Transformative effect of a Humanitarian Program for individuals affected by rare diseases: building support systems and creating local expertise

Table 2 Individuals enrolled in the Humanitarian Program by disease

Disease	Year disease introduced to program	Historical data		Current data^b
Disease	Year disease introduced to program	Number of individuals since inception	Number of countries supported since inception	Number of individuals receiving humanitarian treatment annually	Number of countries supported today	Average number of new cases approved each year; over past 5 years	Average time current individuals have received humanitarian treatment (years)
Gaucher disease	1991	1248	75	652	52	48.2	9.8
Fabry disease	2003	972	50	150	21	59.2	3.4
Pompe disease	2006	766	70	142	37	45.8	4.8
MPS I	2003	390	55	55	20	9.8	6.6
MPS II^a	2014	35	7	24	6	5	3.0
Total		3411	103	1023	69	168	5.5

Data as of May 2021
MPS, Mucopolysaccharidosis
^aHumanitarian access in MPS II is limited to Sanofi Genzyme territory only
^bData for individuals entering the Humanitarian Program are dynamic; with a rolling submission process, new individuals are approved throughout the year. Individuals also depart the program owing to a number of reasons, including death, individual or physician decision to stop treatment, and commercial transition

ISSN: 1750-1172