Canada s Long Journey Toward An Orphan Drug Policy

Transcription

1 Canada s Long Journey Toward An Orphan Drug Policy DURHANE WONG-RIEGER, PHD PRESIDENT CANADIAN ORGANIZATION FOR RARE DISORDERS September 15, 2011

2 Components of Canadian Plan for Rare Disorders Definition of Rare Disease Orphan Drug Policy Access to Diagnosis, Treatment, Social Services Research on Rare Diseases Rare Disease Centers of Expertise/Reference Screening, including Prenatal and Newborn Empowered Patient Organizations

3 Canadian Rare Disease Time Line Feb 1996: Health Canada refutes need for Orphan Drug Act Sep 2005: Ontario expands NBS to 21 disorders 2005: BIOTECanada proposes Orphan Drug Policy Sep 2005: CORD, Fabry, MPS lobby at FPT Health Ministers Conference on drug access Feb 2006: CORD proposes Orphan Drug/Rare Disease Policy Jun 2006: FPT Ministers announce 3-year funding for Fabry s Apr 2007: CORD hosts 1st Canadian Conference on Orphan Drugs and Rare Disorders 2007: Quebec announces intention to create Rare Disease Policy

4 Canadian Rare Disease Time Line Feb 2008: Canada takes part in 1 st Intl Rare Disease Day Feb 2008: HC Standing Committee on Health asks CADTH for strategy for rare disease drugs May 2008: Parliament approves MP Bell Motion on Orphan Drugs and Rare Disorders (almost) unanimously Dec 2008 AB announces Rare Diseases Drug Program Jan 2009: ON introduces Drugs for Rare Diseases Program Dec 2009: CORD hosts consultation on draft OD regulations Jan 2010: ADM writes Health Canada addressing drugs for rare diseases regulatory & legislative modernization efforts

5 Canadian Rare Disease Time Line May 2010: CIHR Genetics Institute announces 5-year Research Competition on Rare Diseases June 2010: CORD presents to House Standing Committee on Health proposal for Canadian Plan for Rare Disorders Oct 2010: SC Health recommends to Health Minister national plan for rare diseases in collaboration with provinces Oct 2010: Health Canada consults on orphan drug regulations May 2011: Provinces signal interest in collaborating on drugs for rare diseases Jul 2011: Ontario announces bulk purchase of drug for rare disease

6 Need for Canadian Rare Disease Policy q Definition of Rare Disease q Orphan Drug Policy q Access to Diagnosis, Treatment, Social Services q Research on Rare Diseases q Rare Disease Centers of Expertise/Reference q Screening, including Prenatal and Newborn q Empowered Patient Organizations

7 Challenges without Orphan Drug Policy No definition, no orphan drug policy; few orphan drugs. Many Orphan Drugs in USA and EU In USA: approximately 300 products in 26 years of Orphan Drug Legislation In EU, 75 new orphan drugs in 10 years Benefit: estimates of up to 10 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 Concerns over separate and slower approval and lack of reimbursement 7 Data Uncertainty/Rare Disorders 3/13/09

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19 Milestone: Canada Draft Regulatory Framework Access to Drugs Canada Define Orphan Drug Patient Population as < 1 in 2,000 Unmet need (no existing therapy) Benefits with OD Status Early protocol advice, including binding advice, without charge; Priority review for market authorization; Benefit-risk takes into account small size of populatio Remission or waiver of filing fees Harmonization with FDA and EMA Orphan Drug Committee Review and recommend orphan status to Health Canada 19 May 2010

20 HTA Roadblocks For Orphan Drugs 20 CDR same HTA 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 HTA Roundtable 19 March 2007

21 CDR Recommendations Drug Indication Date recommend Replagal Fabry s Nov 24, 2004 No Zavesca Gaucher s Nov 24, 2004 No Fabrazyme Fabry s May 18, 2005 No Aldurazyme MPS I Jul 14, 2005 No somavert Acromegaly Aug 2, 2006 No Exjade Iron Overload Apr 19, 2007 With Conditions Nexavar Kidney Cancer Feb 28, 2007 No Sutent (Renal) Kidney Cancer Apr 26, 2007 No Sutent (GSIT) GIST Mar 28, 2007 With Conditions Myozyme Pompe s Jun 14, 2007 With Conditions Elaprase MPS II Dec 19, 2007 No Xyrem Narcolepsy Jan 28, 2009 No Revlimid MDS JODR - No Catena Friedreich s Ataxia No Soliris PNH Feb 20, 2010 No Naglazyme (MPS VI) Not Submitted ND Ilaris CAPS Jan 26, 2011 No Kuvan PKU Jan 26, 2011 NO Vpriv Gauchers Apr 28, 2011 With CondiAons Cayston CysAc Fibrosis Jul 18, 2011 Yes total CDR NO 70% Yes/With CondiAons 25% 21

