How to use RWE to reduce cost and improve benefit in pharmaceutical industry

Without raising the price of drugs, the external pressure on the pharmaceutical industry to maintain innovation has never been stronger How can the pharmaceutical industry actively use real world evidence (RWE) to significantly reduce costs and improve efficiency? Evidence generation in the pharmaceutical industry is usually sequential and isolated In terms of regulatory decision-making, this "one size fits all" approach was sufficient in the past, but it is no longer applicable Many external factors, including the increasing regulatory requirements for real world data (RWD); the end point related to payers and patients, playing an increasingly important role in reimbursement decision-making; in addition, the increasingly strict constraints on health care budget, to deal with these external factors, a new comprehensive approach is needed Dr Melvin Olson, director of global real world evidence (RWE) strategy and innovation of Novartis, put forward an evidence driven operating framework (epof) based on the previous "call to action" Through the application of comprehensive evidence generation strategy in the pharmaceutical industry, the pharmaceutical industry can change its use of real world evidence World identity, RWE) In 2018, in Deloitte's benchmark survey of the pharmaceutical industry, the executive leadership team interviewed said that as part of the relevant company's strategy, the importance of RWE will increase dramatically in the next two years This situation is reflected in that 90% of the surveyed companies have invested a lot in RWE throughout the product life cycle in order to achieve cost savings and benefits Indeed, it is expected to shift the focus of RWE applications to optimizing trial design and execution, value-based contracts, and supporting enrollment submission / tagging extensions (in addition to current commonly used methods, such as burden of disease, patient safety, and evidence of efficacy of patient subgroups) These trends are challenging the traditional operation mode of the pharmaceutical industry and promoting the earlier application of RWE in the product life cycle There are reasons for such investment: 70% of clinical trials are plagued by recruitment barriers; for each extra week spent on new drugs entering the market, the resulting sales loss will be as high as $300000 In addition, with the increasing interest and use of RWE for decision-making by drug regulatory agencies in many countries and regions, some companies actively respond and invest a lot of manpower, material resources and financial resources as part of the strategy to obtain RWD and generate RWE In 2015, for example, Novartis launched a feasibility study to assess the interest of leading rheumatologists in jointly creating a European research collaboration network to collect data on patients with spinal arthritis (SPA) Then, the eurospa research collaboration network of 16 independent registration agencies from 16 countries was formed Through the coordination center of leading and supervising research and collaboration, researchers can share data, technology and clinical expertise, and establish infrastructure By the end of 2017, through this network, research could be conducted on about 63000 patients who are using arthritis treatment drugs and biological products Therefore, through such collaboration, it can provide more in-depth clinical insights and enhance the real-world research capabilities for patients In addition, in the European network project, Novartis has also contributed to the electronic medical record data of the innovative drug program The project aims to carry out large-scale analysis of health data from about 100 million EU citizens through the establishment of a joint data network from 22 partners The entire joint data network covers 12 countries The comprehensive method of evidence generation mentioned here is to provide effective use of RWE to support activities within the product life cycle and between multiple internal functions of the company, so as to meet the needs of many external stakeholders more effectively In developing a comprehensive evidence strategy, RWE can provide several concepts of information, including endpoint strategy, phase 3 population identification in clinical trials, randomized controlled trial recruitment, patient reporting outcome, outcome protocol, and effectiveness prediction Although the relevant concepts are remarkable, they are difficult to put into practice without specific direction and background What you need is an evidence driven operating framework (epof), which is used in pharmaceutical organizations to develop evidence generation strategies in a truly integrated way The purpose of the evidence driven operational framework (epof) is to bridge the usual gap between research, clinical development, market access and business insight For example, while RWE is often used to identify patient insights (for example, to validate and inform patient-centered endpoints / outcomes), and to provide business strategies (for example, to ensure market access) Its scope of application can be extended to preclinical and clinical research (for example, to improve the output rate through accurate target and patient queue identification), as well as to optimize the design and implementation of trials (for example, to identify potential patients and research sites through identification) All of these pillars are linked to natural organizational functions