Economy of rare surgical technologies Vladimir Zorenko , MD, PhD, Professor Hematological Scientific Centre, Russian Aca

1. Economy of rare surgical technologies Vladimir Zorenko, MD, PhD, Professor Hematological Scientific Centre, Russian Academy of Medical Sciences Moscow, Russia

2. Causes of slow development of rare (orphan) surgical technologies in Russia • Lack of patient registers • Lack of diagnostic in regions • Lack of specialized centres • No governmental program of financing • Lack of standards and protocols of care

3. Examples of rare surgical technologies • Treatment of hyperlipidemia • Transplantation • Orthopedic treatment of patients, suffered from hemophilia with antibodies to antihemophilic factor

4. Treatment of hyperlipidemia • Should be about 100 000 patients in Russia • Discovered and received treatment 120 patients • All patients treated in Moscow and Saint-Petersburg • Treatment – LDL aphaeresis 1 time in a week • There is no more than 6000 procedures per year in Russia • One procedure of aphaeresis costs about 1000 USD

5. Transplantation in Russia • No common register of needy patients • Problems with tissue banks and tissue typing • No clear and valid information about patients after transplantation • No information about success and effectiveness of transplantation • No planning of number of transplantation and total expenditures • Weak legislation

6. Why treatment of hemophilia is successful? Valid register of patients Standard and protocol of medical care Governmental program of financing Appropriate supply with drugs Several centres with trained specialists Clarity of accounts

7. Register of patients with hereditary coagulopathy There is a data on 7374 patients (1761 under 18 years old) Number of patients with antibodies to antihemophilic factor- 140

8. Titre of antibodies to antihemophilic factor (% of patients) Titre below 10 ВЕ Titre above 10 ВЕ 18% 82%

9. Features of clinical course in patients with antibodies to antihemophilic factor Affection of joints: 2 joints – 35 % 3 -5 joints - 43% More than 5 joints – 22% Inpatients 6-15 years old there is15times more often affection of bones by the type of pseudotumor 13% of patients hospitalized with purulent and septic complications

10. Regimes of hemostatic treatment during surgical treatment of patients with antibodies to antihemophilic factor • High-dose therapy with antihemophilic factor VIII • Drugs consists of activated prothrombin complex • Recombinant acivated antihemophilic factor VII

11. Types of surgical interventions in patients withantibodies to antihemophilic factor *82% of patients had high titre of antibodies

12. Endoprosthesis replacement of knee-joint and hip joint 95% - good clinical results

13. There were 151 procedures of endoprosthesis replacementof knee-joint and 32 procedures endoprosthesis replacement of hip joint made since 1992 (includes 91 procedures of endoprosthesis replacement of knee-joint and 17 procedures endoprosthesis replacement of hip joint since 2005) 6 procedures of endoprosthesis replacement of knee-joint were made in a patients with antibodies to antihemophilic factor All surgical intervention in patients with antibodies to antihemophilic factor were made in Hematological Scientific Centre of Russian Academy of Medical Sciences

14. Therapy, recieving during surgical intervention and after that • Eptacog alfa (activated)120 mcg/kgevery 2 hours, than interval extension to 3 and 4 hours OR • Drugs consists of activated prothrombin complex (FEIBA) 100 units/kgevery 12 hours

15. How many units of drugs needed for surgical intervention in patients with antibodies to antihemophilic factor? How much does it cost? • It is necessary about 250 bottles ofNovoSeven with dose of 2,4 mg… • It cost about ≈ 650 000 USD

16. Thank you for attention!