22 Issues Cited in Recommendation 22 n=12 drugs cited as positive factor cited as negative factor not cited Is there biological rationale? 67% 0 33% use of (only) surrogate Markers? clinical endpoints proven or linked? evidence of quality of life benefits? concerns about adverse events? data from (Lack of) Longterm studies 0 83% 17% 8% 92% 0 8% 67% 25% 0 83% 17% 8% 75% 17% appropriate control group? 0 17% 83% statistical significance in findings? COST EFFECTIVENESS: EXCESSIVE $/QALY 0 42% 58% 0 100% 0

23 Access to Drugs for Rare Disorders 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 23

24 Pathways Toward Orphan Drug Access : FPT Proposed national strategy for drugs rare disorders Response to geographic imbalance of some rare disorders, causing disproportionate impact and creating incentives for patients to move Based on pilot program for ERT and Fabry s Disease Evidence-based guidelines defining eligible patients Time-limited to allow for review and decision to continue funding Potentially harmonious with HC Progressive Licensing Framework May or may not require CDR recommendation 2008: National strategy abandoned 2008: Quebec review of drugs for rare metabolic disorders 2008: Alberta announces Drugs for Rare Diseases (no details) 2008: Ontario launches DRD Program based on Markov model 2010: Ontario Citizen s Council asked to recommend on conditions for funding rare disease drugs 2011: Bulk purchase agreement for national access to orphan drug HTA Roundtable 19 March 2007

25 Advocacy Gateways to Drug Access 25 Advocacy for access to drugs for rare disorders Province-by-province advocacy difficult, time-consuming Despite no recommendations, have achieved access for almost every drug (though not for all patients and not in all provinces) Meeting with drug plan managers, persona testimonies, political appeals, use of media Drug Access Strategies Individualized protocols (based on clinical judgment) with individualized targets (based on evidence) Managed access, based on modeling, limited use criteria, risk sharing Coverage with evidence developing Data Uncertainty/Rare Disorders 3/13/09

26 Experience in Drug Access Options Examples of controlled coverage schemes Hospital-controlled access (with drug plan reimbursement) (MPS I) Named Expert Physician/Clinic (Inherited Metabolic Disorders, Pulmonary Hypertension) Individual Client Review/Case-by-Case (Kidney Cancer) Common Drug Review Process/Provincial (Pompé s) Evidence-Based Modeling - Selective Patient Access (MPS II, Pompé s)

27 Experiences in Controlled Funding Examples of controlled funding schemes Envelope Funding (Cystic Fibrosis, Gaucher s) Special Access (MPS VI) Limited Access Listing (Pulmonary Hypertension, Iron Overload) Research Study (Fabry s)

28 Patients We Must Not Leave Behind No Labeled Indication (Off-Label) Older drugs (Growth hormone for Prader-Willi) where use over time has evolved to standard of care Evidence based on non-rcts and real usage (Alpha-1 Antitrypsin) Combination or sequential treatments Usage based on extrapolation from clinical trials to real-life patients (advanced renal cancer) Targeted therapies requiring screening and testing Genetic-based therapies (lung, breast, prostate cancer) Expanded patient population based on mechanism of action CTs limited to specific patients (MPS II with cognitive involvement)

29 Near Horizon for Rare Diseases in Canada All jurisdictions accept <1 in 2,000 as definition of rare disease Orphan Drug Regulatory Framework and Legislation similar to FDA and EMA (and updated as others evolve) National standards for diagnosis, treatment and support similar across all provinces and best in developed world Research on Rare Diseases with support from public and private sectors Rare Disease Centers of Expertise/Reference National standards on prenatal screening with appropriate genetic counselling equal to best in world Empowered Patient Organizations with support from all sectors, including government and private