Brazilian Experience with drug policy for rare diseases Fábio Mataveli, MD, PhD Novartis Oncology, Brazil Former Brazilian...
Brazil Fact Sheet  <ul><li>Population: 188,696,389 people </li></ul><ul><li>Area: 8 ,514, 215.3 km2, 27 states </li></ul><...
Demographic Transition 1980 2010 Costs with chronic and rare disorders jumped from US$323,8 million  in 2002 to US$ 809,5 ...
Drug Access to Rare Diseases <ul><li>In Brazil, orphan drugs are included in a special drug access program.  </li></ul><ul...
Orphan drug policy started in the 70’s <ul><li>Brazilian government concern with rare diseases is not recent. </li></ul><u...
Orphan drug policy started in the 70’s Special drug stock  should be maintained  for the treatment  of patients with rare ...
Current Scenario <ul><li>There are 226 drugs listed and partially (80%) reimbursed by federal government as exceptional it...
New treatments options vs. Established Clinical Protocols <ul><li>Access to treatment is granted by the Brazilian Constitu...
Health Judicialization Burden <ul><li>In 2006, 30 % of exceptional drug dispensation </li></ul><ul><li>was done by Judicia...
Orphan Drug Prioritization Resolution Published April 4 th , 2007 – 60 days to start <ul><li>Definition of Orphan drug : D...
Orphan Drug Current Public Reimbursement  Process February 1 st  through March 31 st   August 1 st  through September 30 t...
HTA assessment for Rare disorders <ul><li>Evidence and Good Sense Based Medicine </li></ul>EBM EGSBM <ul><li>FAST TRACK AP...
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First Canadian Conference on Rare Disorders &amp; Orphan Products Policy - Brazilian Experience

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First Canadian Conference on Rare Disorders &amp; Orphan Products Policy - April 24-25, 2007 -Ottawa, Ontario
International Policies and Practices That Promote Access to Orphan Treatments - Brazil
International Invited Speaker

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First Canadian Conference on Rare Disorders &amp; Orphan Products Policy - Brazilian Experience

