Transcript Moving Forward on Healthcare Prioritisation

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
• Near-universal lack of good outcome
information, even for pharmaceuticals
• Especially for long-term outcomes in realworld settings
• RCTs should be built into outcomeassessment infrastructure – Scandinavian
registries incl. W Denmark good example
• Most future information will come from
routinely collected observational data
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
• 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
(http://www.fda.gov/ForConsumers/ByAudience/ForPatientAdvocate
s/SpeedingAccesstoImportantNewTherapies/ucm128291.htm;
updated 28 May 2010
• Kolata G. Evidence Gap: New Arena for Testing New Drugs: Real
World. New York Times 24 November 2008
http://www.nytimes.com/2008/11/25/health/research/25trials.html?_r
=2&pagewanted=all
Canada’s Progressive Licensing
Framework
•
Introduced into legislation 2008
•
Would permit “flexible departure” from usual standards for licensing
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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 premarket assessment. This represents a fundamental shift from the idea that the pre-market testing
of a drug assures its safety and efficacy.” http://www.hc-sc.gc.ca/dhp-mps/alt_formats/hpfbdgpsa/pdf/prodpharma/proglic_homprog_concept-eng.pdf, p.4
Initial Reaction Mixed
• Manufacturers, patient groups support Ottawa's new
drug safety proposals. CBC News April 10, 2008
http://www.cbc.ca/health/story/2008/04/09/drug-bill.html
• 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
• “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?”
(http://www.newscientist.com/article/mg1982652
3.800-canada-to-release-trial-drugs-topatients.html)
• Concerns about ‘guinea pig’ fear could mitigate
concerns about differential access – need to find
right balance
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 costeffective 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
• In a perfect position to serve as world’s realworld 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 payfor-results approaches
• Could negotiate favourable drug prices (free?)
PHARMAC
• 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
• 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
• 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
• 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 intratx. No outcome data.
• CMS recently negotiating with NOPR for more
comprehensive data on PETs it pays for
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
•
•
•
Multiple sclerosis risk sharing scheme: a costly failure
BMJ 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 Abrams
BMJ 2010 340: c1786. [Extract] [Full Text]
•
Commentary: Outcome measures were flawed G C Ebers
BMJ 2010 340: c2693. [Extract] [Full Text]
•
Commentary: Scheme has benefited patients Alastair Compston
BMJ 2010 340: c2707. [Extract] [Full Text]
•
The multiple sclerosis risk sharing scheme Neil Scolding
BMJ 2010 340: c2882. [Extract] [Full Text]
Italy’s outcome-based schemes
•
•
•
•
•
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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
•
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
• 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
• http://www.ema.europa.eu/pdfs/human/reg
affair/50995106en.pdf 2006
• “Conditional marketing”
• Restricted to serious or emergency use
PLF Reports
• Health Canada. Real World Drug Safety and
Effectiveness. 10.11.2006
• http://www.hc-sc.gc.ca/hcs-sss/pharma/npssnpp/securit/index-eng.php
• Approaches to Strengthening the Evaluation of
Real World Drug Safety and Effectiveness 2006
• http://www.hc-sc.gc.ca/hcs-sss/pharma/npssnpp/securit/guide_4-eng.php
• Bouchard RA, Sawicka M. Canada’s
Progressive Licensing Framework for drug
approval. McGill J Law Health 2009; 3: 50-84.