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Access to Drugs for Rare Diseases in Canada - April 2014


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Access to Drugs for Rare Diseases in Canada - April 2014

  1. 1. CANADIAN ORGANIZATION FOR RARE DISORDERS Access to Drugs for Rare Diseases in Canada
  2. 2. Canadians Suffer Without Orphan Drug Policy  No definition, no orphan drug policy; few orphan drugs.  In 1970’s, only 10 new drugs approved worldwide for rare diseases  Orphan Drug Policies in USA (1983) and EU (2000)  In USA: about 425 approved drugs in 30 years since Orphan Drug Act  In EU: about 100 new approved orphan drugs in 13 years  Benefit: estimates of up to 15 million people  Canadian patients have NO access to half of these drugs  Canada has approved 47% of orphan drugs licensed in3/13/09Data Uncertainty/Rare Disorders 2
  3. 3. NEW Canadian Orphan Drug Regulatory Framework  OD designation criteria and processes aligned with US and EU (promotes collaboration and simultaneous filing)  Formal advice for clinical trials (Canada and/or international), including design, sample, outcome measures, follow-up plan  Transparency and information sharing throughout drug lifecycle, available to HCPs, HTAs, and patients to inform decisions  Life-cycle approach to accommodate evidence from multiple sources before and after a drug is marketed  Patient input: designation, CTs, approval, access, monitoring  Approvals with required post-market monitoring and data evaluation with mandatory reporting (≈5 years) May 2010Access to Drugs Canada 3
  4. 4. Lifecycle Approach to Access for Orphan Drugs • Similar scientific requirements (benefits, harms, uncertainties); built-in flexibility for small clinical trials, adaptive designs, surrogate markers, subgroup analyses • Pre-Market: Pre-clinical/clinical data, quality, labelling; designation; regulatory status elsewhere, evidence limitations; post-market data collection plan • Authorization: Require gathering and dissemination of information to reduce uncertainties re: benefits/harms • Post-Market: Reassess MA; require compiled information, tests or studies; safety monitoring, label changes May 2010 4
  5. 5. Evaluation Throughout Lifecycle Drug Information 5
  6. 6. Final decision made by Executive Officer Drug Funding Process Health Canada Issues NOC & DIN Interim decision made by Executive Officer Non-CDR products / non-pCODR products Manufacturer submits NOC= Notice of Compliance – indicating drug is safe and effective DIN= Drug Identification Number CDR =Common Drug Review CDEC = Canadian Drug Expert Committee pCODR = pan-Canadian Oncology Drug Review PERC = pCODR Expert Review Committee PCPA = panCanadian Pricing Alliance Ontario’s CED reviews Health Canada status, CDR recommendation, pCODR recommendation and PCPA; conducts Ontario-specific review CED provides recommendation to Executive Officer to reimburse (or not) through publicly funded program Common Drug Review products (NCE / new combination product / new indication) CDEC recommendation to drug plans Manufacturer submits pCODR Products (NCE / new combination product / new indication) pERC recommendation to drug plans specific to oncology drugs Up to 2 years Non-transparent Up to 1 year Transparent panCanadian Pricing Alliance Negotiations Up to 2 mths Transparent ~ varied; not ntransparent Open ended
  7. 7. Rare Disease Drugs: Challenges for Reimbursement  Incremental Value Added (effectiveness, side effects, tolerability, improved quality of life) may not equal incremental costs  Pricing criteria may not be established, and willingness to pay may have little impact on pricing  Medicines for rare and unmet needs tend to have high R&D, high uncertainty, high cost  Reimbursement strategies may be directed toward reducing uncertainty in safety, effectiveness, appropriate use, and budget impact.  Managed access schemes include registries, CED, prior authorization, limited use, $ capitation. 7
  8. 8. CDR Limits Access for Orphan Drugs  CDR same as for common drugs  RCTs = small samples, short timeframes, surrogate markers  High $ development, small population = High $/patient  Cost-utility: $/QALY below theoretical $50k threshold  Small # = Low budget impact  CDR recommends “no” to most drugs for rare disorders  50% of common drugs = “Do not list”  70% of DRD = “Do not list”  20% of DRD = “list with conditions”  5% of DRD = “yes”  Provincial drug plans usually adopt CDR recommendations  Private drugs plans usually cover (but some do not)  EU varies but best = 80% to 100% funding 8