to ensure that evidence of excellence is provided throughout the product life cycle A framework based on these pillars will help ensure that evidence strategies and decisions are optimized through the use of RWE; that patients are placed first in evidence planning, that all activities are evidence-based and resource efficient; and that such a framework has a real competitive advantage over other products In terms of improving the efficiency of pharmaceutical development and commercialization, there is a huge space to save costs A 2016 report estimated that the pharmaceutical industry could save $1 billion a year by using RWE Some of the most successful companies have realized great value through the use of RWE platform in the product life cycle Therefore, evidence driven operational framework (epof) can help to achieve cost savings The expected cost savings can be mapped to the epof pillar to be adopted; some companies realize and maintain the significant value of products in the whole product life cycle by using the integrated evidence generation framework For example, through the cooperation with trinetx (global health research network), Novartis has used the cloud platform to analyze RWD collected from contract research organizations, healthcare institutions and data partners, using advanced analysis methods Since 2018, it has conducted early feasibility analysis on more than 150 clinical trials in multiple disease areas Trinetx is a global healthcare research network organization that optimizes clinical research and enables new drug discovery by creating RWE Trinetx will access longitudinal clinical data in real time, and combine with the most advanced analysis technology to quickly answer complex research questions From the perspective of regulation and market access, these feasibility analyses may lead to an increase in the probability of success of the trial, a reduction in recruitment time and a higher correlation with the trial population In addition, by observing how airlines and power companies use technology-based "crisis centers" to prevent failures and power outages, Novartis has developed a global monitoring center to map and monitor 500 clinical trial networks in 70 countries / regions, predicting and correcting potential problems around the clock In this way, Novartis plans to reduce recruitment time by 15% There are many successful examples of using RWE in the initial approval of drugs or the approval of indications expansion These methods are beyond the traditional safety signal assessment, risk management, and benefit risk assessment methods, showing the importance of RWE for successful regulatory decisions In the case of kymriah ®, RWE was used to identify and consider the response rates observed in single arm clinical trials in adult patients with recurrent or refractory diffuse large B-cell lymphoma (DLBCL) Because diffuse large B-cell lymphoma is a rare disease, randomized controlled trials are not considered feasible In addition to the strong unmet needs of patients, RWE is considered acceptable and sufficient in this case Through the strategic use of RWE, for example, by early identification of target populations and end points / outcomes, to improve test criteria, In order to increase the probability of success of the feasibility study, strategic choice of the place to successfully register patients, innovative trial design (for example, cluster randomization, as well as the use of external control and bridging studies), and ensure the optimal reimbursement conditions can achieve significant cost savings Although this requires upfront investment in data and infrastructure, which means that net savings will be reduced in the initial stage, the final implementation of the strategic use of RWE will enable users to make better decisions In order to make full use of RWE, improve efficiency and make wise decision, we need to establish a framework similar to evidence driven operation framework (epof) The successful implementation of these frameworks requires close cooperation and strong leadership among multiple functional departments to form a joint force, promote awareness change and integrate RWE into specific actions Without raising the price of drugs, the external pressure on the pharmaceutical industry to maintain innovation has never been stronger It is a self-restraint method for the pharmaceutical industry to deal with this situation through continuous cost reduction; the real goal is to improve efficiency and output rate with RWE as the driving force Although some senior management teams in the pharmaceutical industry may be worried about a large amount of early investment, in the increasingly demanding healthcare system, in order to maintain a leading position, it is necessary to try first, generate meaningful evidence to meet the needs of stakeholders, and effectively save costs These cost savings may be difficult to quantify, or may not reach the estimated $1 billion per year, but the cost savings will be considerable and worthy of action It is necessary for the pharmaceutical industry to invest in and implement new business models and frameworks to promote a comprehensive approach to generating evidence throughout the product life cycle This method has completely changed the understanding of how to generate evidence, which is a pioneering way Original title: viewpoint | real world evidence use case: can Novartis' framework approach save the pharmaceutical industry $1 billion a year? 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