Increasing The Worldwide Supply Of Safe, Affordable Factor Replacement Therapy

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Increasing The Worldwide Supply Of Safe,
Affordable Factor Replacement Therapy
Mark W. Skinner WFH President

• 26 September 2005 Fourth WFH Global Forum -
  Montreal, Canada

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Thank You!

• We gratefully acknowledge the support of the
• André de la Porte Foundation
• Canadian Blood Services
• Héma-Québec
• Quebec Ministry of Health and Social Services
• Quebec Ministry of International Relations
• Public Health Agency of Canada

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Prevalence Of Bleeding Disorders

• Hemophilia A and B
• 400,000 people
• 200,000 severe
• 121,000 known to WFH
• 93,116 FVIII
• 18,830 FIX
• Von Willebrand
• 600,000 people
• 43,000 known to WFH
• Very rare bleeding disorders non FVIII, FVIX,
  VWD
• 10,496 known to WFH - no global estimate

• Making Progress!
• 5 increase in patients diagnosed
• GAP countries Now diagnosing 5 new
  patients/day

Source 2005 WFH Global Survey
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Key WFH Objective

• Provision of safe, efficacious, affordable
  replacement therapy in adequate amounts as part
  of a sustainable national hemophilia program.

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Todays Reality

• Availability of concentrates varies according to
  economic capacity
• 75 lack adequate treatment

Source WFH Global Survey
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Clotting Factor Essential!

• The Committee recommended that factors VIII
  and IX concentrates be retained on the Model
  List, accepting the inherent inconsistency caused
  by the fact that haemophilia is a rare disease.
  The Committee also recommended that a policy
  advisory group on rare diseases be established to
  determine a more general approach to the
  management of rare diseases.

"Essential medicines are those that satisfy the
priority health care needs of the population.
Source WHO What Are Essential
Medicines?
Source Report of the WHO Expert Committee on
the Selection and Use of Essential medicines
(7-11 March 2005, unedited report)
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The Need

• Increased worldwide supply
• of safe affordable
• factor replacement therapy

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Integration Of Stakeholders

• The Winning Coalition!
• Patients and their families
• Health care professionals
• Physicians - Nurses - Hospitals
• Orthopedists - Physiotherapists - Dentists
• Social Workers - Lab Technicians - Blood
  Services
• Governments / Health Ministries
• Vital Strategic Partners
• Regulators
• Industry

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Increasing The Worldwide Supply

• Twin Economic Challenges
• Bringing patients to market
• Bringing products to market

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Increasing The Worldwide SupplyBringing Patients
to Market

• Outreach identification
• Health care provider education
• Laboratory diagnosis (training)
• Registry
• Data collection analysis
• Development of a case for support
• Government purchase, tender or reimbursement

WFH Core Expertise Building Sustainable Care
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Building Sustainable Care

• If We Build It,
• Will Affordable Treatment Follow?

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Increasing The Worldwide SupplyMultivariate
Approach

• Management of existing resources
• Utilization of all treatment options
• Developing new markets
• New indications
• Identify new patient populations
• Reducing manufacturing cost
• Innovative pricing structures
• Humanitarian aid

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Increasing The Worldwide SupplyTreatment
Guidelines

• Replacement Therapy
• Management of existing resources
• Varying aims of treatment
• Optimal dosing

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Plasma-Derived RecombinantBoth Important

• Robust record of safety for both
• Supply of either alone insufficient to meet need
• Range of product cost (economic capacity)
• Patient or physician selection / preference
• Differences in therapeutic benefit
• Treatment for very rare bleeding disorders vWD
• Maintain flexibility for unknown unforeseeable

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Limitations Of Replacement Therapy

• Unknown pathogen risk
• Inhibitor risk management
• Frequency of treatment (short half-life)
• Factor replacement is a therapy, not a cure

Global Collaboration Required
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Increasing The Worldwide SupplyBetter Safer Cryo?

