Prioritization of therapy uncertainties in congenital ichthyosis results from a priority setting partnership
Hernández-Martín A(1), Dávila-Seijo P(2), de Lucas R(3), Baselga E(4), Redondo P(5), Martín-Santiago A(6), Azaña-Defez JM(7), González-Viejo I(8), Jiménez-Ferreres L(9), González-Enseñat MA(10), Arroyo-Manzanal MI(11)(12), Soria JM(12), Garcia-Doval I(13).
(1) Department of Dermatology, Hospital Infantil Niño Jesús, Madrid, Spain.
(2) Dermatology and Venereology Department, Umeå University Hospital, Umeå, Sweden.
(3) Department of Dermatology, Hospital Universitario La Paz, Madrid, Spain.
(4) Department of Dermatology, Hospital Santa Creu y Sant Pau, Barcelona, Spain.
(5) Department of Dermatology, Clínica Universidad de Navarra, Pamplona, Spain.
(6) Department of Dermatology, Hospital Son Espases, Palma de Mallorca, Spain.
(7) Department of Dermatology, Complejo Hospitalario Universitario, Albacete, Spain.
(8) Department of Ophthalmology, Hospital Universitario Miguel Servet, Zaragoza, Spain.
(9) Department of Ear-Nose -and-Throat, Hospital San Rafael, Madrid, Spain.
(10) Department of Dermatology, Hospital Sant Joan de Deu, Barcelona, Spain.
(11) Centro de Referencia Estatal de Atención a personas con Enfermedades Raras y sus familias, Burgos, Spain.
(12) ASIC (Spanish Association of Ichthyosis Patients), Spain.
(13) Research Unit. Fundación Academia Española de Dermatología y Venereología, Madrid, Spain.
Ichthyosis patients suffer important impairment of their quality of life not only because of their dermatological condition but also due to pruritus, sweating abnormalities, delayed growth in childhood, recurrent keratitis, hearing disturbances and social relationship issues.
Current evidence about therapy is limited and research priorities of academic researchers, pharmaceutical companies, health care professionals (HCP), patients and their caregivers may focus on different aspects. Detecting the lack of knowledge in rare diseases is particularly important for establishing a research agenda that matches the interest of academic research and patients' needs.
CITA DEL ARTÍCULO Br J Dermatol. 2015 May 21. doi: 10.1111/bjd.13914.