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Progressive Drug Licensing for New Zealand?. David Hadorn, M.D., Ph.D. Director, Centre for Assessment and Prioritisation Dept of Public Health U Otago Wellington School of Medicine 1 July 2010. Information Requirements for Service Assessment.

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progressive drug licensing for new zealand

Progressive Drug Licensing for New Zealand?

David Hadorn, M.D., Ph.D.

Director, Centre for Assessment and Prioritisation

Dept of Public Health

U Otago Wellington School of Medicine

1 July 2010

information requirements for service assessment
Information Requirements for Service Assessment
  • Near-universal lack of good outcome information, even for pharmaceuticals
  • Especially for long-term outcomes in real-world settings
  • RCTs should be built into outcome-assessment infrastructure – Scandinavian registries incl. W Denmark good example
  • Most future information will come from routinely collected observational data
new zealand in unique position
New Zealand in Unique Position
  • NZ is the only country with universal health data based on unique ID #
  • Able to link across many health databases
  • PHOs largely responsible for usefulness
  • NZ could become major resource for assessing health outcomes and treatment effectiveness
  • Most obviously in pharmaceuticals
evolving shift in risk attitude for drug licensing
Evolving shift in risk attitude for drug licensing
  • Worldwide move away from precautionary principle and towards improving early access
  • Based on risk management principle
  • How much risk should people be allowed to take?
  • Growing emphasis on real-world assessment over drug’s ‘life-cycle’
usa fda
  • IOM report (2006) and FDA’s Sentinel Initiative (2008) for life-cycle assessment
  • Fast Track, Accelerated Approval and Priority Review Accelerating Availability of New Drugs for Patients with Serious Diseases
  • Allows the agency to approve a drug to treat serious diseases with an unmet medical need based on a surrogate endpoint (; updated 28 May 2010
  • Kolata G. Evidence Gap: New Arena for Testing New Drugs: Real World. New York Times 24 November 2008
canada s progressive licensing framework
Canada’s Progressive Licensing Framework
  • Introduced into legislation 2008
  • Would permit “flexible departure” from usual standards for licensing
  • Community access to drugs under monitored ‘real-world’ conditions following demonstration of safety and efficacy in Phase I and II trials, in addition to or instead of Phase III RCTs
  • Threshold for marketing is where ‘benefits outweigh the risks”
  • Trade off: less scrutiny pre-market in exchange for ‘life-cycle-long’ post-market assessment
  • Scheme possible because of good Provincial data (on over-65’s)
  • “The emphasis of the new framework is to identify opportunities [for assessment] within the progression over the full life-cycle of a drug, rather than placing the focus primarily upon pre-market assessment. This represents a fundamental shift from the idea that the pre-market testing of a drug assures its safety and efficacy.”, p.4
initial reaction mixed
Initial Reaction Mixed
  • Manufacturers, patient groups support Ottawa's new drug safety proposals. CBC News April 10, 2008
  • Experts Sound Alarm on Drug Approval Plan: Under Sweeping New Changes, Drug Companies Only Have to Prove That Benefit of Product Outweighs the Harm. Globe and Mail 9 April 2008.
canada to release trial drugs to patients new scientist 21 april 2008
Canada to release trial drugs to patients. New Scientist 21 April 2008
  • “Are Canadians about to be turned into guinea pigs for testing drugs? Or will they be blessed with a pioneering system of ‘smart’ regulation that sweeps aside the usual obstacles between new drugs and patients?” (
  • Concerns about ‘guinea pig’ fear could mitigate concerns about differential access – need to find right balance
plf may be used for funding decisions
PLF may be used for funding decisions
  • “Ongoing assessments of the new information that emerges about a drug can be used to help all decision makers in the health care system: . . . provincial and private funders . . . Is this a cost-effective therapy for our drug plan?” Ibid., p.16
  • “. . . optimize resource management” p.22
  • “Do provincial payers pay for the drug at this early stage? What sort of grounds do they need to be able to justify paying?” p.24
new zealand should consider progressive licensing
New Zealand Should Consider Progressive Licensing
  • In a perfect position to serve as world’s real-world pharma ‘laboratory’
  • On national or international basis
  • Only way to achieve early-adopter status for pharmaceutical innovation
  • Would provide built-in post-market surveillance
  • Incorporate value-based purchasing and pay-for-results approaches
  • Could negotiate favourable drug prices (free?)
  • Created in 1993; goal is to produce ‘best outcomes’ within budget
  • Contractual-operational approach to prioritisation, e.g., maximal use of generics, reference pricing, sole supply, deal-making
  • All in context of fixed budget
  • Cost-utility and other decision criteria considered but most action is around deal-making
  • Major question: How generalisable is PHARMAC’s approach? Touted as possible model in other areas.
how plf compares with pharmac s current approach
How PLF Compares With PHARMAC’s Current Approach
  • PHARMAC’s current approach probably able to obtain lower prices through negotiation in context of fixed budget
  • Purchasing decisions almost independent of traditional value-for-money concerns
  • But: Is price focus with lack of outcome assessment or consideration of VFM the best approach?
  • Relevance to FTA negotiations
many questions to be addressed within plf
Many questions to be addressed within PLF
  • How to allocate patients? Who will pay? Role for large simple RCTs?
  • How can information be used to determine subsidy?
  • My main message is that it’s worth considering
  • Similar approach and questions could be applied outside pharmaceuticals, esp. implanted medical devices
pay by results risk sharing coverage with evidence development
Pay by Results – Risk Sharing – Coverage with Evidence Development
  • Medicare CED started with ICDs and PET scans used in workup of cancer
  • National Oncology PET Registry (NOPR)
  • NOPR data used in 2008 to decide to cover PET for initial w/u of pancreatic, cervical and ovarian but not prostate.
  • Scope of registry limited due to lobbying by radiologists and industry
  • Data inadequate to determine role of PET post- or intra-tx. No outcome data.
  • CMS recently negotiating with NOPR for more comprehensive data on PETs it pays for
politically difficult
Politically Difficult
  • CMS sought to cover CT angiography to detect CAD under CED, but lobbying from docs and industry made them back down and pay for it
  • Redberg R, Walsh J. Pay now, benefits may follow- the case of cardiac computed tomographic angiography. NEJM 2008; 359; 2309-11.
nhs ms trial 2 year results
NHS MS trial – 2 year results
  • Multiple sclerosis risk sharing scheme: a costly failureBMJ 2010; 340: c1672 James Raftery
  • Continuing the multiple sclerosis risk sharing scheme is unjustified Christopher McCabe, Jim Chilcott, Karl Claxton, Paul Tappenden, Cindy Cooper, Jennifer Roberts, Nicola Cooper, and Keith AbramsBMJ 2010 340: c1786. [Extract][Full Text]
  • Commentary: Outcome measures were flawed G C EbersBMJ 2010 340: c2693. [Extract][Full Text]
  • Commentary: Scheme has benefited patients Alastair CompstonBMJ 2010 340: c2707. [Extract][Full Text]
  • The multiple sclerosis risk sharing scheme Neil ScoldingBMJ 2010 340: c2882. [Extract][Full Text]
italy s outcome based schemes
Italy’s outcome-based schemes
  • Sorafenib for hepatocarcinoma,
  • Dasatinib and nilotinib for acute myeloid leukaemia,
  • Temsirolimus for renal-cell carcinoma
  • Pagaptanib and ranibizumab for age-related macular degeneration
  • Implemented in 2007
  • “Have allowed a large number of patients to be treated with very critical drugs in terms of cost -effectiveness.”
  • In addition, “the regulatory need to register these treatments on a national website has generated a database of all Italian patients receiving these treatments that will hopefully be used for nationwide observational studies.” De Ambrosis P. Risk sharing e rimborso in base al risultato. Dialogo sui farmaci 2008;:235-7.
other examples
Other examples
  • Garber AM, McClellan MB. Satisfaction guaranteed -- "payment by results" for biologic agents. N Engl J Med 2007;357:1575-1577. 
  • Australian scheme for bosentan in pulmonary artery hypertension more successful:
  • “Smaller patient group (528 patients), a well defined outcome measure (death), and a health system in which negotiation of drug prices is common. However, it too has struggled with issues of governance, ethical permission, and data collection.” (Raftery, BMJ, 2010)

