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Digital Health Technologies and Innovations
Published in Kelly H. Zou, Lobna A. Salem, Amrit Ray, Real-World Evidence in a Patient-Centric Digital Era, 2023
Kelly H. Zou, Mina B. Riad, Shaantanu Donde, Joan van der Horn, Tarek A. Hassan
For example, the 21st Century Cures Act, signed into law in the US on December 13, 2016, has defined real-world evidence (RWE) in Section 3022, where “the term ‘real world evidence’ means data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than randomized clinical trials” (U.S. Congress, 2016; FDA, 2022b).
Designing and Delivering a DTx Clinical Research Program: No Need to Re-invent the Wheel
Published in Oleksandr Sverdlov, Joris van Dam, Digital Therapeutics, 2023
Colin A. Espie, Alasdair L. Henry
The chapter has considered the types of evidence and types of study that may comprise elements of the clinical research pipeline. While recognizing the advantages of a “mix” of methodologies, for the foreseeable future, the randomized controlled trial should remain the staple clinical trial, supplemented, but not replaced by real-world evidence. Indeed, the fairly unique opportunity to recruit large numbers of participants into RCTs should be capitalized upon in DTx. SaMD offers scalability that should make for more efficient, more cost-effective, and quicker trials than is possible for potential competitor industries like pharmaceuticals and in-person care. How to conduct clinical trials is also fairly “settled science” internationally and is well documented. So much of the heavy lifting has already been done. Therefore, the DTx industry primarily requires a commitment to such scientific rigor and must recognize that regulatory approvals are a relatively low bar compared with clinical guideline care.
Using Real-World Evidence to Transform Drug Development: Opportunities and Challenges
Published in Harry Yang, Binbing Yu, Real-World Evidence in Drug Development and Evaluation, 2021
In the past several years, the FDA published several guidelines for the industry regarding use of RWE, including (1) “Submitting Documents Using Real-World Data and Real-World Evidence to the FDA for Drugs and Biologics Guidance for Industry” (FDA 2019b), (2) “Rare Diseases: Natural History Studies for Drug Development” (FDA 2019a), (3) “Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices” (FDA 2017), and (4) “Final Guidance for Industry: Use of Electronic Health Record Data in Clinical Investigations” (FDA 2018a). In addition to the above guidelines that have already been issued, the FDA will be providing additional guidance documents including (1) guidance on how to assess whether RWD from medical claims, EHRs, and registries are fit for use to generate RWE to support effectiveness; (2) guidance for using RWD in RCTs for regulatory purposes, including pragmatic design elements; (3) guidance on the use of RWD to generate external control arms; and (4) guidance about observation study designs, and how these might provide RWE to support effectiveness in regulatory decision-making.
Costs of radium-223 and the pharmacy preparation 177Lu-PSMA-I&T for metastatic castration-resistant prostate cancer in Dutch hospitals
Published in Journal of Medical Economics, 2023
S. W. Quist, J. H. J. Paulissen, D. N. J. Wyndaele, J. Nagarajah, R. D. Freriks
It is important to relate clinical trial data to real-world evidence. Currently, several real-world data projects in prostate cancer are being conducted, which might help homogenize treatment patterns for patients with mCRPC.42 Older Dutch real-world data suggest that the patient characteristics of mCRPC in ≥ third-line treatment slightly deviate from the clinical trial data (i.e. similar age but higher ECOG).14,16,43 However, patient characteristics and treatment effects do not necessarily affect the treatment course and therefore, in our case, the study outcomes. To account for the possibility that subsets of patients receive different numbers of injections, we provided a detailed overview of the different cost parameters to make the results translatable to different clinical practices.
Real world data for rare diseases research: The beginner’s guide to registries
Published in Expert Opinion on Orphan Drugs, 2023
Federica Pisa, Ariel Arias, Emily Bratton, Maribel Salas, Janet Sultana
Recently, the US Food and Drug Administration (FDA) as well as European investigators have published definitions of real-world evidence (RWE) and multiple guidance documents to use RWE for regulatory purpose. According to the FDA, real-world evidence (RWE) is defined as ‘the clinical evidence regarding the usage, and potential benefits or risks, of a medical product derived from analysis of RWD’ [1], while in Europe, RWE is defined as ‘the information derived from the analysis of RWD’ [3]. In earlier stages of the drug lifecycle, RWE enables quantifying and characterizing the target population, understanding the disease characteristics, contributing to the planning of clinical trials, and complementing risk-benefit assessment [5]. In later stages, RWE makes it possible to confirm drug effectiveness in real-world patient populations, to identify subgroups, and to assess drug safety.
Effectiveness of polyene phosphatidylcholine and its combination with other drugs in patients with liver diseases based on real-world research
Published in Expert Review of Clinical Pharmacology, 2022
Ying Li, Anni Chen, Zhizhen Li, Xiuliang Cui, Guoqing Zhang
With the rapid development of information technology, real-world evidence from medical records has become an important data source for clinical medical research. Real-world research is rooted in clinical practice and comes from a wide range of sources, including electronic medical records, laboratory examination, imaging data, and follow-up records during diagnosis and treatment. There were multiple studies based on real-world evidence demonstrating the effectiveness of different drugs, such as erenumab for headache and apatinib for metastatic colorectal cancer [15,16]. Our aim was to explore the effectiveness of PPC in patients with liver diseases based on real-world research, and compare the treatment effectiveness with other hepatoprotective drugs (alone or combination medication), in order for optimal regimen therapy in clinics to treat hepatopathies.