Conditional Coverage: past experience and future trends in the US and the UK

1
Conditional Coverage past experience and future
trends in the US and the UK

• Kalipso Chalkidou
• NICE, UK

2
Capturing value the payers toolbox

• Direct
• Value-based pricing
• Indirect
• Using comparative effectiveness data for coverage
  decisions (NICE)
• Coverage with evidence development / only in
  research
• Risk sharing schemes

3
UK developments in drug pricing

• We recommend that Government reform the PPRS
  replacing current profit and price controls with
  a value based approach to pricing to ensure the
  price of drugs reflect their clinical and
  therapeutic value to patients and the broader
  NHS.

OFT, February 2007
4
But NICE does not do pricing!

• NICE does not negotiate or set prices for medical
  technologies in the UK

5
NICE Only In Research

• In the case of promising interventions not yet
  supported by sufficiently robust evidence to
  justify an unqualified recommendation, the
  Institute will
• recommend that further research is carried out to
  see whether the potential promise of the
  intervention can be realised,
• indicate in broad terms the questions this
  research should address and
• advise clinicians that, in the meantime, they
  should only use the new intervention as part of a
  well-designed programme of research intended to
  answer these questions
• Department of Health, Faster Access to Modern
  Treatment How NICE Appraisal Will Work. London,
  1999, paragraphs 33-36

6
Policy and research a two-way interaction

• NICE evidence-informed guidance for best
  practice
• Comparative clinical effectiveness
• Comparative cost-effectiveness
• Legal framework
• Social value judgements and equity impact
• Systematic identification and prioritisation of
  evidence gaps
• Direct access with NHS RD programme
• Over 10 studies advertised or commissioned since
  June 2007
• CCBT vs. psychotherapy vs. drugs for adolescents
  with depression (RCT) bariatric surgery long
  term costs and effects (registry) improving
  efficiency in pre-op testing for elective surgery
  (review and decision model)

7
How does it work?

• Who pays for the research?
• Public
• Private
• Mixed
• What happens in the meantime? Safety and access
  issues
• Registries, prospective cohort studies
• RCTs practical clinical trials
• Are there mechanisms in place for updating advice
  based on evidence?
• NICE regular review and update based on new
  evidence

8
Management of localised CaP UK

• Cryotherapy and HIFU licensed by NICE for their
  safety and efficacy Interventional Procedures
  guidance
• NICE Guideline February 2008 considered new
  technologies IMRT HIFU cryotherapy
• Lack of comparative effectiveness data long-term
  survival and QoL
• Threshold economic analysis showed these would be
  cost effective with modest increases in QoL, but
  
• Due to weak evidence of clinical effect
• High-intensity focused ultrasound and
  cryotherapy are not recommended for men with
  localised prostate cancer other than in the
  context of controlled clinical trials comparing
  their use with established interventions
• Proton Beam Therapy not included

9
NICE is aware that if all the cases of prostate
cancer lurking in the glands of older men were
treated regardless of cost it would bankrupt the
NHS.
10
USA Proton Beam diffusing fast
11
CMTP initiative PBT vs. IMRT

• AHRQ Review-- Evidence for Tx of Clinically
  Localized Prostate Cancer
• Limited evidence on relative safety and
  effectiveness associated with major treatment
  options
• Particular concern regarding PSA-detected tumors
• New technologies (laparoscopic surgery, robotic
  assisted) being rapidly adopted with little study
• Rigorous trials needed to directly compare
  treatment options for effectiveness or adverse
  effects
• ICER review of clinical and cost effectiveness
• Weak clinical evidence
• Very high /adverse event prevented
• CTAF IMRT review
• Little evidence but radiation oncologists
  supportive
• CMTP invited to explore opportunities for
  collaborative evidence development

12
Prostate Cancer Workgroup

• ASTRO
• Camino Medical Group
• CET Cancer Center
• MD Anderson CRC
• Fred Hutchinson CRC
• Patient Representative
• University Researchers
• ICER

• AHRQ
• NCI
• CMS (unofficial)
• Aetna
• Wellpoint
• Blue Shield California
• Siemens Medical
• Varian Medical Systems

13
Examples of Medicare CED

• Lung volume reduction surgery
• FDG-PET for suspected dementia
• Implantable defibrillators
• Off-label use of drugs for colorectal cancer
• FDG-PET for oncology
• Home testing for sleep apnea
• Artificial heart
• Coronary CT angiography (almost)

14
Medicare Evidence Standard

• Adequate evidence to conclude that the item or
  service improves net health outcomes
• Costs not considered
• Though evidence reviewed more carefully for
  high cost interventions
• Debate focuses on quality of evidence, or degree
  of uncertainty
• Most drugs (parts B and D) covered for
  FDA-approved indications

15
Medicare Review of CCTA

• EPC report from Duke (April 2006)
• Limited evidence of clinical utility in any
  population
• Suggest RCTs, natural experiments or models
• MedCAC mtg (May 2006)
• Uncertain confidence about existing evidence
• CMTP convened workgroup to design study
• Medicare LCDs provide coverage of CCTA
• Used ACCF/ACR/SCCT appropriateness criteria
• CMS opened NCD in June 2007
• Draft NCD in 12/07 proposed CED

16
New York Times, June 2008
And Medicare gave way. There are a lot of
technologies, services and treatments that have
not been unequivocally shown to improve health
outcomes in a definitive manner, Dr. Barry
Straube, Medicares chief medical officer,
explained when announcing that the agency would
keep covering the tests. In other words, the
lack of evidence that the CT scans provide
measurable medical benefit would not stop
Medicare from paying for them.
17
Coverage Without Evidence Development