  1. 1. Brazilian Experience with drug policy for rare diseases Fábio Mataveli, MD, PhD Novartis Oncology, Brazil Former Brazilian Drug Regulatory Agency
  2. 2. Brazil Fact Sheet <ul><li>Population: 188,696,389 people </li></ul><ul><li>Area: 8 ,514, 215.3 km2, 27 states </li></ul><ul><li>GDP: US$ 1.616 trillion, with a per capita gdp of US$ 8,600. </li></ul><ul><li>Public Health Care System Coverage: Universal Access </li></ul><ul><li>Private Coverage: 25% of the actual population (≈ 44,7 million) </li></ul><ul><li>Public Health Annual Budget: US$ 21,76 billion </li></ul><ul><li>Total Annual Drug Expenditure: US$ 2 billion </li></ul><ul><li>Basic Assistance: US$ 34,29 million (1,7% budget) </li></ul><ul><li>AIDS Program: 170,000 patients, US$450 million/year </li></ul><ul><li>Exceptional Drugs (including rare disorders): US$ 666,6 million </li></ul>
  3. 3. Demographic Transition 1980 2010 Costs with chronic and rare disorders jumped from US$323,8 million in 2002 to US$ 809,5 million in 2005. 250% increase in 3 years
  4. 4. Drug Access to Rare Diseases <ul><li>In Brazil, orphan drugs are included in a special drug access program. </li></ul><ul><li>Each drug request must be submitted to a specific approval, in a process involving a referral public health care center, the state secretary of health and federal government. </li></ul><ul><li>Drugs are only obtained in Public HealthCare Centers </li></ul><ul><li>Drug dispensation occurs according to specific Clinical Protocols and are made patient by patient for a better control. </li></ul><ul><li>Drug costs are usually shared : Federal (80%) and State (20%) </li></ul><ul><li>Some States dispense drugs that are not in the federal list, but they have cover it’s cost 100%. </li></ul>
  5. 5. Orphan drug policy started in the 70’s <ul><li>Brazilian government concern with rare diseases is not recent. </li></ul><ul><li>1971 – Public health policy established that a special drug stock should be maintained for the treatment of patients with rare incidence diseases. </li></ul><ul><li>1976 – Brazilian government requested University of Brasilia to develop a program to locally produce Growth Hormone. </li></ul><ul><li>Patient advocacy group associated with Endocrynolgists of health public system pressured the government to provide GH for patients with hypophyseal nanism. </li></ul><ul><li>1977 – Drug dispensation according to a established Clinical Protocol for Nanism Treatment. </li></ul>
  6. 6. Orphan drug policy started in the 70’s Special drug stock should be maintained for the treatment of patients with rare incidence diseases. 1971 Brazilian government requested University of Brasilia to develop a program to locally produce Human Growth Hormone 1971 1977 Drug dispensation according to a established Clinical Protocol for Nanism Treatment 1982 15% of the total Drug Budget For Exceptional Drugs 1986 Reimbursement List with 55 items
  7. 7. Current Scenario <ul><li>There are 226 drugs listed and partially (80%) reimbursed by federal government as exceptional items in the last update. There are only four 100% federal reimbursed drugs: imiglucerase (Gaucher), eritropoeitin (Kidney failure), INF-α (hepatitis C) and Immunoglobulin (immunodeficiency). This 4 drugs alone account for a US$ 238,1 million annual expenditure. </li></ul><ul><li>Some drugs for Rare Disorders are pretty inexpensive Hydroxiyurea in Sickle Cell Anemia or Psoriasis: US$ 0,24/pill </li></ul><ul><li>States have autonomy to allocate resources to special drugs according to local demands </li></ul><ul><li>Although recent advances, the list must be updated </li></ul>
  8. 8. New treatments options vs. Established Clinical Protocols <ul><li>Access to treatment is granted by the Brazilian Constitution to all people living in Brazil ( citizens or not ) </li></ul><ul><li>The list of reimbursed drugs does not include all innovations </li></ul><ul><li>Based on the Constitution, Patients with rare disorders suit the public health care system to have access to new drugs, sometimes not yet approved by the Brazilian Drug Regulatory Agency </li></ul><ul><li>The acquisition of drugs not included in Clinical Protocols has an negative impact on the Public Healthcare System Budget. </li></ul><ul><li>Allocation of resources not provisioned for this purpose </li></ul><ul><li>Burden to local Public Healthcare State Budget </li></ul>
  9. 9. Health Judicialization Burden <ul><li>In 2006, 30 % of exceptional drug dispensation </li></ul><ul><li>was done by Judicial Order </li></ul><ul><li>If the decision is positive for the patient, Treatment must be provided in a maximum 24 hour period. </li></ul><ul><li>Disobedience to a Judicial Order: State or County Secretary of Health is arrested ( goes to jail ) </li></ul><ul><li>Not all drugs acquired by Judicial Order has proven efficacy </li></ul><ul><li>Need to update exceptional drug public reimbursement list </li></ul>
  10. 10. Orphan Drug Prioritization Resolution Published April 4 th , 2007 – 60 days to start <ul><li>Definition of Orphan drug : Drug that has shown proven efficacy in the treatment or diagnosis of rare or neglected diseases </li></ul><ul><li>Definition of Rare disease: low incidence </li></ul><ul><li>Deadlines for the Regulatory Agency: 75 days after the request for drug approval (registration) was considered a Priority. </li></ul><ul><li>IMPORTANT ISSUE: Brazilian Drug Regulatory Agency does not approve phase II drugs </li></ul>
  11. 11. Orphan Drug Current Public Reimbursement Process February 1 st through March 31 st August 1 st through September 30 th Reimbursement Dossier Submission CITEC Health Technology Assessment Committee <ul><li>Evidence: clinical Efficacy, </li></ul><ul><li>Effectiveness, Safety </li></ul><ul><li>Economic Evidence: local data, </li></ul><ul><li>QALY ( value not set </li></ul><ul><li>WHO: 3 x PIB per capita) </li></ul><ul><li>Comparison to existent </li></ul><ul><li>technologies </li></ul>NO YES Work on Reimbursement Value (minimize gap), Clinical Protocol (1 st or 2 nd line treatment, clinical indication)
  12. 12. HTA assessment for Rare disorders <ul><li>Evidence and Good Sense Based Medicine </li></ul>EBM EGSBM <ul><li>FAST TRACK APPROVAL – most of orphan drugs won’t reach phase 3 </li></ul><ul><li>Biotech Innovation – pay attention to truly new </li></ul><ul><li>technological Advances, what is for a small group today may </li></ul><ul><li>Be used for a large population tomorrow </li></ul><ul><li>Disease Incidence – search for the number of patients that </li></ul><ul><li>Make an evidence statistically significant </li></ul><ul><li>QALY is not the only pharmacoecomic measure and may </li></ul><ul><li>Underevaluate the benefits of some drugs </li></ul>
  13. 13. THANK YOU!!!
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