Rare Disease access issues 2015 v2
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The document discusses challenges in accessing rare disease drugs in Canada. It notes that Canada approves only around half of orphan drugs approved in the US and EU. Several provinces have established specialized programs to review rare disease drugs on a case-by-case basis. The document advocates for a "lifecycle approach" where patients are engaged throughout the drug development and review process to improve access and appropriate use. This could include managed access programs combining early approval with ongoing data collection to expand access while managing costs and uncertainties.
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Rare Disease access issues 2015 v2
- 1. Durhane Wong-Rieger, PhD President, Canadian Organization for Rare Disorders November 2015 Canada’s Rare Disease Strategy: Making It Work for Patients
- 2. November 2015Rare Disease Drug Access Rare Disease/ Personalized Drug Development Win-Lose Drug Funding and Access
- 3. November 2015 Innovative Trial Designs Pinnock, H. Pills, People, and Preferences: Evaluating Real Life Practice in Pragmatic Trials Rare Disease Drug Access
- 4. November 2015Rare Disease Drug Access View from HTA and Payers
- 5. November 2015Rare Disease Drug Access Promise of New Medicines Reality of Access to New Medicines
- 6. Rare Diseases: Unaddressed Public Health Issue But Canada’s federation makes national approach a challenge! 2 in 3are Children 80% Genetic BUT 50% No Family History November 2015Rare Disease Drug Access
- 7. # Canadians with Common vs. Rare Diseases 0 0.5 1 1.5 2 2.5 3 3.5 Milions Affected Cancer Diabetes Heart disease Rare Diseases 2.4 M 1.4 M 2.8 M 1.8 M November 2015Rare Disease Drug Access
- 8. USA Orphan Drug Act: A Mother, a Congressman, an Actor November 2015Rare Disease Drug Access
- 9. Challenge of NO Orphan Drug Policy in Canada — In 2002, US FDA approved a breakthrough drug, also called the “miracle drug” or “silver bullet” for rare cancer, chronic myeloid leukemia because a “complete hematological response” was achieved in 98% of patients. Imatinib was approved in Canada in 2007. — In 2010, US FDA approved the first medicine that not only manages but prevents “painful swelling” in patients with hereditary angioedema. The drug was submitted to the EU but the small biotech firm had no intention of coming to Canada until a chance conversation on a plane from Poland convinced the VP to at least consider us. It was approved by Health Canada in 2013. November 2015Rare Disease Drug Access
- 10. Canadian Patients Do Not Access Many Orphan Drugs — No definition, no orphan drug policy; few orphan drugs. ¡ Prior to 1983, only 10 new drugs approved worldwide for rare diseases — Many Orphan Drugs in USA and EU ¡ In USA: about 500 products in 30 years since Orphan Drug Act ¡ In EU: about 100 new 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 in USA ¡ Canada has approved 51% of orphan drugs approved in Europe ¡ Almost half of approved drugs denied reimbursement in Public Drug Plans November 2015Rare Disease Drug Access
- 11. Negotiations with Provincial Decision Maker on Managed Access/Risk Sharing Rare Disease Drug Access Health Canada Issues NOC & DIN Manufacturer submits NOC: Notice of Compliance –! DIN: Drug Identification Number! PMPRB: Patented Med Pricing Review Board! CDR: Common Drug Review! CDEC: Canadian Drug Expert Committee! pCODR: pan-CA Oncology Drug Review! PERC = pCODR Expert Review Committee! INESSS: Institut national d'excellence en santé et en services sociaux! PCPA = panCanadian Pricing Alliance! Provincial Drug Committee Reviews Health Canada status, CDR/pCODR recommendation, Province-specific assessment Provincial process recommends 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 Pharmaceutical Alliance (Pricing Negotiations) Up to 2 mths Transparent ~ varied; not transparent Open ended PMPRB Reference-Based Ceiling Price Private Drug Plans Provincial Exceptional Access Programs Ontario Drugs for Rare Diseases Alberta Special Access/Off-Label Drugs BC Rare Disease Drugs Committee New Brunswick Rare Disease Drugs Fund INESSS for Quebec drug plan Final Decision by Executive Officer/DOH/Minister November 2015
- 12. November 2015Rare Disease Drug Access
- 13. HTA Related Challenges Translate clinical controlled trials data into estimates of clinical effectiveness in a real-world setting Limited evidence: No control arm Small study population No validated outcome Disease heterogeneity No long-term data Uncertainty
