficiency, vignettes, time trade-off, standard gamble

Published
2021

7. EE657 Health Resource Utilization by Disease Stage for People with Amyotrophic Lateral Sclerosis (pALS) in the French National Health Data System (SNDS)

Author

Stenson, K., primary, Hadjrabia, H., additional, Boer, F., additional, Doutriaux, A., additional, Avot, D., additional, Issa, S., additional, Marguet, S., additional, Bernard, F., additional, Nasanbat, E., additional, Nowacki, G., additional, de Pouvourville, G., additional, Corcia, P., additional, and Couratier, P., additional

Published
2023
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8. PT41 Real-World Evidence in Rare Diseases in France: Treatment Patterns, Healthcare Resource Use, and Related Costs in Von Willebrand Disease (FORvWARD Study)

Author

Trossaërt, M., primary, Biron Andreani, C., additional, Marant Micallef, C., additional, Nerich, V., additional, Lefèvre, C., additional, Née, M., additional, Walter, M., additional, Belhassen, M., additional, Favre-Besse, F., additional, Chatelanaz, C., additional, de Pouvourville, G., additional, Beoletto, J., additional, and Polack, B., additional

Published
2023
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11. HTA261 Preparing for the EU Regulation on HTA and Joint Clinical Assessments of Vaccines

Author

Largeron, N., primary, D'Agostino, P., additional, Curran, D., additional, Dankó, D., additional, Eskola, J., additional, Godfroid, P., additional, Hanley, R., additional, de Pouvourville, G., additional, Postma, M.J., additional, Puig-Barberà, J., additional, de Waure, C., additional, Schmitt, J., additional, Vicere', A., additional, and Beck, E., additional

Published
2023
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12. EE668 Cost-Effectiveness of Pembrolizumab as an Adjuvant Treatment for Adults and Adolescents Aged 12 Years and Older With Resected Stage IIB/IIC Melanoma in France

Author

Saiag, P., primary, Meyer, N., additional, Zhang, S., additional, Jiang, R., additional, Mamane, C., additional, Chaker, O., additional, Bensimon, L., additional, Babonneau, A., additional, Hidra, R., additional, Rey, L., additional, Leproust, S., additional, Rai, A., additional, Vohra, D., additional, Khatri, G., additional, and de Pouvourville, G., additional

Published
2023
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13. PCR130 Factors Associated With Adjuvant Treatment Options in Resected Stage III Melanoma, From a Patient and Physician Perspective: Preference Assessment Using the Discrete Choice Experiment Method (Pamela Study)

Author

Grob, J.J., primary, Dalac, S., additional, De Pouvourville, G., additional, Verdoni, L., additional, Chaker, O., additional, Le Moine, G., additional, Babonneau, A., additional, Bensimon, L., additional, Bennani, M., additional, and Lesimple, T., additional

Published
2023
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33. Out‐of‐pocket expenditures in France to manage psoriasis in adult patients: results from an observational, cross‐sectional, non‐comparative, multicentre study

Author

Richard, M.‐a., Paul, C., de Pouvourville, G., Jullien, D., Mahe, E., Bachelez, H., Seneschal, J., Misery, L., Aubert, R., Reguiai, Z., Shourick, J., Taieb, C., Joly, P., Ezzedine, K., Essec Business School, Hôpital Edouard Herriot [CHU - HCL], Hospices Civils de Lyon (HCL), Centre Hospitalier Victor Dupouy, Imagine - Institut des maladies génétiques (IMAGINE - U1163), Université Paris Descartes - Paris 5 (UPD5)-Institut National de la Santé et de la Recherche Médicale (INSERM), Université de Bordeaux (UB), Laboratoire sur les interactions Epithéliums Neurones (LIEN), Université de Brest (UBO), Département de Dermatologie [CHU de Reims], Centre Hospitalier Universitaire de Reims (CHU Reims), CHU Toulouse [Toulouse], European Market Maintenance Assessment [Fontenay-sous-Bois] (EMMA ), Physiopathologie, Autoimmunité, maladies Neuromusculaires et THErapies Régénératrices (PANTHER), Université de Rouen Normandie (UNIROUEN), Normandie Université (NU)-Normandie Université (NU)-Institut National de la Santé et de la Recherche Médicale (INSERM), Service de dermatologie [Mondor], and Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Hôpital Henri Mondor-Université Paris-Est Créteil Val-de-Marne - Paris 12 (UPEC UP12)

