Real-world evidence cases improve the efficiency of clinical trials

Clinical trials often involve a large number of subjects, but subjects tend to worry that they will end up being randomly assigned to a placebo groupThese are two of the most troubling issues for the institutions that conduct clinical trialsCan such a question be mitigated by adopting innovative real-world use cases?a cure for overcoming difficulties?professionals believe that as the cost of clinical trials soars and digital data surges, the FDA has promised to consider real-world data (RWD) in regulatory decisions and to start experimenting with synthetic control armsPharmaceutical product development is on the verge of a new era of evidence generation, which often means that the environment for disruptive innovation has maturedThe next step is a degree of risk-taking, but this approach is not what the pharmaceutical industry is good atsynthetic control arm, it has the potential to be a safe and secure (saving time and money) way to use real-world evidence (real world evidence, RWE)With synthetic control arms, data is not collected from patients with incoming control arms or standard therapeutic armsThe synthetic control arm approach uses RWDs previously generated from sources of health data such as daily medical care to generate and compare data using modeling methods; data sources, including electronic medical records; health insurance claimdata; data generated from fitness trackers, or home medical devices; disease registration; and previous clinical trial datain this way, the benefits to the pharmaceutical industry are clearReducing the number of patients enrolled in the control group, or eliminating the need for group control, allows the use of synthetic control arm methods to increase efficiency, reduce delays, reduce the cost of clinical trials, and speed up the time-to-market of life-saving drugsAssuming a clinical trial, 500 participants need to be included in the treatment group to demonstrate the effectiveness of a new treatmentIf the synthetic control arm method is used, only 500 participants are recruited, and there is no need to recruit 1,000 patients (if synthetic control arm method is not used, usually 500 each for the therapeutic arm and 500 each for the control arm)this mixed trial design, providing the bidders with a way to introduce RWD elements into clinical trials for drug registrationAt the same time, with more accurate information, based on that information, make a decision to move forward, or decisively stop research and development, and reduce the risk of late-stage research and development failureconcerns about being grouped into placebo-controlled groups were one of the main reasons patients chose not to participate in clinical trialsThis concern is exacerbated when an individual has a poor prognosis, or if the effectiveness of the current treatment criteria is limitedThe use of synthetic control arms instead of standard control arms ensures that all participants receive active treatment, thus eliminating concerns about treatment into the groupThe synthetic control arm approach can address the important issues of concern for clinical trial participants and remove the main obstacles to patient recruitmentIf patients use disease-supporting social networks and synthetic control arms are used in clinical trials, social networking posts to introduce treatment details, progress, and communication of side effects that may harm the integrity of clinical trials can help eliminate the risk of patient sacper being blindedregulatory case sitsAlthough synthetic control arms may be a new concept for many professionals or patients, synthetic control arms have been successfully used in regulatory decisionsRoche, for example, applied synthetic control arm clinical trials to meet eu coverage requirements for Alecnsa (Alectinib, Allotini) in 20 European marketsIn December 2015, Alecensa received accelerated FDA approval for the treatment of certain forms of lung cancer, and in February 2017 received conditional approval from the EMAin order to make a pricing decision, the relevant EU authorities asked Roche to provide additional evidence of the effectiveness of Alecensa's treatment for the standard treatment (ceritinib)Instead of waiting for the results of Phase 3 clinical trials, Roche used a comprehensive control group of 67 patients to provide the necessary evidence of relative efficacyThe use of synthetic control arms allowed Alecensa to enter the coverage of 20 European countries by 18 monthsAnother example is Amgen, which uses a synthetic control arm to accelerate approval for blinatumomab for the treatment of rare leukemiasAlthough so far, the application of synthetic control arms has been limitedIn many cases, the chart needs to be manually checked to generate the necessary data;regulator, the