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17 Nov 2021

Updated 22 Nov 2021

The expanding role of Real-world Evidence in early clinical development

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The expanding role of Real-world Evidence in early clinical development

Why collecting Real World Data is important to optimize clinical trials and get new medical product approved? 

In clinical development, the traditional approach to demonstrate efficacy and safety of a new drug has always been in performing the traditional Randomized Clinical Trials (RCT) programs since this is considered by Regulatory Agencies as the best compromise to get sufficient, well-structured data from a subgroup of the future patient population in a reasonable time frame. However, these programs are highly cost-consuming and require the involvement of human resources (physicians, clinical research associates, etc.)

When a drug is assessed, Regulators and Health Technology Assessment (HTA) bodies require getting a far more extended set of data from a larger population and closer to normal life conditions. Having access to real life data enable to have a better understanding of disease and treatment burden, patient experiences, preferences to be able to establish how much the drug will cover unmet needs. 

What is Real-World Data (RWD) and what are the different sources? 

Real-world data is the data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources, for example: 

  • Electronic health records (EHRs) 
  • Claims and billing activities 
  • Product and disease registries 
  • Patient-generated data including self-reported data entered by the patient or its relatives (e.g Patient-Reported Outcomes – PROs) 
  • Data gathered from other sources that can inform on health status, such as mobile devices 

What is Real-World Evidence (RWE)? 

Real-world evidence is the clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of RWD. RWE can be generated by different study designs or analyses, including but not limited to, randomized trials, including large simple trials, pragmatic trials, and observational studies (prospective and/or retrospective). RWE is increasingly used in drug development and evaluation as shown by the recent guidelines published by the FDA. 

What is new in July 2021 FDA guidelines? 

Since 2016, after the publication of the 21st century Cure Acts, the FDA is taking into account RWD for the evaluation of new drugs/medical devices, mostly to monitor post-marketing safety or support extension of indications.  

In July 2021, the FDA published guidelines to illustrate the interest of implementing RWD during clinical development phase and not only at the end of that lengthy phase. There are many approaches from including RWD in the RCT up to implement specific trials in parallel to the pivotal program. RWE is not a substitute for RCTs but should be seen as complementary, each type of study design providing information the other cannot. 

Due to long processes during new drug/medical device development, pharmaceutical companies should evaluate the best approach to collect data as early as possible. Indeed bringing patient perspective into clinical projects can accelerate clinical trials, save costs and ensure the success of new products. .  

For Medical Devices, the FDA is also relying on RWE to support their decisions and to evaluate risk/benefit.  

EMA (European Medicines Agencies) is also working with European National Agencies on a strategic plan for 2023 road map in favor of the use of RWE in clinical development considering the benefit of having access upfront to real world data. 

Some examples to Illustrate how FDA rely on RWE to support regulatory decision-making

In July 2021, U.S. Food and Drug Administration approved a new indication for Prograf (tacrolimus) based on an observational, non-interventional study providing real-world evidence (RWE) of effectiveness. FDA approved Prograf for use in combination with other immunosuppressant drugs to prevent organ rejection in adult and pediatric patients receiving lung transplantation.   

In a more recent time, FDA has decided for the Pfizer-BioNTech COVID-19 Vaccine to allow for use of a single booster dose, to be administered at least six months after completion of the primary series in specific subset of population: the decision was partly based on real-world data on the vaccine’s effectiveness over a sustained period of time provided by both U.S. and international sources, including the CDC (Centers for Disease Control and Prevention), the UK and Israel.  

How Carenity can help you? 

Carenity, part of the EvidentIQ group, offers Real-World Evidence solutions to collect and analyze self-reported patient information (ePROs, patient preference information using Discrete-Choice Experiment designs, e-diaries, etc.). 

Carenity relies on its global digital platform that engages more than 500,000 patients and caregivers in Europe and North America to conduct prospective non-interventional studies (cross-sectional and longitudinal designs). Theses observational studies can generate meaningful information about quality of life, disease and treatment burden, product effectiveness, patient preferences, etc. 

Carenity supports its Life Sciences Industry clients at each step of the clinical development to ensure the success of patient recruitment and retention thanks to patient-centric decision-making:  

  • Target Product Profiles (TPP) definition 
  • Study design optimization and protocol evaluation 
  • Study feasibility assessment 
  • Endpoint optimization 
  • Test of patient materials (Informed Consent Form, Information notice, etc.) and patient services 

Carenity also offers PatientLive®, a self-service platform to collect real-time patient insights.  This unique solution allows to get fast answers from patients and caregivers and make better decisions via a quick poll (1-5 questions). 

Contact us for more information: info@evidentiq.com 

Image Credit:Foto istock by metamorworks

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