RE-DEFINING LONG-TERM SURVIVAL- SURVIVAL ENDPOINTS WITHIN HEMATOLOGICAL CANCERS – A TREND ANALYSIS OF THE PIPELINE PRODUCTS
Author(s)
Manansala K1, Jain A2, Bathija S1
1PAREXEL International, Horsham, PA, USA, 2PAREXEL International, Chandigarh, India
OBJECTIVES: The long-term survival (LTS) benefits of candidate therapies is increasingly being assessed in clinical trials. As this data is generally not available for pivotal studies at time of pricing and reimbursement review it does not influence initial access decisions. We conducted a trend analysis of landmark survival endpoints, particularly LTS, in hematological cancer indications to inform considerations for future clinical trial designs and the potential impact on P&R reevaluations. METHODS: A targeted review using Citeline’s Trialtrove database was conducted to extract landmark survival data (≥3months) for progression free survival (PFS) and overall survival (OS) for products across leukemia, lymphoma, multiple myeloma (MM), and myelodysplastic syndromes (MDS) between June 2014 and June 2016. RESULTS: A total of 961 observations were extracted across phase I/II (n=265), phase II (n=552), phase II/III (n=20) and phase III (n=124) across MDS (n=105), MM (n=162), leukemia (n=346), and lymphoma (n=348). Overall, 53% (516/961) of trials included landmark OS and PFS across indications, 64% (329/516) included long-term endpoints of ≥1 year PFS and 83.5% (431/516) ≥1 year OS across immune oncologic (IO) (n=135) and non-IO (n=370) trials. The most commonly seen long-term endpoints across 516 trials were PFS at years 1 (10%,n=51), 2 (23%,n=117 ), 3 (11%,n=59) and 5 (10%,n=52), and OS at years 1 (17%,n=86), 2 (22%,n=115 ), 3 (17%,n=86) and 5 (11%,n=57), and the proportion did not differ significantly between IOs and non-IOs. Interestingly, 5% (n=27) of trials measured PFS between years 6 and 15, 8% (n=43) measured OS between years 6 and 16, and about 2% (n=11) trials measured PFS and/or OS until progression or death. CONCLUSIONS: LTS endpoints are becoming an increasingly frequent inclusion in hematologic cancer therapy trials. Future monitoring and primary research would be required to understand if newer drugs will be re-evaluated in light of long-term data becoming available.
Conference/Value in Health Info
2017-05, ISPOR 2017, Boston, MA, USA
Value in Health, Vol. 20, No. 5 (May 2017)
Code
PCN51
Topic
Clinical Outcomes
Topic Subcategory
Relating Intermediate to Long-term Outcomes
Disease
Oncology, Systemic Disorders/Conditions