A LOSS OF CHANCE INDEX- A NEW TOOL FOR OPTIMIZING PATIENT ACCESS TO INNOVATIVE DRUGS

Author(s)

Gavini F1, Sarkozy F2, Jouan-Flahault C3, Fumoleau P4
1ROCHE SAS - FRANCE, Boulogne Billancourt, France, 2FSNB Health & Care, Paris, France, 3LEEM, Paris, France, 4Centre Georges François Leclerc, Dijon, France

OBJECTIVES: Our objectives were to report time-lags between European Marketing Authorisation (EUMA) and French Pricing and Reimbursement Decision (FPRD) for recent innovative anti-cancer drugs and to quantify the corresponding patient loss of chance METHODS: We included 2012-2013 EMA-approved anti-cancer drugs in solid tumours and calculated the time-lag between EUMA and FPRD.  Official pivotal trials’ data were retrieved from the Haute Autorité de Santé (HAS) website and the Number Needed to Treat (NNT) to prevent one event (progression, death) was estimated. An Absolute Loss of Chance (ALOC) index was calculated over the time-lag period and per time unit combining NNT and HAS target population size. Drugs with level 5 Added Medical Value (ASMR, Amélioration du Service Médical Rendu) were excluded. RESULTS: Twelve drugs were granted a EUMA for solid tumours in 2012 and 2013 with level 2-4 Added Medical Value: one drug of level 2-important (trastuzumab emtansine), five drugs of level 3-moderate (cabazitaxel, vemurafenib, critzotinib, pertuzumab, and enzalutamide), and six drugs of level 4-minor (eribuline, ipilimumab, abiraterone, vandetanib, axitinib, and vismodegib). The time-lags between EUMA and FPRD ranged from 7.4 months (enzatulamide) to 29.9 months (cabazitaxel). Concurrently, the overall ALOC ranged from 9 to 799 medical events which might have not been prevented unless alternative access (Compassionate, Clinical Trials), depending on drug, indication and time-lag.  CONCLUSIONS: Early patient access for innovative drugs in oncology might help preventing a substantial number of progressions and deaths. ALOC might support patient access decision making process.

Conference/Value in Health Info

2015-11, ISPOR Europe 2015, Milan, Italy

Value in Health, Vol. 18, No. 7 (November 2015)

Code

PCN259

Topic

Health Policy & Regulatory, Health Service Delivery & Process of Care, Health Technology Assessment, Organizational Practices, Real World Data & Information Systems

Topic Subcategory

Academic & Educational, Coverage with Evidence Development & Adaptive Pathways, Decision & Deliberative Processes, Health & Insurance Records Systems, Health Care Research, Health Disparities & Equity, Reimbursement & Access Policy

Disease

Oncology

Your browser is out-of-date

ISPOR recommends that you update your browser for more security, speed and the best experience on ispor.org. Update my browser now

×