  9. 9. Access Mostly by Individual Approval Drug Indication recommend Access Replagal Fabry’s No 3 Yr Research zavesca Gaucher’s No Individual Fabrazyme Fabry’s No 3 Yr Research Aldurazyme MPS I No Yes somavert Acromegaly No Individual Exjade Iron Overload Conditional Individual Nexavar Kidney Cancer No Individual Sutent (Renal) Kidney Cancer No Individual Sutent (GSIT) GIST Conditional Individual Myozyme Pompe’s Conditional Individual Elaprase MPS II No Individual Xyrem Narcolepsy No Revlimid MDS JODR - No Individual Catena Friedreich’s Ataxia Quebec Only Soliris PNH No Individual Naglazyme MPS VI Not Submitted Individual Cayston Cystic Fibrosis Yes In Submission Ilaris CAPS No Individual Kuvan PKU NO Individual
  10. 10. Canadian Budget Impact Orphan Drugs  Private Insurers  Private insurers reimbursed $10.1 billion in drug costs in 2011  Over 50% of all drugs are purchased privately in Canada  High-cost, specialty drugs a growing challenge in the future  Currently specialty drugs represent about 20% of plan costs for employers, but less than 1% of total claims  Specialty drug costs forecast to be 25% of cost by 2015 – 61% of drugs in Phase III trials are specialty drugs  Public Drug Plans  Only 50%-60% OD have market approval in Canada  OD accessed through Special Access Program mostly NOT reimbursed by public drug plans  Rare disease drugs less than 1% total public drug spend Drug Information 10
  11. 11. Provincial Drug Access for Rare Diseases  2010: Ontario Drugs for Rare Diseases Program; uses Markov Model to set “start-stop” criteria for gaining access, staying on therapy, or stopping  2010-11: BC Rare Disease Committee reviews on case-by- case basis  2011: Alberta establishes Special Authorization Process including rare disease drugs; Start-Stop criteria; separate fund for off-label drugs  2011-2012: Ontario leads implementation of panCanadian Pricing Alliance to negotiate single price for all; 1st drug for rare disease  Quebec: 2012 announces Rare Disease Plan (but no action)  NEW! April 2014: CADTH says no separate pathway for
  12. 12. Lifecycle Approach Sets Up Managed Entry Programs  Propose “coverage with evidence development” for orphan drugs  Early approval based on life-threatening or severely debilitating condition with no other effective treatments  Regulatory approval for ODs require on-going data collection and resubmission of outcomes data (5 years)  Patient registries to collect post-market safety and effectiveness  On-going studies with expanded patient population  Challenge of “no funding” to expanded data collection  Limits access to patients beyond clinical trials  Limits “real world” data collection Nov 2010USA CA EU Access to OD 12
  13. 13. Examples in Oncology Evidence Building Program  Principles for funding CTs within funded indication  Equitable and timely access to treatments that are safe, offer maximum clinical benefits, and align with best practices;  Ensure coverage decisions are evidence-based, fiscally responsible, and consistent with the OPDP policies  Fair, transparent, and accountable process  Examples of EBP Clinical Trials  Change in dosing/schedule or combination with another agent  Population not funded  For re-treatment  Approved indication but not “cost-effective”  Indication under trial  Same or different pharmacological class as funded drug 13NIHTA November 2013
  14. 14. Sustainable Access to Orphan Drugs  Prices remain high because of lack of competition for small markets (biosimilars not that easy); international pressure for:  Value-based pricing assessed not at market entry but after period of “real-world” use; consensus benchmarks and outcome indicators (Need sufficient database and appropriate patient subgroups)  Pricing transparency (actual negotiated prices, including rebates)  Automatic price reduction at expiration of patent and market exclusivity period  Patented Medicines Pricing Review Board “ceiling price” not reality  PMPRB: Starting point for price negotiation but CONFIDENTIAL  panCanadian “Product Listing” Alliance: starting point for universal pricing, listing, and re-evaluation/re-negotiation  Compatible with Health Canada lifecycle model with evaluation plan submitted at time of market authorization Drug Information 14
  15. 15. CORD ARCTIC QUEST—August 2011 What can people with rare disorders do? Anything!
  16. 16. Thank You! Nov 2010USA CA EU Access to OD 16 Durhane Wong-Rieger, PhD President Canadian Organization for Rare Disorders 416-969-7435