application to all types of observational studies conductible in the health and epidemiological field . The paper made several recommendations for the use of RWE , but these have yet to be introduced into legislation .
Spain High-cost therapies have led to the introduction of a RWD platform ( VALTERMED ) to assess their therapeutic value in real practice in Spain . 13 The software platform facilitates registries of patient ’ s outcomes across the Spanish regions with the objective to provide patient access to high-cost , innovative medicines under a framework that allows decision-makers to assess efficiency . VALTERMED shares RWD among clinicians and decision-makers , and prioritises drugs reimbursed under a special situation , or for which there is a need to reduce uncertainty . 13 It therefore supports the implementation of innovative payment-by-results schemes , as well as maximising available healthcare resources within a system of universal healthcare . It has already been used for several products that gained reimbursement via payment-by-results models which include Orkambi ( cystic fibrosis ), Symkevi ( cystic fibrosis ), Kalydeco ( cystic fibrosis ),
14 , 15
Kymriah and Yescarta .
United Kingdom The UK HTA body NICE ( National Institute for Health and Care Excellence ) has identified RWE as one of the nine research areas on which to develop guidance . Also , in 2017 , NICE took part in the GetReal Initiative as part of the Innovative Medicines Initiative ( IMI ) project to develop tangible solutions to key challenges associated with using real-world data in drug development and subsequent regulatory and health technology assessment . 16 However , NICE continues to rely heavily on RCTs in drug evaluation as key comparative effectiveness data are provided by them . 15 RWE can be a useful addition to the decision-making process when the evidence base is limited . This has been observed in several managed entry agreements , including both Kymriah and Yescarta where NICE stated that data from the Systemic Anti-Cancer Therapy ( SACT ) dataset would be used in follow-up appraisals . 17 , 18 Increasingly , RWE is also being used by NICE when requirements for comparative and randomised data are being relaxed in order to speed up drug approval . 19
Overall , it can be observed that regulatory and HTA bodies are adapting to the rising need for RWE in their assessments . However , a conservative approach is taken and traditional RCTs remain the key study type on which access decisions for medicines are made . From a medicines access standpoint , RWE looks as though it can act as good supplementary evidence to RCTs to help inform better decision making .
Beyond regulatory and HTA authorities , many stakeholders benefit from RWE . It is increasingly recognised as an additional source of reliable , scientifically valid data that can inform scientific societies , governments and public health authorities , health care providers , patients and the industry about the real-life outcomes of healthcare interventions . RWE can improve healthcare in a broad diversity of ways , from simply improving the knowledge on treatments , to identifying organisational issues in healthcare systems that may have big budgetary and health impacts . RWE will undoubtedly progress both in terms of methodology and scientific validity , and in terms of its routine incorporation in decision-making .
References 1 The London School of Economics and Political Science . RWE in Europe Paper V . Policy challenges around Real World Evidence adoption in Europe [ Internet ]. London ; 2018 . www . lse . ac . uk / business-andconsultancy / consulting / assets / documents / rwe-in-europepaper-v . pdf ( accessed March 2021 ). 2 IQVIA . RWE : From “ Nice to Have ” to “ Must Have ” [ Internet ]. London ; 2018 . www . iqvia . com / - / media / iqvia / pdfs / library / whitepapers / rwe-from-nice-to-haveto-must-have . pdf ( accessed March 2021 ). 3 ICER . Real World Evidence for Coverage Decisions : Opportunities and Challenges [ Internet ]. 2018 . https :// icer-review . org / wp-content / uploads / 2018 / 03 / ICER-Real- World-Evidence-White- Paper-03282018 . pdf ( accessed March 2021 ). 