PROS In Oncology HTA Decisions, Do They Matter?

OBJECTIVES: Healthcare decision-making is becoming more patient-centric, but it is unclear to what extent European payers are using patient reported outcomes (PRO) and Quality of Life (QoL) data in their assessment of the value of new oncology drugs. The objective was to investigate how inclusion of PRO/QoL evidence in Health Technology Assessments (HTA) has evolved over the past five years and to what extent PRO/QoL data influenced the recommendation by HTA agencies. METHODS: HTAs for new oncology products by four agencies (G-BA, HAS, NICE and SMC) from 2011-2016 were identified. Original assessments, resubmissions and extensions of indications were included. HTAs were reviewed for inclusion of PRO data. PRO instruments were classified as collected with a general measure, disease-specific measure or utility measure. Both numerical counts of HTA submissions and qualitative evaluation of agency statements were used to understand the attitude of agencies towards PRO data. RESULTS: A total of 376 HTA reports for 133 drug-indication combinations were identified. 72% of the drug indication combinations included PRO data in one or more submissions. Of the instruments used to capture PRO data, 36% were disease-specific, 38% were generic, 17% were utility-oriented and 9% were unspecified. Submission of PRO data increased over the years, in 2016 50 - 90% submissions per country included PRO data. Demonstrating improvement in QoL led to higher benefit ratings by G-BA and HAS and supported clinical benefit assigned by SMC and NICE. QoL was considered in the recommendation of 89 HTAs (35% of HTA submissions including PRO). In a number of cases, strong PRO data led to a positive recommendation despite lack of overall survival data. CONCLUSIONS: HTA agencies value the submission of PRO data, however submission is not yet standard practice. Although lack of PRO data does not negatively impact decision-making, PRO data inclusion can have a positive influence on recommendations.