Subjects
vitiligo, Mesures adjuvantes, MESH: Interferon-gamma, Associated measures, MESH: Long-Term Care, MESH: Skin, interferon-γ, MESH: Melanocytes, MESH: Physicians, MESH: Surveys and Questionnaires, skin and connective tissue diseases, Prescriber, Systemic glucocorticoid therapy, MESH: Humans, MESH: Long Term Adverse Effects, MESH: Middle Aged, integumentary system, Corticothérapies systémiques, pathogenesis, MESH: Polypharmacy, cytotoxic CD8+ T cells, targeted therapies, MESH: Keratinocytes, MESH: Male, Prescripteur, MESH: Practice Patterns, Physicians', MESH: Specialization, MESH: Vitiligo, [SDV.MHEP.DERM]Life Sciences [q-bio]/Human health and pathology/Dermatology
Abstract

International audience; Vitiligo is the most common depigmenting disorder affecting 0.1%-2% of the population worldwide. The characteristic white patches result from the selective loss of melanocytes. Sustained recent efforts have resulted in a detailed understanding of the genetic architecture of vitiligo. About 80% of vitiligo risk is attributable to genetic factors; and the rest (20%) is attributable to the environment. Over the past decade, substantial progress has been made in our understanding of the pathogenesis of vitiligo which is now clearly classified as an autoimmune disease. Melanocytes from patients with vitiligo are more susceptible to oxidative stress which begets the release of exosomes and inflammatory cytokines that will lead to activation of the innate immune response and subsequently to adaptive immune response through activation of autoreactive cytotoxic CD8+ T cells. These produce interferon-γ (IFN-γ) which promotes disease progression through IFN-γ-induced chemokine secretion from surrounding keratinocytes to further recruit T cells to the skin through a positive feedback loop. CD8 tissue-resident memory T cells are in turn responsible for long-term maintenance and potential relapse of vitiligo in human patients through cytokine-mediated recruitment of T cells from the circulation. This review summarizes the current knowledge on vitiligo and attempt to give an overview of the future in vitiligo treatment.

Published
2021

37. Impact de la séquence première-deuxième ligne de traitement, acétate d’abiraterone-docétaxel versus docétaxel-acétate d’abiratérone, sur la survie dans le cancer de la prostate résistant à la castration et métastatique ; Étude de cohorte dans le SNDS

Author

Thurin, N.H., primary, Rouyer, M., additional, Gross-Goupil, M., additional, Haaser, T., additional, Rébillard, X., additional, Soulié, M., additional, de Pouvourville, G., additional, Messaoudi, F., additional, Stitou El Amine Demnati, H., additional, Bignon, E., additional, Jové, J., additional, Lamarque, S., additional, Droz-Perroteau, C., additional, Moore, N., additional, and Blin, P., additional

Published
2021
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43. Out‐of‐pocket expenditures in France to manage psoriasis in adult patients: results from an observational, cross‐sectional, non‐comparative, multicentre study

Author

Richard, M.‐A., primary, Paul, C., additional, De Pouvourville, G., additional, Jullien, D., additional, Mahe, E., additional, Bachelez, H., additional, Seneschal, J., additional, Misery, L., additional, Aubert, R., additional, Reguiai, Z., additional, Shourick, J., additional, Taieb, C., additional, Joly, P., additional, and Ezzedine, K., additional

Published
2020
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45. PCN19 Effectiveness and Medical Costs of Abiraterone Acetate Versus Docetaxel in First-LINE Treatment of Metastatic Castration-Resistant Prostate Cancer from the French Nationwide Claims Database (SNDS): Camerra Study

Author

Thurin, N., primary, Rouyer, M., additional, Gross-Goupil, M., additional, Haaser, T., additional, Rébillard, X., additional, Soulie, M., additional, Messaoudi, F., additional, Capone, C., additional, Bignon, E., additional, Lamarque, S., additional, Jové, J., additional, Droz-Perroteau, C., additional, Moore, N., additional, de Pouvourville, G., additional, and Blin, P., additional

Published
2020
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