4 PharmaBoardroom . Real World Evidence : From Market Access to Drug Approval ? [ Internet ]. 2019 . https :// pharmaboardroom . com / articles / real-world-evidencefrom-market-access-to-drugapproval / ( accessed March 2021 ). 5 Accelerated assessment - European Medicines Agency [ Internet ]. European Medicines Agency . [ cited 20 October 2020 ]. www . ema . europa . eu / en / human-regulatory / marketingauthorisation / acceleratedassessment ( accessed March 2021 ). 6 US Food & Drug Administration . Framework for FDA ’ s Real-World Evidence Program . Published 2018 . [ cited 20 October 2020 ] www . fda . gov / science-research / science-and-research-specialtopics / real-world-evidence ( accessed March 2021 ). 7 Accelerated assessment – European Medicines Agency [ Internet ]. European Medicines Agency [ cited 20 October 2020 ]. www . ema . europa . eu / en / human-regulatory / marketingauthorisation / acceleratedassessment ( accessed March 2021 ). 8 Conditional marketing authorisation – European Medicines Agency [ Internet ]. European Medicines Agency [ cited 20 October 2020 ]. www . ema . europa . eu / en / human-regulatory / marketingauthorisation / conditionalmarketing-authorisation ( accessed March 2021 ). 9 European Medicines Agency . Final report on the adaptive pathways pilot [ Internet ]. 2016 . www . ema . europa . eu / en / documents / report / final-reportadaptive-pathways-pilot _ en . pdf ( accessed January 2021 ). 10 Haute Autorité de Santé . Plan d ’ action pour l ’ évaluation des médicaments innovants [ Internet ]. 2020 . www . hassante . fr / upload / docs / application / pdf / 202001 / plan _ daction _ pour _ les _ medicaments _ innovants _ 27.01.2020 . pdf ( accessed January 2021 ). 11 Concepts for the generation of routine practice data and their analysis for the benefit assessment of drugs according to § 35a Social Code Book V ( SGB V ). IQWiG Reports – Commission No . A19-43 . 12 Documento Programmatico sulla Ricerca Osservazionale [ Internet ]. 2019 . https :// sifwebsite . s3 . amazonaws . com / uploads / document / attachment / 70 / Documento _ Programmatico _ Studi _ Osservazionali _ 1.3 _ 20190903 . pdf ( accessed March 2021 ). 13 Ministerio De Sanidad , Consumo y Bienestar Social . Preguntas y respuestas frecuentes sobre el sistema de información para determinar el valor terapéutico en la práctica clínica real de los medicamentos de alto impacto sanitario y económico en el sistema nacional de salud ( VALTERMED ) [ Internet ]. 2019 . www . mscbs . gob . es / profesionales / farmacia / valtermed / docs / VALTERMED _ Preguntas _ y _ Respuestas . pdf ( accessed March 2021 ). 14 Ministerio De Sanidad , Consumo y Bienestar Social . Protocolo farmacoclínico del uso de lumacaftor / ivacaftor 100 / 125 ( Orkambi 100 / 125 ® ) y de tezacaftor 100 mg e ivacaftor 150 mg ( Symkevi ® ) + ivacaftor 150 mg ( Kalydeco ® ) en el tratamiento de la fibrosis quística en el sistema nacional de salud [ Internet ]. 2019 . www . mscbs . gob . es / profesionales / farmacia / valtermed / docs / 20191220 _ Protocolo _ farmacoclinico _ fibrosis _ quistica . pdf ( accessed March 2021 ). 15 HIS Markit . How to democratize access and prevent the next supermodel medicine [ Internet ]. 2019 . https :// ihsmarkit . com / research-analysis / how-todemocratize-access-preventnext-supermodel-medicine . html ( accessed March 2021 ). 16 National Institute for Health and Care Excellence ( NICE ). Current Research & Project [ Internet ]. 2017 . www . nice . org . uk / about / what-we-do / ourresearch-work / our-projects-andpartners ( accessed March 2021 ). 17 NICE . Final Appraisal Document . Kymriah in DLBCL . 2018 . www . nice . org . uk / guidance / ta567 / documents / final-appraisal-determinationdocument ( accessed March 2021 ). 18 NICE . Final Appraisal Document . Yescarta in DLBCL 2019 . www . nice . org . uk / guidance / ta559 / documents / final-appraisal-determinationdocument ( accessed March 2021 ). 19 NICE . The use of real world data for the estimation of treatment effects in NICE decision making [ Internet ]. 2016 . http :// nicedsu . org . uk / wp-content / uploads / 2018 / 05 / RWD-DSU-REPORT-Updated- DECEMBER-2016 . pdf ( accessed March 2021 ).
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