RATIONALE, AIMS AND OBJECTIVES: The debate about whether to dedicate funds to rare diseases (RD) may hinge on the existence of a societal preference for prioritizing rarity. There are scarce studies about the interest of doctors and general society in RD. METHOD: Four groups of future health and non-health professionals were surveyed in a region of Spain to evaluate their opinion on RD. Questions of the study were organized into four groups: general knowledge on RD, prioritization of research of RD, willingness to assign resources to RD and real distribution of resources to RD. RESULTS: A total of 234 students were surveyed. The mean age of the whole study population was 24.0 years and the 83.3% were female. Only around 25% of the survey sample knew the definitions of RD and orphan drugs. Questions related to willingness to assign resources to RD did not reveal statistically significant differences between study groups. Seventy-three per cent considered that the majority of the budget should be used to treat common diseases. However, resident doctors recognized that they have neither experience nor time to diagnose and treat RD. CONCLUSION: Although resident doctors have a little more general knowledge on RD than other surveyed groups, health and non-health future professionals have a low general knowledge on this topic and none of them prioritized the assignment of funds to RD.
RATIONALE, AIMS AND OBJECTIVES: The debate about whether to dedicate funds to rare diseases (RD) may hinge on the existence of a societal preference for prioritizing rarity. There are scarce studies about the interest of doctors and general society in RD. METHOD: Four groups of future health and non-health professionals were surveyed in a region of Spain to evaluate their opinion on RD. Questions of the study were organized into four groups: general knowledge on RD, prioritization of research of RD, willingness to assign resources to RD and real distribution of resources to RD. RESULTS: A total of 234 students were surveyed. The mean age of the whole study population was 24.0 years and the 83.3% were female. Only around 25% of the survey sample knew the definitions of RD and orphan drugs. Questions related to willingness to assign resources to RD did not reveal statistically significant differences between study groups. Seventy-three per cent considered that the majority of the budget should be used to treat common diseases. However, resident doctors recognized that they have neither experience nor time to diagnose and treat RD. CONCLUSION: Although resident doctors have a little more general knowledge on RD than other surveyed groups, health and non-health future professionals have a low general knowledge on this topic and none of them prioritized the assignment of funds to RD.
Authors: Aaron M Cohen; Steven Chamberlin; Thomas Deloughery; Michelle Nguyen; Steven Bedrick; Stephen Meninger; John J Ko; Jigar J Amin; Alex J Wei; William Hersh Journal: PLoS One Date: 2020-07-02 Impact factor: 3.240
Authors: Enrique Ramalle-Gómara; Elena Domínguez-Garrido; María Gómez-Eguílaz; María Eugenia Marzo-Sola; José Luis Ramón-Trapero; Josefa Gil-de-Gómez Journal: Orphanet J Rare Dis Date: 2020-01-17 Impact factor: 4.123