scholarly journals PBI43 REAL-WORLD EVIDENCE RECOMMENDATIONS FOR GENE THERAPIES: REGULATORY VERSUS HEALTH TECHNOLOGY ASSESSMENT

2020 ◽  
Vol 23 ◽  
pp. S21
Author(s):  
G. Allen ◽  
D.M. Summers ◽  
J. Evans
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Real World ◽  
Health Technology ◽  
Gene Therapies ◽  
Real World Evidence

scholarly journals Real-world evidence: perspectives on challenges, value, and alignment of regulatory and national health technology assessment data collection requirements

2021 ◽  
Vol 37 ◽  
Author(s):  
Hannah Sievers ◽  
Angelika Joos ◽  
Mickaël Hiligsmann
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Real World ◽  
Health Technology ◽  
Practical Experience ◽  
Added Value ◽  
Real World Data ◽  
Conflicting Demands ◽  
Real World Evidence ◽  
Semistructured Interviews

Abstract Objective This study aims to assess stakeholder perceptions on the challenges and value of real-world evidence (RWE) post approval, the differences in regulatory and health technology assessment (HTA) real-world data (RWD) collection requirements under the German regulation for more safety in drug supply (GSAV), and future alignment opportunities to create a complementary framework for postapproval RWE requirements. Methods Eleven semistructured interviews were conducted purposively with pharmaceutical industry experts, regulatory authorities, health technology assessment bodies (HTAbs), and academia. The interview questions focused on the role of RWE post approval, the added value and challenges of RWE, the most important requirements for RWD collection, experience with registries as a source of RWD, perceptions on the GSAV law, RWE requirements in other countries, and the differences between regulatory and HTA requirements and alignment opportunities. The interviews were recorded, transcribed, and translated for coding in Nvivo to summarize the findings. Results All experts agree that RWE could close evidence gaps by showing the actual value of medicines in patients under real-world conditions. However, experts acknowledged certain challenges such as: (i) heterogeneous perspectives and differences in outcome measures for RWE generation and (ii) missing practical experience with RWD collected through mandatory registries within the German benefit assessment due to an unclear implementation of the GSAV. Conclusions This study revealed that all stakeholder groups recognize the added value of RWE but experience conflicting demands for RWD collection. Harmonizing requirements can be achieved through common postlicensing evidence generation (PLEG) plans and joint scientific advice to address uncertainties regarding evidence needs and to optimize drug development.

OP170 Regulatory And Health Technology Assessment Considerations In Alzheimer's Disease

2018 ◽  
Vol 34 (S1) ◽  
pp. 61-62
Author(s):  
Jacoline Bouvy ◽  
Pall Jonsson
Keyword(s):  
Alzheimer’S Disease ◽  
Alzheimer's Disease ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Real World ◽  
Data Access ◽  
Health Technology ◽  
Advisory Group ◽  
Real World Evidence ◽  
Disease Modifying

Introduction:There has been a move towards the development of disease-modifying agents in Alzheimer's disease (AD) and it is likely that early disease-modifying treatments will initially be offered to people who have positive AD markers and mild cognitive impairment (MCI). Consequently, disease-modifying drugs will have distinctive features as compared to currently licensed symptomatic treatments, which makes the implications of these new agents for regulatory and health technology assessment (HTA) processes unclear.Methods:The ROADMAP (Real-world Outcomes across the AD spectrum for better care: Multi-modal data Access Platform) project provides the foundation for a European data platform for real-world evidence in AD, and established an expert advisory group (EXAG) consisting of regulatory and HTA experts. This presentation will summarize the key lessons from the first year of ROADMAP's EXAG and identifies the next steps that are required to prepare Europe's healthcare systems for a disease-modifying drug.Results:The EXAG identified a need for: (i) establishing the rationale for the selection of priority outcomes in pre-clinical AD and MCI; (ii) establishing accepted outcomes for defining prevention of AD or delayed AD onset; (iii) exploring modern technology that could assist in testing cognition that could easily be used in clinical practice; and (iv) establishing caregiver-relevant outcomes (e.g. quality of life, loss of income, carer time) that are important to capture; and found that not all evidence to support modelling assumptions can be generated through RCTs, making the case for using real-world evidence.Conclusions:Many of the challenges that the EXAG identified can be solved by generating better real-world data in AD. There is a clear need to agree on the outcomes that will facilitate and inform regulatory and HTA decision-making. Once the gaps in the availability of outcomes in AD will be closed, we will be one step closer towards being ready for a disease-modifying drug.

OP29 Building A Global, Public Repository Of Patient Experience Data

2019 ◽  
Vol 35 (S1) ◽  
pp. 7-7
Author(s):  
Catherine Holliday
Keyword(s):  
Health Technology Assessment ◽  
Patient Experience ◽  
Technology Assessment ◽  
Real World ◽  
Muscular Atrophy ◽  
Health Technology ◽  
Structured Interview ◽  
Real World Evidence ◽  
Qualitative Component ◽  
The Impact

IntroductionThe Patient Experience, Expectations and Knowledge (PEEK) protocol was developed so that a holistic, comprehensive, independent, proactive and systematic approach could be taken to inform decisions made in the context of health technology assessment and other parts of the health sector. Each PEEK study is made publicly available which over time will result in a global repository of patient experience data.MethodsThe PEEK protocol is a single protocol that can be implemented across disease areas and includes a quantitative and qualitative component. The quantitative component is based on a series of validated tools that provide baseline health and demographic data for the study population. The qualitative component is the result of two years of protocol testing to develop a structured interview that solicits comprehensive and holistic patient experience data, and provides participants with the opportunity to provide advice on their future expectations.ResultsPEEK studies in breast cancer, bladder cancer, lung cancer, spinal muscular atrophy, atopic dermatitis, chronic kidney disease, chronic heart failure and mitochondrial disease have been completed in the Australian context (www.cc-dr.org/peek). Holistic patient experience themes are presented commencing with symptoms and diagnosis experience, through to communication, information, treatments experienced and quality of life. Information is also available in relation to participant's expectations of future treatment, care, information and communication. The result is a freely available repository of patient experience data that anyone in the sector can access to complement clinical and economic evidence.ConclusionsThe process of providing patient feedback and real-world evidence in the context of health technology assessment is often ad-hoc. The lack of consistency means that it has been difficult to assess the impact of patient engagement and feedback in the context of health technology assessment. The PEEK protocol and program is an example of a systematic, independent and holistic approach to patient experience and real-world evidence data collection that provides the sector with an opportunity to proactively engage the community in decisions that are made about treatment, care and support.

R WE ready for reimbursement? A round up of developments in real-world evidence relating to health technology assessment: part 4

2021 ◽  
Author(s):  
Alex Simpson ◽  
Sreeram V Ramagopalan
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Real World ◽  
Clinical Benefit ◽  
Health Technology ◽  
Real World Evidence

In this month’s round up, we discuss a number of recent publications and guidelines addressing the use of real-world evidence to evaluate the clinical benefit of health technology assessments and what the publications mean practically for manufacturers.

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