SLIDE 1
THE BIG PROBLEM
Getting to know your team, the Big Problem and its Systemic Root Causes Dorica Dan’s team working for rare diseases
PHASE 1: SYSTEM CHANGE JOURNEY
CLARIFYING THE BIG PROBLEM
- 1. Big Problem statement + scale
More th than 90 90% of
- f th
the rare diseases pati tients ts in in Romania are undiagnosed and struggle to access the services and the support they need, about 50 50% are ch
- children. 65
65% of
- f pati
tients have to to visit at at least 7 different physici cians before diagnosis, 40 40% pati tients are misdiagnosed at at least once. The waiti ting ti time for ge gett tting tr treatment is is at at least 4.8 years from th the first sympto toms which means that many children die before the age of
- five. Altogether, this significantly affects the quality of >1.000
000.000 000 undiagn gnosed pati tients ts in in Romania and approx. 4.000 000.000 people who are th their family members/ ca caregi givers, which ch co constitu tutes 20 20% of
- f th
the to tota tal pop population of
- f Rom
Romania.
- 2. Consequences if problem is not solved (relevance)
- 3. Systems that drive the big probem
2a, Poor
- or quali
quality ty of
- f life for
- r pa
patie tients ts and nd fam amil ilie ies
- 1.000.000 patients with RD in Romania suffer without
diagnose or treatment, ~20% of population
- 35
35% of RD patient ents die e befor efore age ge of f 5
2b 2b, Severe econ
- nomic
ical bur burden for
- r fam
amili ilies and nd soc socie iety
- > 4h/da
/day spent ent by by avrg. caregi giver er on treatment related tasks
- 85
85% of
- f aff
ffec ected ed families fac ace high or
- r very high
gh cost of care
- 63
63% of
- f car
areg egiver ers repo eported ed limited ed jo job opt ptions ns, affecting the economies workforce
2c 2c, He Heavy y emot
- tio
ional l bur burden for
- r car
aregivers
- 58
58% of
- f caregi
giver ers lost st their relat ation
- nshi
hips ps, fam amily or
- r frien
ends ds
- 80
80% of
- f caregi
giver ers suff uffer from
- m ment
ntal al heal alth issu ssues
System A: The system of education
Onl nly 70 70 genet eneticist sts in in Ro Roman ania and 6 genet enetic cent nter ers; There is no no a med edical al sp special alty for rare diseases and it requires collaboration and multidisciplinarity. No No curricul ula for RDs in the universities.
System B: The system of diagnosis and care for rare diseases in Romania
Car are pa pathw hway ays for for RD RDs in in Ro Roman ania ar are not not st structur ured ed or don't exist and patients often struggle to access the services and the support they need; Fr Frag agment entat ation of
- f the
he few ew ex exist sting ng car are ser services es & lac ack of
- f coo
- ordi
dinat ation
- n
(different ministries, different coordination, budget, etc.). No No common visi sion of
- f heal
alth and and social car are for RDs.
Our focus:
System of diagnosis and care for rare disease patients in Romania
System C: The system of funding
No No pr prop
- per
er fun unding ng of
- f the NPR
PRD and not enough investment on integrated care. National programs for care of RDs are not holistic and cover only the drugs. No No regi gistries, s, no no accurat ate data. http://insp.gov.ro/sites/cnepss/date-statistice-boli-rare/ http://www.mmuncii.ro/j33/images/buletin_statistic/dizab_2019.pdf