Owen AJ, Spinks J Meehan A, Robb T, Hardy M, Kwasha D, et al. A new model to evaluate the long term cost effectiveness of orphan and highly specialised drugs following listing on the Australian pharmaceutical benefits scheme: the Bosentan patient registry. J Med Econ 2008;11:235-43.

uk nhs value based purchasing
UK/NHS Value-based Purchasing
  • Recommended by Office of Fair Trading 2007
  • Government has accepted need to shift to VBP
  • Alternative to current Prescription Price Regulation System
  • Based on costs/QALY
  • Would apply only to initial evaluation
  • No post-marketing assessment planned
  • Applies to all drugs, not just for life-threatening or emergency use
  • Claxton K, Briggs A, Buxton MJ, Culyer AJ, McCabe C, Walker S, Sculpher MJ. Valued based pricing for NHS drugs – an opportunity not to be missed? BMJ 2008;336:251-254 (2 February).
european union ema
European Union - EMA
  • 2006
  • “Conditional marketing”
  • Restricted to serious or emergency use
plf reports
PLF Reports
  • Health Canada. Real World Drug Safety and Effectiveness. 10.11.2006
  • Approaches to Strengthening the Evaluation of Real World Drug Safety and Effectiveness 2006
  • Bouchard RA, Sawicka M. Canada’s Progressive Licensing Framework for drug approval. McGill J Law Health 2009; 3: 50-84.