name will be the name of theFlatiron Health helped pioneer the use of RWD in clinical research Dr Amy Abernethy, former Chief Medical Officer of Flatiron Health, serves as the FDA's Executive Deputy Director and Chief Information Officer, reflecting the FDA's interest and expectations in the use of RWD It was Flatiron that designed the synthetic control arm method for Roche to provide evidence of the EU's decision to underwrite Alecensa , with Dr Abernethy's expertise and experience, is expected to generate evidence of regulatory use from real-world data, including the use of synthetic control arms, the industry should expect this particular hybrid design to meet regulatory expectations to some extent The RWE use case (data source: resources, drug Mingconde content team mapping) there is no cure even if the FDA uses RWD as a strategic priority, the synthetic control arm is still not a complete replacement for the control arm Synthetic control arms require that the associated diseases are predictable (e.g igoing pulmonary fibrosis) and that the standard of treatment must be clear and stable; is equally important to consider that even if information is available from the RWD source, it may be difficult to extract data or to have poor data quality The daily capture of healthcare data, such as electronic medical records, is often isolated, fragmented and unstructured, often incomplete, and difficult to access New tools and methodologies are needed to integrate, organize, and structure RWDs to generate evidence that can be used for research, and to ensure that promiscuous variables are taken into account in the analysis In order to extract relevant information from structured and unstructured data, analytical techniques such as natural language processing and machine learning are required Despite these challenges, the synthetic control arm approach still has a significant and beneficial effect in drug development, producing low-risk/high-return benefits where appropriate In Phase 2 clinical trials, synthetic control arms were used as comparators for internal decision-making, perhaps as the most concise entry point For bidders and drug developers, the synthetic control arm is familiar before it is intended to be used in critical clinical trials Such an approach would be a business-minded and attractive one For bidders who are prepared to incorporate synthetic control arms into critical clinical trials, focusing on rare diseases with small patient populations, or treatment areas that are relatively easy to obtain information from RWD sources, can help speed implementation and help eliminate barriers to approval a synthetic control arm is a good way for the pharmaceutical industry that wants to start using RWD Compared to traditional randomized controlled trials, the use of synthetic control arm methods is expected to save a significant amount of time and cost, while addressing patients' concerns about being randomized into a placebo-controlled arm although synthetic control arms do not solve all the challenges faced by randomized trials, nor do they fully realize all the promises of real-world evidence in drug development, they are an ideal starting point References: Scott Gottlieb Statement from FDA Director Scott Gottlieb, M.D., on FDA's new strategic framework to advance use of real-world evidence to support development of drugs and biologics Dec 6, 2018 The Future of Clinical Trials: From continuous patient data to trials by algorithm Aug, 2019 Retrieved Nov 26, 2019 https://#zoom=50 S Jenkins, L Fallowfield Reasons for for the inding or nesin ing to the randomined clinical trials for cancer therapy Br J Cancer 2000 Jun; 82(11):1783-8 DOI: 10.1054/bjoc.2000.1142 How Facebook and Twitter can be the next bed a dissona force in clinical trials Nov 13, 2018 Nature 563, 312-315 (2018) doi: 10.1038/d41586-018-07351-8 Concerns over social media and clinical trials Nov 18, 2018 https://cen.acs.org/pharmaceuticals/drug-development/Concerns-over-social-media-clinical/96/i46 Deborah Borfitz Control Arm Supplement: First Step No-Placebo Trials Jul 16, 2019 Retrieved Nov 29, 2019 from http:// CASE STUDY IN NON-SMALL CELL LUNG CANCER Retrieved Nov 28, 2019 from https:// Rebecca A Miksad, Amy P Abernethy Harnessing the Power of Real-World Evidence (RWE): A Checklist to Ensure Regulatory-Grade Data Quality CLINICAL PHARMACOLOGY S FOFORS, 103 (2), Feb 2018, p202-205 doi: 10.1002/cpt.946 9, Vineeta Agarwala, Sean Khozin, Gaurav Singal, et al Real-World In Support Of Precision Medicine: Clinico-Cancer A Case Study Health Affairs, 37 (5), 2018 p765-772 Synthetic control arms can save time and money in clinical trials Feb 5, 2019 Retrieved Nov 12, 2019 from https:// original title: Viewpoints - Real World Evidence Use Cases: Improving clinical trial efficiency and reducing costs, try this approach?