HOW TO MAXIMISE THE VALUE AND PAYERS’ ACCEPTANCE OF SINGLE ARM TRIAL DATA IN ONCOLOGY?

Author(s)

Hoyle C¹, Wright J², Ede J³, van Engen A⁴, Ryan J¹
¹AstraZeneca, Cambridge, UK, ²Quintiles, Reading, UK, ³AstraZeneca, Cambridgeshire, UK, ⁴Quintiles Advisory Services, Hoofddorp, The Netherlands

OBJECTIVES: Single arm trials (SATs) are used to accelerate patient access to innovative medicines or when large randomised control trials (RCTs) are not feasible or ethical. This is particularly true for diseases with small populations or diseases with no reasonable comparator treatment, such as orphan diseases and diseases with severely limited treatment options in oncology. Marketing authorisation based on SAT data is a key strategy for accelerating patient access to innovative medicines in oncology. The objective of this research was to understand key payer concerns of the value of SAT data and to recommend strategies which may maximise future value of oncology products with evidence from SATs. METHODS: An in-depth review was performed on assessments for six products (ofatumumab, vismodegib, ponatinib, ibrutinib, idelalisib and ceritinib) by nine agencies (NICE, SMC, AWMSG, HAS, G-BA, pCODR, PBAC, ZIN and TLV) from January 2011 to December 2015. RESULTS:

Conference/Value in Health Info

2016-10, ISPOR Europe 2016, Vienna, Austria

Value in Health, Vol. 19, No. 7 (November 2016)

Code

PRM207

Topic

Study Approaches

Disease

Oncology

Presentation