Funding for Haemophilia Care in Europe: A Taste of Things to Come

1
Funding for Haemophilia Care in Europe A Taste
of Things to Come?

• Adam Hutchings
• GMAS LLP
• WFH 24 September 2009

Disclosure Adam was previously employed by
Baxter Healthcare and currently provides
consultancy services to pharmaceutical companies,
including Baxter Healthcare
2
Until now
Haemophilia care in Europe has been well funded
in the recent past

• Funding has mostly kept track with improving care
  levels in Western Europe
• Prophylaxis treatment has become standard of care
  in children
• Total FVIII usage has increased steadily over 20
  years
• Patients are receiving surgery when required
• Majority of patients are managed in specialist
  haemophilia treatment centres
• Substantial amount of research has been funded
  and conducted (basic science, pharmaceutical, and
  disease management)

European FVIII Usage
Source PPTA IMS GERS UKHCDO
3
Haemophilia care has not been a primary target
for cost savings
Until now

• Funding has often been provided direct to HTCs
  from central government/sick funds
• No Health Technology Assessment of haemophilia
  treatment approaches to date
• Most new products launched in the last 10 years,
  all are available for use in nearly every market.
• (Anecdotally) most patients who need it are
  receiving orthopaedic surgery including
  patients with FVIII inhibitors

4
Causes for concern
European public finances are deteriorating
Fiscal balance as of GDP
Source IMF. The state of public finances
outlook and medium term policies after the 2008
crash.
5
Causes for concern
Competition for funding within specialty care is
increasing
New drugs designated for the treatment of rare
diseases in Europe 2000 - 2009.
Source European Commission. European orphan drug
designations 200-2009
6
Causes for concern
Haemophilia expenditure is now very visible
In many European countries, FVIII represents one
of the biggest area of pharmaceutical expenditure
Source Swedish National Board of Health and
Welfare (2008)
7
Economics of haemophilia are not unambiguous
Causes for concern
Published cost per QALY estimates of FVIII
prophylaxis in children
50,000 per QALY is often considered to be the
maximum that healthcare providers will pay for an
intervention or treatment
8
Perception of unmet need in haemophilia is
diminishing?
Causes for concern

• Multiple effective treatments available
• Product safety has progressed dramatically
• Life expectancy is rising
• Patient quality of life is close to population
  norms
• Patients are starting to develop conditions of
  elderly (e.g osteoporosis)
• Knowledge of disease history is lower in younger
  payers

9
Health Technology Assessment
Possible threats

• HTA is a systematic method for assessing the
  relative costs and benefits of a treatment or
  intervention
• It is increasingly common in Europe (e.g NICE in
  the UK, IQWIG in Germany)
• First HTA of haemophilia treatment is ongoing in
  Sweden (TLV)
• Possible outcomes
• Maintain status quo
• Certain factors removed from reimbursement
• Certain treatment protocols recommended over
  others (on-demand vs prophy?)

EU countries that use HTA to ration healthcare
10
Capped haemophilia budgets
Possible threats

• Capping haemophilia budgets at a national/local
  level moves the responsibility for rationing
  decision to the physician
• Funding caps would require one or more of the
  following actions
• Reduce factor usage (constrain prophylaxis
  regimens reduced ITI)
• Use cheaper products
• Restrict access to surgical interventions
• Seek savings from HTC operating expenses

11
National factor tenders
Possible threats
Done badly, they can limit prescriber freedom,
endanger supply, and reduce complex treatment
decisions to the level of hospital toilet paper
procurement.
Done well, they can provide a strong framework
for procurement securing a safe supply of factor
and bringing together patients, physicians and
payers in a collegial decision making process.
12
Defending funding
Be aware

• People often say it couldnt happen here, until
  it does
• Physicians and patient representatives need to
• Understand local payer systems, influencers and
  decision makers
• Learn from encounters in other disease areas
• Share experience with international colleagues
  (payers do!)
• Be vigilant changes can happen quickly without
  much prior notice

13
Defending funding
Be involved

• Establish relationships with policy makers and
  payers
• Educate them about haemophilia
• Early warning about impending action
• Allows opportunity to sculpt the nature/scope of
  any review
• Proactively develop local guidelines
• German example annual haemophilia roundtable
  with G-BA, patient representatives, physicians
  and industry

14
Defending funding
Be prepared

• Data is key to defending treatment levels
• Clinical
• Economic
• Quality of life
• Experiential
• These can be collected through
• Patient records
• Registries
• Clinical trials
• Patient surveys
• Patients need to understand that the quality of
  their future care is dependent upon their
  participation in data collection programs

15
Defending funding
There are better ways for payers to reduce
healthcare expenditure!
Over 70bn of annual expenditure on blockbuster
drugs will go off patent in the next 5 years
Source company annual reports.