CONFIRM, COMPLEMENT, CONTEXTUALIZE, THE 3CS OF RWE: HOW REAL-WORLD-EVIDENCE CAN SUPPORT ACCESS DECISIONS FOR NEW MEDICINES
Author(s)
Stacey Hickson, PhD1, Kate Burslem, BSc, MSc2, Rachel M. Black, PharmD3, Neha Pethad, Pharm D4, Susan Hogue, MPH, RPh, PharmD5, W. Jackie Kwong, PharmD, PhD6, Christoph Glaetzer, Dipl. Kfm.7.
1Senior Director, Global Access Policy, Johnson & Johnson, Raritan, NJ, USA, 2AESARA, Exeter, United Kingdom, 3AESARA, Austin, TX, USA, 4AESARA, Sheffield, United Kingdom, 5AESARA, Chapel Hill, NC, USA, 6Johnson & Johnson, Hillsborough, NJ, USA, 7Janssen (J&J), Princeton, NJ, USA.
1Senior Director, Global Access Policy, Johnson & Johnson, Raritan, NJ, USA, 2AESARA, Exeter, United Kingdom, 3AESARA, Austin, TX, USA, 4AESARA, Sheffield, United Kingdom, 5AESARA, Chapel Hill, NC, USA, 6Johnson & Johnson, Hillsborough, NJ, USA, 7Janssen (J&J), Princeton, NJ, USA.
OBJECTIVES: Characterize use of real-world evidence (RWE) in HTA for new medicines, furthering the 3Cs concept introduced at ISPOR ASIA 2025.
METHODS: RWE was systematically extracted from publicly available HTA documentation in Australia, Canada, England, France, Germany, Italy, and Scotland, for EMA-approved oncology products from 2023-2024. RWE was classified into the previously defined 3Cs categories (Contextualization of global clinical trials into local relevance, Complementary effectiveness evidence to clinical trial results (for example, indirect treatment comparisons), and Confirmation of clinical trial findings). The relationship between the role of RWE and reimbursement outcome was examined.
RESULTS: 12 products with assessments by at least 5 HTA agencies were identified; 74 assessments and decisions across HTAs were reviewed. RWE was included in 69 (93%) assessments; all included RWE for contextualization. Complementary and confirmatory RWE were noted in 22% (n=16) and 9% (n=7), respectively. 66 (93%) that included contextualizing RWE had a positive reimbursement decision. A higher rate of positive reimbursement decisions was observed with inclusion of confirmatory RWE versus complementary RWE (100% vs. 87%). Reimbursement decisions (reimburse, conditional, restricted, conditional & restricted, and do not reimburse, respectively) where RWE was considered by category were: Contextualize only: 48%(22/46), 33%(15/46), 4%(2/46), 9%(4/46), 7%(3/46); Contextualize + Complement: 19%(3/16), 31%(5/16), 13%(2/16), 25%(4/16), 13%(2/16); Contextualize + Confirm: 28.6%(2/7), 28.6%(2/7), 42.9%(3/7), n/a, n/a
CONCLUSIONS: RWE categorized according to the 3Cs was identified across almost all HTA assessments, regardless of product or HTA body, reflecting the utility of this classification and the global recognition of their importance in value assessment. While there remains opportunity to better leverage RWE to complement trial data, expanding the timeline and number of assets in the research may show greater use and impact of RWE, especially for confirm data and in disease areas with a longer outcome period and distinguished disease characteristics.
METHODS: RWE was systematically extracted from publicly available HTA documentation in Australia, Canada, England, France, Germany, Italy, and Scotland, for EMA-approved oncology products from 2023-2024. RWE was classified into the previously defined 3Cs categories (Contextualization of global clinical trials into local relevance, Complementary effectiveness evidence to clinical trial results (for example, indirect treatment comparisons), and Confirmation of clinical trial findings). The relationship between the role of RWE and reimbursement outcome was examined.
RESULTS: 12 products with assessments by at least 5 HTA agencies were identified; 74 assessments and decisions across HTAs were reviewed. RWE was included in 69 (93%) assessments; all included RWE for contextualization. Complementary and confirmatory RWE were noted in 22% (n=16) and 9% (n=7), respectively. 66 (93%) that included contextualizing RWE had a positive reimbursement decision. A higher rate of positive reimbursement decisions was observed with inclusion of confirmatory RWE versus complementary RWE (100% vs. 87%). Reimbursement decisions (reimburse, conditional, restricted, conditional & restricted, and do not reimburse, respectively) where RWE was considered by category were: Contextualize only: 48%(22/46), 33%(15/46), 4%(2/46), 9%(4/46), 7%(3/46); Contextualize + Complement: 19%(3/16), 31%(5/16), 13%(2/16), 25%(4/16), 13%(2/16); Contextualize + Confirm: 28.6%(2/7), 28.6%(2/7), 42.9%(3/7), n/a, n/a
CONCLUSIONS: RWE categorized according to the 3Cs was identified across almost all HTA assessments, regardless of product or HTA body, reflecting the utility of this classification and the global recognition of their importance in value assessment. While there remains opportunity to better leverage RWE to complement trial data, expanding the timeline and number of assets in the research may show greater use and impact of RWE, especially for confirm data and in disease areas with a longer outcome period and distinguished disease characteristics.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
RWD100
Topic
Clinical Outcomes, Health Technology Assessment, Real World Data & Information Systems
Disease
Oncology, Rare & Orphan Diseases