• Payers/researchers
• Want PCT with low/mod risk, CCTA vs. other
  non-invasive dx
• Acute MI and death at 2 years
• 20k patients estimated sample size
• Vendors / clinicians
• existing evidence sufficient
• Clinicians prepared to launch registry
• Medicare final decision (March 2008)
• No adequately designed studies show improved
  outcomes
• We believe large, well-designed prospective
  trials needed
• Broad coverage by local contractors retained

18
Contact Information

• sean.tunis_at_cmtpnet.org
• www.cmtpnet.org
• 410-963-8876

19
US and UK how different?

• New (unevaluated) technologies diffusing fast
  unwarranted variation
• Policy makers (different perspective in US and
  UK) need more information
• CED/conditional coverage a means for dealing
  with uncertainty
• Convergence in the UK and the US (often for
  different reasons!)
• Opportunity for collaboration through trials and
  data analyses
• HTAi Special Interest Group Use with Evidence
  Development
• sean.tunis_at_cmtpnet.org kalipso.chalkidou_at_nice.org
  .uk

20
US and UK different viewpoints
No, unless
Yes, but
21
NICE only in research 1999-2007

• Approx. one in 20 NICEs technology appraisal
  recommendations published between 1999 and 2007
  were only in research
• Almost half (46) of procedures reviewed by the
  interventional procedures programme, recommended
  with special arrangements for clinical
  governance, research and audit
• Clinical and public health guidance
• OIR usually for diffused public health
  interventions and management strategies

22
Risk sharing and pricing reform OFT

• Where sufficient information at the time of
  launch was not available to take an informed
  viewrisk sharing arrangements can help
  coordinate the expectations of the payer and
  manufacturersallow for more predictable uptake
  for manufacturers, and predictable health gains
  for a given expenditure when an agreement may
  not be able to be reached otherwise.
• OFT Report, Feb 2007

23
Risk sharing and negotiating price
24
NICE risk sharing schemes

• b-IFN and glatiramer for multiple sclerosis -
  2002
• Uncertainty around clinical effect
• Bortezomib for multiple myeloma -2007
• NO uncertainty around clinical effect
• Ranibizumab dose capping scheme - 2008
• NO uncertainty around clinical effect
• Erlotinib for SCLC cost capping scheme - 2008
• NO uncertainty around clinical effect

25
Uncertainty as a political problem

• CEA closer to licensing NICE single technology
  appraisal
• Methodological fix
• Value of Information analysis
• NICE Methods Manual - 2008
• Ongoing debate
• Evidence of lack of effectiveness vs. lack of
  evidence of effectiveness
• Whos responsible for producing the information?
• Should/can we say no when we do not know?

26
What does the public think? Citizens Council

• In what circumstances is it justified for NICE to
  recommend that an intervention is used only in
  the context of research?
• Feasibility
• Access
• Timeliness
• Value for money of research
• Implications of positive decision

• Patients would be reassured to know that
  clinicians and the healthcare system in general
  could face up to uncertainty, and were confident
  enough to deal with it in a mature, scientific
  way, and avoid wasting money on unproven
  technologies

27
New critical path collaboration in evidence
generation

• Industry, research funders and regulators
• HTA and NICE should seek to work with the ABPI
  and individual pharmaceutical companies to
  identify new medicines under development that
  might be best suited to piloting earlier HTA /
  NICE involvement (Phase III)
• NICE scientific advice service to industry

28
NICE and NHS RD Direct access

• a more systematic approach to and expansion of
  HTA, ensuring that key NICE recommendations for
  further research are followed up, and creating a
  system for following up initial assessments
  using data emerging from post-launch use
  Cooksey Report, 2006
• Direct access NICE priority research
  recommendations
• head-to-head trials
• little incentive for private sponsor support
• inform NICE guidance updates

29
NICE/NIHR comparative effectiveness research
agenda (Jan 08)
30
Professional-led initiatives

• Antihypertensives head-to-head trial (ANBP2) -
  with PBAC funding support (Australia)
• Extra Corporeal Membrane Oxygenation trial -
  Society of Neonatologists (UK)
• Hip registry - Association of Orthopaedic
  Surgeons (UK) with NICE and NHS support
• Avastin vs. Lucentis trial - Ophthalmic Surgeons
  (UK) with NHS RD and NHS Commissioners support

31
International examples

• France
• EUnetHTA Commission initiative to promote
  evidence generation for new drugs
• Drug pricing based on HAS determined tiers
  currently only clinical effectiveness but
  economic evaluation being considered as
  additional input
• Germany
• New Social Insurance Code ceiling price on
  innovations
• Economic evaluation required by law (2008) for
  IQWiG
• Potential for prospective studies, especially for
  medical devices, upon IQWiG request
• The Netherlands
• Conditional reimbursement for expensive inpatient
  and ultra-orphan drugs
• Italy
• 5 tax on marketing to fund comparative
  effectiveness research
• Used funding to undertake head-to-head trial of
  b-IFN against azathioprine the latter shown to
  be better value
• USA
• Coverage with Evidence Development for Medicare
  recent (Aug 2008) alteration in CMS payment
  language to allow randomisation
• Institute for Comparative Effectiveness Research
  multiple bills latest Conrad/Baucus Aug 2008

32
Thank youkalipso.chalkidou_at_nice.org.uk