Day 1: Status of Canadian access for RD drugs Panel
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Canada lags behind Europe in approving and publicly reimbursing drugs for rare diseases: - Of 63 drugs approved by EMA from 2015-2020, only 24 completed negotiations for public coverage in Canada. - Less than half of 41 drugs approved by Health Canada received public reimbursement. - The time from regulatory approval to reimbursement is typically 1-2 years in most Canadian provinces.
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Day 1: Status of Canadian access for RD drugs Panel
- 1. What is the status of Canadian access to drugs for rare disease? CORD Conference June 8, 2022 Alexandra Chambers Manager, National Oncology Policy Health Policy & Patient Access
- 2. To understand how access to rare diseases in Canada compared to Europe, we conducted an analysis of public reimbursement of drugs for rare disease approved by EMA Volume Rate of access Timelines Drugs with Orphan designation from EMA from January 2015 to March 2020 Ward, L.M. et al. Orphanet J Rare Dis 17, 113 (2022). https://doi.org/10.1186/s13023-022-02260-6 2
- 3. Of the 63 drugs that received EMA approval, 24 drugs completed a negotiation in Canada 63 drugs with EMA approval and orphan designation (between Jan 2015 and Mar 2020) 41 drugs with Health Canada approval 30 drugs with positive CADTH recommendation (20 INESSS) 24 drugs with completed pCPA negotiation Ward, L.M. et al. Orphanet J Rare Dis 17, 113 (2022). https://doi.org/10.1186/s13023-022-02260-6 3
- 4. Fewer than half of the drugs with Health Canada approval are publicly reimbursed in any Canadian province Ward, L.M. et al. Orphanet J Rare Dis 17, 113 (2022). https://doi.org/10.1186/s13023-022-02260-6 4
- 5. In most provinces, the time from regulatory approval to reimbursement is between 1-2 years Ward, L.M. et al. Orphanet J Rare Dis 17, 113 (2022). https://doi.org/10.1186/s13023-022-02260-6 5 Time to reimbursement <1 year Time to reimbursement 1-2 years Time to reimbursement >2 years
- 6. Updated Analysis: Jan 2015 to Dec 2020* * Updated the analysis from March 2020 to Dec 2020 from the original publication: Ward, L.M. et al. Orphanet J Rare Dis 17, 113 (2022). https://doi.org/10.1186/s13023-022-02260-6 6 85 drugs with EMA approval and orphan designation 52 drugs with Health Canada approval 30 drugs followed the priority review (or accelerated NOC/c) pathway 22 drugs followed the standard review pathway 36 positive CADTH Recommendations 8 negative CADTH Recommendations 28 Pre-NOC submissions to CADTH 16 Post-NOC submissions to CADTH 48 drugs submitted to CADTH for review 44 drugs with CADTH Recommendations (4 reviews ongoing as of May 27, 2022)
- 7. Updated Analysis: Jan 2015 to Dec 2020* * Updated the analysis from March 2020 to Dec 2020 from the original publication: Ward, L.M. et al. Orphanet J Rare Dis 17, 113 (2022). https://doi.org/10.1186/s13023-022-02260-6 7 Overall (range) Pre-NOC (range) Post-NOC (range) Mean days from Health Canada Approval to CADTH Recommendation 228 (34-610) 137 (34-315) 383 (231-610) Mean days from Health Canada Approval to pCPA negotiation 528 (91-1041) 399 (91-1041) 747 (510-1010) We need a strategy for timely and equitable access to therapy for rare disease for Canadians that includes benchmarks for success.
- 8. Which Drugs in the List of Essential Drugs for Rare Disorders are Funded in Canada? Nigel S B Rawson, PhD Saskatoon, SK
- 9. Methods § Rare Disease Treatment Access Working Group of the International Rare Diseases Research Consortium’s list of essential medicines for rare conditions based on US, EU and Chinese regulatory agency approvals. § List includes 202 medicines for 140 disorders. § Drugs for rare cancers deliberately excluded by Working Group. § Medicines unlikely to be covered by government drug plans excluded. § Medicines with multiple indications included only once. § 117 medicines remained – compare with 138 in US. § Listing in formulary or special access list at end of August 2021. Case- by-case coverage excluded. No account taken of access conditions. § Medicines categorized based on prevalence of disorder being treated: ≤1 case per 100,000; >1 case per 100,000 to 1 case per 10,000; >1 case per 10,000.
