Evaluación económica en Onco-hematología y
Enfermedades Raras
Roberto Marín Gil
FEA Farmacia Hospitalaria
Servicio Andaluz de Salud
Provost G "Homeless" or "orphan" drugs Am J Health
Syst Pharm November 1, 1968 25:609
2 preguntas clave
• ¿Debemos realizar EE de
medicamentos huérfanos?
• ¿Qué podemos hacer / debemos
no hacer?
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.
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.
Escaso impacto presupuestario
460 potential rare disease treatments in the late-stage pipeline (in
clinical trials or filed for regulatory review) in 2011 in the US (PhRMA)
Investigación costosa
Value-based pricing
Valium-based
pricing
2 preguntas clave
• ¿Debemos realizar EE de
medicamentos huérfanos?
• ¿Qué podemos hacer / debemos
no hacer?
Valores umbrales en diferentes tipos de medicamento
300.000 £
<1/50.000
Ultra-huérfanos
200.000 £ - 300.000£
(250.000 €- 373.000€)
Coste AVAC
200.000 £
En cambios…
100.000 £
< 7000 pac.
Criterios End of Life (EoL)
40.000 £ - 50.000 £
(50.000 € - 62.500 €)
50.000 £
0,2
1,29
Huérfanos
30.000 £ - 40.000 £
Nº?? pac.
20.000 £ - 30.000 £
(37.000 € - 50.000 €)
(25.000 € -37.000 €)
5
Prevalencia por 10.000 habitantes
Ref: Olga Delgado: Propuestas y estimaciones
Hughes-Wilson et al 2012
75%
50%
25%
Coste-efectividad
Estandarizar
Evaluación Económica
Incertidumbre beneficio
Riesgo NO compartido
Agrupar pacientes
Limitaciones de los registros
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.’
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)
And then… media:
Media responds shocked:
Should costs be the reason to
stop reimbursement of drugs that
only affect a very small
population?
MUCHAS GRACIAS
[email protected]
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