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picture1_Strategy Slides 78064 | Ie Cra Biogen Eu Sma Policy And Access Tracker Ireland V Aug2021 C278d96f1a


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File: Strategy Slides 78064 | Ie Cra Biogen Eu Sma Policy And Access Tracker Ireland V Aug2021 C278d96f1a
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 ...

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               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                                National strategies for rare Currently valid national rare                      Expired/outdated national rare              No national rare disease strategy 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
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...Glossary of terms abbreviations national international organisation acronyms chop the children s hospital philadelphia infant test cpu corporate pharmaceutical unit intend neuromuscular disorders cl confidence limit doh department health coe centre excellence ema european medicines agency ens extended newborn screening hpra products regulatory authority hta technology assessment hse service executive nmd neuro muscular diseases ncpe for pharmacoeconomics omp orphan medicinal product who world rd rare disease sma spinal atrophy soc standard care access tracker metric descriptions areas key policy components improving to strategies currently valid expired outdated no strategy genetic political leadership dedicated patient group organisations and supporting patients with advocacy both support politically mandate focusing on advocate rather than country specific epidemiology incomplete data reliable estimate from registry or literature limited reliability granularity population estimated c...

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