Which data and when?

Transkript

1 PRO-data for market access in Germany where and when? Frank-Ulrich Fricke PRO-data for market access in Germany where and when? AMNOG the German assessment Which data and when? Requirements to be met Seite 2 Seite 1

2 AMNOG The German assessment approach Gemeinsamer Bundesausschuss PKV Nutzenbewertung (Beratung) Übernahme Rabatt- Preis Geltungserstreckung nach 78 (3a) AMG Bundesinstitut für Arzneimittel und Medizinprodukte erlaubt Zulassung Quelle: nach BMG Seite 3 Elements of the German assessment dossier 1. Authorised therapeutic indication, 2. Medical benefit, 3. Medical additional benefit in relation to the appropriate comparator, 4. Number of patients and patient groups, for whom there is a therapeutically important additional benefit, 5. Costs of the therapy for the statutory health insurance fund, 6. Requirement for a quality-assured therapeutic indication. Source: G-BA Seite 4 Seite 2

3 Accepted patient-relevant dimensions Seite 5 Patient-Reported Outcomes Any outcome evaluated directly by the patient himself and based on patient s perception of a disease and its treatment(s) is called patientreported outcome (PRO). The term PRO is proposed as an umbrella term to cover both single dimension and multi-dimension measures of symptoms, health-related quality of life (HRQL), health status, adherence to treatment, satisfaction with treatment, etc. Source: EMEA, Reflection Paper on the Regulatory Guidance for the Use of Health-related Quality of Life (HRQL) Measures in the Evaluation of Medicinal Products, 2005, p. 3 Seite 6 Seite 3

4 What is the difference? HRQL Patient- Reported Outcomes Compliance/ Adherence Treatment Satisfaction/ Convenience Seite 7 PRO-data for market access in Germany where and when? AMNOG the German assessment Which data and when? Requirements to be met Seite 8 Seite 4

5 AMNOG The German assessment approach Gemeinsamer Bundesausschuss PKV Nutzenbewertung (Beratung) Übernahme Rabatt- Preis Geltungserstreckung nach 78 (3a) AMG Bundesinstitut für Arzneimittel und Medizinprodukte erlaubt Zulassung Quelle: nach BMG Seite 9 Reimbursement price negotiations are mandatory Without Additional Benefit Reimbursement price is subject to 130b (3) SGB V (annual treatment cost of the appropriate comparator) Appropriate comparator can be more than one drug or placebo With Additional Benefit Additional benefit as defined by the G-BA annual treatment cost of the appropriate comparator Prices of comparable drugs European reference prices from a number of countries Seite 10 Seite 5

6 The Role of the dossier in the negotations The dossier is part of the reimbursement price negotations What is in the dossier is on the table for negotations What is not in the dossier needs to be agreed upon separately For strategic reasons relying on agreement might not be the best option Seite 11 PRO-data for market access in Germany where and when? AMNOG the German assessment Which data and when? Requirements to be met Seite 16 Seite 6

7 PRO is great in a proper RCT double-blind Source: IQWiG, Allgemeine Methoden, Version 4.2 vom , p. 69 Seite 17 Validated measures and scales is important Source: G-BA, _AM-RL-XII_ D-157_Fingolimod-Abl-Befr_TrG, p. 11 Source: G-BA, _AM-RL-XII_Elosulfase alfa_ d-114_trg, p. 5 Seite 18 Seite 7

8 Without an MCID PRO is difficult Source: G-BA, _AM-RL-XII_Ramucirumab_ D-150_TrG, p. 6 Source: G-BA, _AM-RL-XII_Elosulfase alfa_ d-114_trg, p. 5 Seite 19 Drop-out rates impact on the acceptance of data Source: G-BA, _AM-RL-XII_Ramucirumab_ D-150_TrG, p. 6 Source: G-BA, _AM-RL-XII_ D-157_Fingolimod-Abl-Befr_TrG, p. 11 Seite 20 Seite 8

9 The EQ-5D is not considered to reflect quality of life Source: G-BA, _AM-RL-XII_Ramucirumab_ D-150_TrG, p. 6 Source: G-BA, _AM-RL-XII_ D-157_Fingolimod-Abl-Befr_TrG, p. 11 Seite 21 Coherence PRO/ CRO Source: G-BA, Tragende Gründe zum Beschluss des Gemeinsamen Bundesausschusses über eine Änderung der Arzneimittel-Richtlinie (AM-RL): Anlage XII - Beschlüsse über die Nutzenbewertung von Arzneimitteln mit neuen Wirkstoffen nach 35a SGB V Siltuximab, p. 8 Seite 22 Seite 9

10 Thank you very much for your attention! Seite 23 Seite 10