R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment

Abstract

Highlighting the latest developments in both real-world evidence as it relates to health technology assessment and acceptance of real-world evidence by health technology assessment agencies.

Role of RWD / RWE in health technology assessment

“…Однако отказ от использования рандомизации имеет ограничения, наиболее важным из которых является повышенный риск смещения и других систематических ошибок. Количественные методы анализа погрешностей помогают изучить влияние учтённых отклонений на оценку лечения и экономической эффективности, но более важными представляются подходы, которые помогают ограничить эти отклонения [16].…”

“…В руководстве ISPOR представлены методы идентификации вмешивающихся факторов (конфаундеров). Руководящие принципы ОТЗ по RWD рекомендуют выявлять вмешивающиеся факторы с помощью систематического поиска прогностических исследований и / или использования мнений экспертов [7,16]. Однако недостаток в знаниях в отношении менее изученных нозологий, небольшие размеры выборки могут ограничивать возможность учёта всех потенциальных вмешивающихся факторов, даже если данные о них доступны.…”

R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 15

“…Oncology is an area of significant innovation, with a total of 115 novel active substances having been launched globally in the past 5 years and 237 over the last 20 years [ 1 ]. This explosion in novel therapeutics leads to substantial use and need for real-world data (RWD), including RWD to understand patient characteristics, disease burden, treatment and outcomes, all of which can be useful for regulatory and health technology assessment (HTA) submissions [ 2–5 ]. When generating this real-world evidence (RWE), manufacturers not only are considering regulatory and HTA stakeholders, but also healthcare professionals (who may also be involved in RWD collection), so it is important for them to bear in mind the needs of all potential users of the RWE generated.…”

R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 12

“…In order for studies using real-world data (RWD) to be more widely accepted for HTA decision making, treatment effectiveness estimates obtained from appropriately designed studies using RWD should be consistent with those from RCTs. This is the premise on which the RCT-DUPLICATE initiative was born [ 2 ], and the final results of this landmark study were just published [ 3 ]. By mirroring the design of 30 completed and two ongoing RCTs with analyses from three US healthcare claims data sources, the initiative aimed to ascertain the level of agreement between RCT results and a replicated version of the trial using RWD.…”

“…This is in line with a general critical view of RWD by IQWIG [ 10 , 11 ], and while we are yet to have a full read out of all demonstration projects definitively stating in which indications and from which data sources RWD can appropriately emulate RCTs, it is a shame that IQWIG do not want to be pragmatic in cases where conventional RCTs are stifled by patient preferences and absence of equipoise. Given the dismissiveness of RWE it will be interesting to see how the German authorities view the results of post approval RWD studies for spinal muscular atrophy they have mandated [ 2 ].…”