1312O - From regulating off-label use to creating an environment for drug repurposing in oncology

Background

Use of anticancer drugs for unapproved indications poses significant challenges to the health care system; Off-label use often lacks robust scientific evidence and due to high treatment costs reimbursement restrictions may apply.

Methods

The assembled Dutch Health Insurance Companies (HIC) and the Dutch Society for Medical Oncology (NVMO) have jointly implemented measures to regulate and facilitate off-label access.

Results

The NVMO has created a desk to assess and implement all valuable free-of-charge and expanded-access programmes. Besides, a dedicated off-label assessment committee has been appointed to work closely with the Drug Evaluation Committee of HIC in appraising the evidence of specific off-label use of anticancer drugs and arranging reimbursement. The main focus of these committees are off-patent drugs (or drugs close to patent expiry) that have no market exclusivity and for which the marketing authorisation holder(s) will probably not request an extension of indication. Furthermore, the HIC have embedded the Drug Rediscovery Protocol (DRUP), a national adaptive, multi-drug, pan-cancer trial within their framework to stimulate evidence generation. By introducing the ‘Personalised reimbursement’ scheme, the HIC have made it possible to reimburse drugs for patients that are clearly benefitting from therapies within the DRUP trial.

Conclusions

While regulation of off-label use may seem restrictive, we believe that by establishing a joint framework for evaluating data and reimbursement decisions we can stimulate evidence generation and rational use of costly drugs. By providing clinical recommendations that are based on transparent and standardised assessments we can also reduce practice variation and improve the quality of healthcare. EMA has recently launched an initiative to support hospitals and non-profit organisations in generating evidence on off-label use and to authorise new indications. Ideally, a European framework should be established in which regulatory approval as well as HTA assessment are realised centrally, along with clear and rapid pathways to reimbursement decisions in each member state. In this way valuable anticancer drugs can be accessible to all within a more reasonable timeframe.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

The authors.

Funding

Has not received any funding.

Disclosure

E.E. Voest: Financial Interests, Personal, Advisory Board, Hourly rate, to charity: Biogeneration Ventures; Financial Interests, Institutional, Advisory Board, Hourly rate, no compensation in 2019-2020: InteRNA; Financial Interests, Institutional, Invited Speaker, DRUP trial: Amgen, AstraZeneca, Boehringer Ingelheim, BMS, Clovis Oncology, Eisai, Ipsen, MSD, Novartis, Pfizer, GSK, Seattle Genetics; Financial Interests, Institutional, Invited Speaker, DRUP trialDRUG Access Protocol: Bayer, Roche; Financial Interests, Institutional, Invited Speaker, DRUG Access Protocol: Sanofi; Non-Financial Interests, Supervisory Board: HMF – Hartwig Medical Foundation; Non-Financial Interests, Principal Investigator, Senior group leader: Oncode Institute; Non-Financial Interests, Advisory Role, Editorial Board: JAMA Oncology; Non-Financial Interests, Leadership Role, Board of Directors: Cancer Core Europe. All other authors have declared no conflicts of interest.