- 10. Medicines Approved in Canada Disorder Medicine Metabolic Fatty acid oxidation disorders Triheptanoin Gaucher disease Eliglustat; imiglucerase; miglustat; taliglucerase; velaglucerase alfa Genetic carnitine deficiency Levocarnitine Homocystinuria Betaine Homozygous familial hypercholesterolemia Evolocumab; lomitapide; rosuvastatin Lysosomal acid lipase deficiency; Wolman disease; cholesterylester storage disease Sebelipase alfa Mucopolysaccharidosis I Laronidase Mucopolysaccharidosis II (Hunter syndrome) Idursulfase Mucopolysaccharidosis IV (Morquio A syndrome) Elosulfase alfa Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) Galsulfase N-acetylglutamate synthetase deficiency Carglumic acid Nephropathic cystinosis Cysteamine (enteric/non-enteric coated and eyedrops) Neuronal ceroid lipofuscinosis type 2 Cerliponase alfa Pediatric onset hypophosphatasia Asfotase alfa Tyrosinemia type I Nitisinone Cobalamin defects Hydroxocobalamin Cholesterol and bile acid synthesis defects Cholic acid Fabry disease (alphagalactosidase A deficiency) Agalsidase beta; migalastat
- 11. Disorder Medicine Hyperphenylalaninemia Sapropterin Hypophosphatemic rickets (X-linked) Burosumab Pompe disease Alglucosidase alfa Urea cycle disorders Sodium phenylbutyrate Wilson disease Penicillamine; trientine Uric acid nephrolithiasis prevention Potassium citrate Neurologic 5q spinal muscular atrophy Nusinersen Lambert-Eaton myasthenic syndrome Amifampridine Lennox-Gastaut syndrome Rufinamide Transthyretin amyloidosis Inotersen; patisiran; tafamidis Amyotrophic lateral sclerosis Radicava; riluzole Complex and rare disease epilepsy Clobazam; lamotrigine; topiramate Dystonia; spasticity Baclofen Huntington disease Tetrabenazine Severe infant myoclonic epilepsy (Dravet syndrome) Stiripentol Tuberous sclerosis complex Everolimus Infantile spasms Vigabatrin Juvenile myoclonic epilepsy; generalized epilepsy Levetiracetam Multiple sclerosis Fingolimod; siponimod; teriflunomide Myasthenia gravis Pyridostigmine Narcolepsy with cataplexy Pitolisant; sodium oxybate Parkinson disease (young and early-onset) Carbidopa/levodopa; pramipexole; rasagiline; selegiline Spina bifida prevention Folic acid
- 12. Disorder Medicine Hematologic Acquired thrombotic thrombocytopenic purpura Caplacizumab Multicentric Castleman's disease Siltuximab Paroxysmal nocturnal hemoglobinuria Ravulizumab Essential thrombocythemia Anagrelide Idiopathic thrombocytopenic purpura; aplastic anemia Eltrombopag Immune (idiopathic) thrombocytopenic purpura Romiplostim Iron overload Deferiprone Polycythemia vera Ruxolitinib Inflammatory Dermatomyositis, atypical hemolytic uremic syndrome, neuromyelitis optica, paroxysmal nocturnal hemoglobinuria Eculizumab Anti-neutrophil vasculitis, Wegener's granulomatosis, Churg-Strauss syndrome Rituximab Familial Mediterranean fever, cryopyrin fevers Canakinumab Hereditary angioedema Icatibant; lanadelumab; tranexamic acid Still's disease, systemic juvenile arthritis Anakinra Juvenile rheumatoid arthritis Abatacept; adalimumab; etanercept; golimumab; infliximab; methotrexate; methylprednisolone; tocilizumab Neurotrophic keratitis Cenegermin Non-infectious uveitis Dexamethasone
- 13. Disorder Medicine Pediatric Crohn's disease Adalimumab; infliximab Pediatric ulcerative colitis 5-aminosalicylic acid (mesalamine); adalimumab; infliximab Primary biliary cholangitis Obeticholic acid Endocrine Acromegaly Lanreotide; octreotide; pasireotide; pegvisomant Adrenal insufficiency Hydrocortisone Growth hormone deficiency in children Somatropin Paget's disease (osteitis deformans) Calcitonin Primary insulin-like growth factor-1 deficiency Mecasermin Pulmonary Idiopathic pulmonary fibrosis Nintedanib; pirfenidone Pulmonary arterial hypertension Ambrisentan; bosentan; macitentan; riociguat; selexipag; sildenafil; tadalafil; treprostinil Cystic fibrosis Ivacaftor; lumacaftor/ivacaftor; tezacaftor/ ivacaftor; aztreonam; colistimethate; levofloxacin; tobramycin Miscellaneous Autosomal dominant polycystic kidney disease Tolvaptan Hepatic veno-occlusive disease; sinusoidal obstruction Defibrotide Short bowel syndrome Teduglutide Inherited retinal dystrophy Voretigene neparvovec Ventricular tachycardia Amiodarone
- 14. Listing status of 117 unique medicines by government plan 0 10 20 30 40 50 60 70 BC AB SK MB ON QC NB NS PEI NL NIHB % Open Access Conditional Access Open or Conditional Access
- 15. Listing (excluding drugs with common indications) by government plan (n=100) 0 10 20 30 40 50 60 70 BC AB SK MB ON QC NB NS PEI NL NIHB % Open Access Conditional Access Open or Conditional Access
- 16. Average listing rate by disorder prevalence* 0% 10% 20% 30% 40% 50% 60% Open or Conditional access Conditional access Open access ≤1 per 100,000 >1 per 100,000 to 1 per 10,000 >1 per 10,000 * Excludes drugs with common indications
- 17. Conclusions § 85% of the medicines approved in the US approved in Canada. § Considerable differences in listing rate between government drug plans. § Listing rate of ultra-rare disorder drugs much lower than listing rate of other rare disorder drugs. Particularly low in BC, Manitoba, PEI and Newfoundland. § Ultra-rare disorder drugs tend to be more expensive. § https://www.canadianhealthpolicy.com/product/availability-and- accessibility-of-essential-drugs-for-rare-disorders-in-canada-2/.