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188x Filetype PPTX File size 0.73 MB Source: smatracker.eu
Glossary of Terms
Abbreviations National & International Organisation Acronyms
CHOP- The Children’s Hospital of Philadelphia Infant Test of CPU Corporate Pharmaceutical Unit
INTEND Neuromuscular Disorders
Cl Confidence Limit DoH Department of Health
CoE Centre of Excellence EMA European Medicines Agency
ENS Extended Newborn Screening HPRA Health Products Regulatory Authority
HTA Health Technology Assessment HSE Health Service Executive
NMD Neuro-Muscular Diseases NCPE National Centre for Pharmacoeconomics
OMP Orphan Medicinal Product WHO World Health Organisation
RD Rare Disease
SMA Spinal Muscular Atrophy
SoC Standard of Care
2
Access tracker metric descriptions (1/3)
Areas Key Policy Components for improving access to SMA care
National strategies for rare Currently valid national rare Expired/outdated national rare No national rare disease strategy
/ genetic disorders disease strategy disease strategy
1 Political leadership Dedicated patient group Dedicated patient group
& policy Patient organisations and supporting SMA patients who supporting SMA patients with No dedicated patient group
advocacy both support and politically mandate focusing on patient supporting SMA patients
advocate for patients support rather than political
advocacy
Country-specific epidemiology Incomplete country-specific data No reliable data on the country’s
Epidemiology Estimate data from registry or literature of limited reliability / granularity SMA population; estimated
with patient characteristics (e.g. (e.g. only total population population is based on global/EU
type, age) number is available, old data) prevalence
Consolidated national patient Consolidated national patient No consolidated national patient
Healthcare system National SMA patient registry that captures both registry that captures only registry (no registry or only
2 preparedness registry epidemiological and clinical epidemiological data and no fragmented local/product-specific
history data report of clinical history registries)
Easy access to designated CoEs Limited access to designated Very limited access to
for the treatment of SMA (defined CoEs for the treatment of SMA designated CoEs for the
Infrastructure by ≥0.80 CoEs per million (defined by 0.21 - 0.79 CoEs per treatment of SMA (defined by
population) million population) 0.00 - 0.2 CoEs per million
population)
CoE – Centre of Excellence, SMA – Spinal Muscular Atrophy
Metric Status ●= Good ●= Room for improvement ●= Not good Detailed definitions of the specific criteria
3 for each metric are provided in slide notes
Variation Status ●= No Variation = Clinical variation = Geographical variation
Access tracker metric descriptions (2/3)
Areas Key Policy Components for improving access to SMA care
Inclusion/commitment to No permanent or pilot inclusion
include SMA in national No commitment to include SMA of SMA in newborn screening
3 newborn screening program in national newborn screening programs; and there is
Diagnosis Efficiency of diagnostic with follow up and provision of but there are ongoing/planned reimbursed access to diagnostic
pathway genetic counselling; and there pilots; and there is reimbursed resources but there have been
is reimbursed and efficient and efficient access to genetic reported diagnostic barriers
access to genetic diagnostic diagnostic resources such as delays in diagnosis
resources
Well established reimbursed Early access programme with No reimbursed early access
Post-MA early access early access programme partial reimbursement; only programme available after MA;
pathways available on a cohort and available for individual only MNF-funded programs are
named-patient basis after MA applicants after MA available
4 Access Pathways
Specialised reimbursement / Standard reimbursement/HTA No specialised reimbursement/
Specialised HTA pathway tailored to orphan pathway with the possibility of HTA pathway tailored for orphan
reimbursement / HTA products for fair and efficient accelerated access to orphan products or orphan products are
pathways access to treatment products required to overcome additional
hurdles to gain access
HTA – Health Technology Assessment, MA – Marketing Authorisation, MNF – Manufacturer, SMA – Spinal Muscular Atrophy
Metric Status ●= Good ●= Room for improvement ●= Not good Detailed definitions of the specific criteria
4 for each metric are provided in slide notes
Variation Status ●= No Variation = Clinical variation = Geographical variation
Access tracker metric descriptions (3/3)
Areas Key Policy Components for improving access to SMA care
The country has adopted The country has adopted
