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Daniel Dhumeaux, Henri Mondor hospital Créteil, France

HCV compassionate use programme The French experience. Daniel Dhumeaux, Henri Mondor hospital Créteil, France. Amsterdam, April 27,2013. daniel.dhumeaux@gmail.com. CUPIC. CUPIC. (Compassionate Use of Protease Inhibitors in viral C Cirrhosis).

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Daniel Dhumeaux, Henri Mondor hospital Créteil, France

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  1. HCV compassionate use programme The French experience Daniel Dhumeaux, Henri Mondor hospital Créteil, France Amsterdam, April 27,2013 daniel.dhumeaux@gmail.com

  2. CUPIC

  3. CUPIC (Compassionate Use of Protease Inhibitors in viral C Cirrhosis) - National multicenter observatory in the setting of ATU * (promoter ANRS) - Evaluation of real-life safety and efficacy of triple therapy (including telaprevir or boceprevir associated with peginterferon plus ribavirin) in HCV genotype 1 patients with compensated cirrhosis who were non responders to previous dual therapy *Autorisation temporaire d’utilisation (temporary authorisation for use) Early access programme

  4. Treatment regimen TVR + Peg-IFN α-2a + RBV Peg-IFN α-2a + RBV Follow-up Peg-IFN + RBV BOC + Peg-IFN α-2b + RBV Follow-up n=205 BOC: 800 mg/8h; Peg-IFNα-2b: 1.5 µg/kg/week; RBV: 800 to 1400 mg/day n=292 TVR: 750 mg/8h; Peg-IFNα-2a: 180 µg/week; RBV: 1000 to 1200 mg/day 72 36 4 0 12 16 48 8 Weeks http://www.afssaps.fr/var/afssaps_site/storage/original/application/4b8c53711bab9d8f7d4c3f947caa90f6.pdf http://www.afssaps.fr/var/afssaps_site/storage/original/application/fa78af08e029caf9d82bcd9d3e77eb09.pdf

  5. Safety (SVR12) Telaprevir Boceprevir Serious adverse events (SAE)* 54.2% 51.0% Premature discontinuation of treatment due to SAE 14.2% 21.3% (*) % of patients with at least one event

  6. Risk of occurrence of death or severe complications Platelets ≤100,000/µL Platelets >100,000/µL 4.3% Albumin ≥35g/L 3.3% 7.1% Albumin<35g/L 44.1%

  7. Risk of occurrence of death or severe complications Platelets >100,000/µL Platelets ≤100,000/µL 4.3% Albumin ≥35g/L 3.3% 7.1% Albumin<35g/L 44.1%

  8. Efficacy (SVR12) SVR (%) All patients - 100 Relapsers Partial responders 53% 51% - 50 40% 41% 40% 32% 0 - Telaprevir Boceprevir Fontaine et al. J Hepatol 2013;58(suppl.1):S27

  9. The French ATU system - Implemented in 1994 - More than 1,000 medicinal products assessed since 1994 - Availability 10 to 12 months before market authorisation application - Therapeutic areas . Oncology-haematology . Central nervous system diseases . Metabolic disorders . Infectious diseases including HIV infection and and viral hepatitis

  10. Criteria for granting ATU 1. The product is a medicinal product (not a preparation) 2. ATU is given for treatment (not for investigation) 3. There is no market authorisation application 4. The patient cannot be included in a clinical trail 5. The disease is serious and/or rare 6. There is no available alternative therapeutic method 7. Efficacy and safety are presumed and benefit is expected for the patient

  11. Two types of ATU : nominative and cohort ATU Nominative ATU Cohort ATU . For one patient, on a name patient basis . On the request and responsibility of the clinician . ATU for the duration of treatment . Usually follow-up of patients and data collection according to a protocol for therapeutic use . For a group of patients . Applied by the company commitment to submit a marketing authorisation . ATU for one-year duration, renewal possible . Always follow-up of patients and data collection according to a protocol of therapeutic use

  12. The ATU system Success and limitations - The ATU system is extremely useful for covering public health needs : . it is strongly supported by patients ans physicians . it is contolled by competent authorities (*) - The risks are (a) to slow down clinical trials and marketing authorisation applications, (b) to overestimate efficacy and to underestimate safety (*) National agency for medicinal product safety

  13. Short and middle-term perspectives for hepatitis C early access programme in France - A nominative ATU was recently implemented for patients having developed severe liver disease recurrence post-transplantation (sofosbuvir plus ribavirin) - A cohort ATU is in progress for (a) patients waiting liver transplantation, and (b) patients having developed severe liver disease recurrence post-transplantation (sofosbuvir plus ribavirin)

  14. Summary • Use of first generation PI in real life is a major step forward in HCV treatment : • increases SVR in all genotype 1 patients including cirrhotics • is associated with more frequent SAEs, including death, severe infections and hepatic decompensation and difficult management of anemia in cirrhotics • Patients with albumin <35 g/L and platelets <100,000 /mm3 should not be treated • Other patients need closer monitoring • TVR can be administrated twice daily • Triple therapy (TVR) for 12 weeks in CC, F0-F3 patients with RVR ?

  15. The compassionate use of medicinal products. An example: the French ATU system

  16. The ATU system Success and limits - The ATU system is extremely useful for covering public health needs : . It is strongly supported by patients ans physicians . It is contolled by competent authorities - The risks are (a) to slow down clinical trials and marketing autorisation applications, (b) to overestimate efficacy and to underestimate safety - Regarding nominative ATU : . Too many . Complex system . No strong regulatory long term status (no mandatory marketing authorisation application)

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