1 / 29

Economic Evaluation in Rare Diseases and Oncohematology

This article discusses the economic evaluation in onco-hematology and rare diseases, including the challenges of funding treatments, societal preferences, and cost-effectiveness analysis.

howed
Download Presentation

Economic Evaluation in Rare Diseases and Oncohematology

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript

  1. Evaluación económica en Onco-hematología y Enfermedades Raras Roberto Marín Gil FEA Farmacia Hospitalaria Servicio Andaluz de Salud

  2. Provost G "Homeless" or "orphan" drugsAm J Health Syst Pharm November 1, 1968 25:609

  3. 2 preguntas clave • ¿Debemos realizar EE de medicamentos huérfanos? • ¿Qué podemos hacer / debemos no hacer?

  4. Main outcome measure Choice between funding treatment for a rare disease versus a common disease and how funds should be allocated if it were not possible to treat all patients, for each of two scenarios: identical treatment costs per patient and higher costs for the rare disease.

  5. Despite strong general support for statements expressing a desire for equal treatment rights for patients with rare diseases, there was little evidence that a societal preference for rarity exists if treatment of patients with rare diseases is at the expense of treatment of those with common diseases.

  6. Escaso impacto presupuestario

  7. 460 potential rare disease treatments in the late-stage pipeline (in clinical trials or filed for regulatory review) in 2011 in the US (PhRMA)

  8. Investigación costosa

  9. Value-based pricing Valium-based pricing

  10. 2 preguntas clave • ¿Debemos realizar EE de medicamentos huérfanos? • ¿Qué podemos hacer / debemos no hacer?

  11. Valores umbrales en diferentes tipos de medicamento 300.000 £ <1/50.000 Ultra-huérfanos 200.000 £- 300.000£ (250.000 €- 373.000€) 200.000 £ En cambios… Coste AVAC 100.000 £ < 7000 pac. Criterios End of Life (EoL) 40.000 £- 50.000 £ (50.000 € - 62.500 €) Huérfanos 30.000 £- 40.000 £ (37.000 € - 50.000 €) Nº?? pac. 20.000 £- 30.000 £ (25.000 € -37.000 €) 50.000 £ 0,2 1,29 5 Prevalencia por 10.000 habitantes Ref: Olga Delgado: Propuestas y estimaciones

  12. Hughes-Wilson et al 2012

  13. 75% 50% 25% Coste-efectividad

  14. Estandarizar Evaluación Económica

  15. Incertidumbre beneficio Riesgo NO compartido Agrupar pacientes

  16. Limitaciones de los registros

  17. Reality: Case study from NL Myozyme for Pompe disease Intramural (IV) orphan drug registered for: ‘long-term enzyme replacement therapy (ERT) in patients with a confirmed diagnosis of Pompe disease (acid α-glucosidase deficiency).Myozyme is indicated in adults and paediatric patients of all ages.’

  18. T=4 (2011) • Therapeutical value only for patients with classical form (n=13) • Real annual costs: € 41 million • Cost-effectiveness: • Infant form: €300 000 / QALY (improved survival and QoL) • Late onset form: €15 million- €33 million/QALY (slightly improved survival and no improvement QoL)

  19. And then… media: Media respondsshocked: Shouldcostsbe the reason to stop reimbursement of drugs thatonly affect a verysmallpopulation?

  20. MUCHAS GRACIAS romagil79@hotmail.com

More Related