Treatment and care guidelines that provide guidelines that provide The country has not adopted
guideline treatment and care recommendations on care that any guidelines and do not
recommendations recommendations that reflect reflect the most recent clinical provide any treatment or care
the most recent clinical consensus and evidence but not recommendations
consensus and evidence treatment
Spinraza Treatment is reimbursed but A negative reimbursement
Treatment Zolgensma Treatment is reimbursed and there are access restrictions decision has been made
Access to availability there are no access restrictions applied to the relevant resulting in no access for any
treatment and As of Jan-21 Evrysdi to the relevant regulatory label* regulatory label* indicated patients
5 care Grey Product does not yet have marketing authorisation; or assessment ongoing/not yet completed
Blue Treatment is reimbursed through a formally agreed early access program
Selected care provisions^ Selected care provisions^
analysed are reimbursed for analysed are reimbursed for Selected care provisions^
Selected care provisions SMA patients and caregivers SMA patients or caregivers but analysed are not reimbursed or
without major issues there are issues for patients to limited for SMA patients or
experienced to gain access to access the necessary care caregivers
these services
EMA – European Medicines Agency, SMA – Spinal Muscular Atrophy
* Comparison to the relevant regulatory label will be made (EMA in most markets; national regulatory agencies in CH, UA, MD, RS) EMA approved labels for Spinraza and Zolgensma are included in the slide notes
^ Treatment-related care will focus specifically on: (1) Physiotherapy and rehabilitation therapies, (2) Home adaptation; and (3) Financial patient/caregiver support
Metric Status ●= Good ●= Room for improvement ●= Not good Detailed definitions of the specific criteria
5 for each metric are provided in slide notes
Variation Status ●= No Variation = Clinical variation = Geographical variation
Summary of key information: Ireland
Areas Summary Metric Status Variation
• There is no national strategy dedicated to SMA but it is indirectly covered by the National strategies for rare / None
Political National Rare Disease Plan for Ireland. genetic disorders ● ●
leadership & • Established in 2019, SMA Ireland is a voluntary organization committed to raising Patient organisations and
1 policy awareness of SMA and campaigning for equitable access to treatment. advocacy ● ● None
• Literature studies of prevalence of SMA in Ireland are sparse in number and Epidemiology Estimate ● ● None
Healthcare largely outdated. The UK SMA registry is also set-up for Irish patients but a
system breakdown of the national epidemiology is not available and the extent to which National SMA patient registry ● ● None
2 preparedness Irish treatment centres participates in submitting patients to the registry unclear.
• Ireland has 0.41 centers of excellence per million population. Infrastructure ● ● None
• There is a national Newborn bloodspot screening program in Ireland which Efficiency of diagnostic
Diagnosis screens for eight conditions. SMA is not included in the program and no planned pathway ● ● None
3 SMA screening pilots were identified.
• In Ireland, early access can be granted to certain clinical trial therapies where Post-MA early access ● ● None
Access physicians accept full responsibility to meet the needs of individual patients. pathways
Pathways • The Rare Disease Technology Review Committee assesses new products for rare Specialised reimbursement/ ● ● None
4 diseases. HTA pathways
• Treatment guidelines are currently based on international agreed Standard of Availability of treatment and ● ● None
Care guidelines for SMA, updated in 2017 care guidelines
• Spinraza was made available for reimbursement in patients with SMA type I, II Spinraza ● ● None
Access to and III up to 18 years old on June 2019. Treatment
treatment and • A final reimbursement decision has not yet been made for Zolgensma but the availability Zolgensma ● ● None
5 care NCPE has recommended that Zolgensma is not considered for reimbursement
based on the joint HTA conducted through the BeNeLuxA initiative Evrysdi ● ● None
• The NCPE is awaiting an HTA submission before a full HTA commences Varies by
• Families affected by SMA are entitled to a range of government financial support. Selected care provisions ● severity
Metric Status ●= Good = Room for improvement = Not good = Early access program = Not authorised/assessed Detailed definitions of the
● ● ● ●
6 specific criteria for each metric
Variation Status ●= No Variation = Clinical variation = Geographical variation are provided in slide notes