- 14. HTA Related Challenges Quantify healthcare costs, utilization and possible savings over the lifetime of the disease or treatment Uncertainty Poor data on economic burden and current management Rapidly growing expenditures on OD Poor cost- effectiveness of OD
- 15. Affordability Equity HTA RELATED CHALLENGES VALUE AND WHAT SOCIETY WANTS
- 16. NO TAILORED METHOD FOR ODs ¥ Most countries do not have HTA and/or pricing and reimbursement-specific orphan drug (OD) decision framework ¥ Under usual circumstances ODs are not cost-effective ¥ Two approaches can be used to make it possible for orphan drugs to be considered cost-effective: ¤ Set higher ICER for ODs ¤ Apply weighted ICER criteria ¥ There is a variation in terms of the criteria that are considered by HTA agencies ¥ Evidence requirements differ between HTA agencies HTA RELATED CHALLENGES
- 17. November 2015Rare Disease Drug Access
- 18. 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. November 2015Rare Disease Drug Access
- 19. 2005: CDR Denies Fabry & MPS-1 Drugs Fabry and MPS patients protest at FPT Health Ministers’ Annual Meeting; demand access to life- saving drugs Solution: Fabry’s Disease and MPS-1, “time-limited research program meeting treatment guidelines” on a risk- sharing basis. Pilot for EXPENSIVE DRUGS FOR RARE DISORDERS?November 2015Rare Disease Drug Access
- 20. 2014: CDR “Yes” but Not at This Price = Delayed Access = Patient Protest November 2015Rare Disease Drug Access
- 21. November 2015Rare Disease Drug Access “...Saskatchewa n decision sets a precedent in Canada because of the External Panel Review that reversed the province’s decision.”
- 22. Specialized Programs for Rare Disease Drugs — 2010: Ontario Drugs for Rare Diseases Program evaluation framework using Markov Model to set access criteria and outcome benchmarks — 2010-11: BC Rare Disease Committee review case-by-case applications based on evidence and physician submission — 2011: Alberta Special Authorization Process for some rare disease drugs; Start-Stop criteria based on submitted evidence; additional $3 million fund for “off-label” or not Special Authorization drugs — 2011-2015: Interprovincial joint action from panCanadian Purchasing Alliance to panCanadian Pricing Alliance to pC Pharmaceutical Alliance — Quebec: Individual approval through “mesure du patient d’exception”; now joining pCPA — 2012: Private drug plans (CLHIA) implements “pooled” insurance fund (full coverage plans only) for all drugs > $25k/year November 2015Rare Disease Drug Access
- 23. Idealized Life-Cycle Approach to Drug Development, Access and Continuous Review Researcher/Clinician: Disease Knowledge; Drug Discovery; Treatment Guidelines Company: Clinical Trials & Outcome Measures; biomedical, clinical, PROs, Real-World Impact Regulator: Approval on Benefits-Risks- Uncertainties; Use & Real-World Monitoring HTA: Comparison Benefits, Risks, Cost w/Alternatives; Place in Therapy Payer: Budget Impact; Access Criteria; R-W Data Collection Patient Input Industry Data November 2015Rare Disease Drug Access
- 24. November 2015 Engaging Patients throughout Life-Cycle Improves Access and Appropriate Use Pre-‐approval: Unmet need, care pathways, resource impact (for HTA) During CTs: Advance/ Special, Crossover, Post-trial access HTA: Experience, values, A/E tolerance, start/stop criteria RW Use: Monitor outcomes; adherence, feedback; adjust therapy Patient partners to improve access criteria and support optimal use Rare Disease Drug Access
- 25. Lifecycle Approach Sets Up Managed Access 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 November 2015Rare Disease Drug Access
- 26. performance based schemes, managed entry agreements outcome based condi5onal coverage performance linked reimbursement / outcomes guarantee financial based pa5ent level popula5on level condi5onal treatment start or con5nua5on coverage with evidence development only in research only with research market share price volume u5liza5on or price cap manufacturer funded treatment ini5a5on Ref: Sullivan S, ISPOR Paris, 2009 November 2015Rare Disease Drug Access
- 27. November 2015Rare Disease Drug Access
- 28. Thank You! November 2015Rare Disease Drug Access Durhane Wong-Rieger, PhD President Canadian Organization for Rare Disorders www.raredisorders.ca 416-969-7435 durhane@sympatico.ca