• The Committee noted that factors VIII and IX
  concentrates are life-saving in the treatment for
  haemophilia, and that the alternative
  (cryoprecipitate of whole blood) is less safe and
  more expensive. In addition, use of the
  concentrates avoids the risk of acquiring HIV and
  hepatitis. Another argument was that national
  blood transfusion services were often built
  around the production of plasma fractions.

Source Report of the WHO Expert Committee on
the Selection and Use of Essential medicines
(7-11 March 2005, unedited report)
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Increasing The Worldwide SupplyVery Rare
Bleeding Disorders

• WFH vision
• To improve access and treatment options
• Very rare disorders - limited therapeutic options
• Multinational studies may be required to obtain
  adequate patient numbers
• Encourage synchronization of European US
  regulatory agencies to prevent repetitive work
  increased financial burden on manufacturers
• Due to rarity of these disorders and lack of
  global availability of therapeutic products,
  trials such as those performed in hemophilia may
  not be feasible

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Patients With Other Heritable Bleeding
DisordersCountries Reporting gt 100 Dx
10,496 Identified
Source WFH Global Survey 2005
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Increasing The Worldwide SupplyUnused Plasma
Proteins

• WFH vision
• To find ways to ensure that no FVIII and FIX
• Proteins are wasted
• In many countries, the FVIII / FIX contained in
  the plasma destined for fractionation is
  discarded
• E.G. Canada 150,000 liters of plasma are
  processed annually
• Assuming a low FVIII / FIX recovery of 10
• 15 million IUs FVIII / 30 million IUs FIX lost
  annually
• Discarded fractions containing FVIII / FIX will
  increase

CBS, Héma-Québec, NBI, WFH Innovative
Collaboration!
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Increasing The Worldwide SupplyRegulatory
Harmonization

• Influence of Key Regulators
• FDA and EMEA play vital role globally
• Decisions made independently
• Decisions monitored by many countries
• Decisions impact products used outside E.U. and
  U.S.
• Licensing confers reassurance

• WFH Interest
• Enhanced portability between countries
• Increased product selection within countries
• Lower costs ? Lower Price
• Open markets
• Increased supply and supply stability

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Increasing The Worldwide SupplyRegulatory
Harmonization

• WFH vision
• To provide the catalyst for change
• WFH will examine regulatory issues where there is
  a potential for a direct benefit to patients
  resulting in improved product cost, choice and
  access to care
• Issues such as product portability, impediments
  to market entry, new product development,
  licensure,
• For example ..
• Harmonization of labeling requirements
• Harmonization of potency testing

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Increasing the Worldwide SupplyTwin-Track Pricing

• WFH vision
• To create strategic and targeted access to
  products at an affordable price
• Lower price for developing/emerging countries
• Specific agreed inclusion criteria
• WHO and industry have expressed willingness to
  move forward
• Development costs recouped from developed
  countries
• Historical precedent HIV/AIDS, vaccines

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Increasing The Worldwide Supplyvon Willebrand
Disease Women

• WFH strategic vision
• To improve diagnosis and access to treatment
• Fully incorporate
• von Willebrand patients
• Women with bleeding disorders
• Expand educational and training programs
• Improve diagnosis
• Access to appropriate treatment

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Patients with von Willebrand DiseaseCountries
Reporting gt 100 Dx
43,000 Identified
Source WFH Global Survey 2005
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Increasing The Worldwide Supply

• Welcome dialogue and discussion
• Open to exploring other avenues
• Encourage innovation research
• Eager to engage, collaborate and partner with all
  stakeholders

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Congress Program Plenary Sessions

• Building for the Future Facing the challenges,
  seizing the opportunities
• Musculoskeletal issues total joint replacements
  in patients with inhibitors
• Molecular basis of vWD
• Pathogenesis of Hemophilic Arthropathy
• Strategies toward a longer-acting factor VIII
• Uterus A recycling model for the physiology of
  coagulation and Fibrinolysis
• Why do inhibitors develop?
• Future of comprehensive care for inherited
  coagulation disorders

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See You There!
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