Real-world evidence is being used by the health care sector to support the safe development of new medical interventions including devices, services and biopharmaceuticals. Before, it was only considered in the post-approval phase when bringing a new therapy to market, but now, it is being considered earlier in the clinical trial process. What is real-world evidence, how is it used, why is it important, and how can you start harnessing it?

What is Real World Evidence?

Evidence obtained from analyzing real-world data (RWD) is referred to as real-world evidence (RWE). To generate RWE, the RW data must be analyzed. Different statistical analysis can be done through modeling, or by performing a variety of different studies. Technology, like IBM’s Watson, can also be used for data analysis where it aggregates and reports on insights derived from multifaceted real-world data sets.

This real-world data comes from routine delivery of medical care. For example, RWD can come from clinical data such as electronic health records (EHRs) and electronic Case Report Forms (eCRFs). Patient generated data including medical charts, data gathered from mobile devices or wearables, and Patient Reported Outcomes (PRO). PROs are completed by patients and give researchers firsthand patient perspectives and an understanding of events that go on outside of appointments, operations, and hospital stays. Available public health data such as disease registries, prescription habits, and the use of healthcare services, can give data for the general population.  Lastly, cost data in the form of claims or billing activities can provide a monetary indicator for healthcare spending. Patient identity markers are expressly removed to maintain patient privacy. The RWE extracted from these data sources provide insight into elements that are not included in randomized clinical trials (RCT).

How is RWE used?

Real-world evidence enables researchers to assess the efficacy of medical treatments or interventions while considering other circumstances and variables not always accounted for with RCTs. A randomized controlled trial (RCT) enrolls a small subset of the population and examines how different groups react to a novel therapy in a controlled setting. While RCTs are considered ‘the gold standard’ in clinical testing, they are expensive and take a long time to complete. Rather than using RCTs alone, RWE can provide a more comprehensive insight of how a new treatment option will work in the “real world” and for a more diverse population. By supplementing RCTs with RWE, researchers can gain a better understanding of what works for different sub-populations.

RCT suffer from a narrow pool of trial participants and are not truly representative of the general population. Because RCTs have strict inclusion and exclusion criteria for enrollment, certain populations are expressly not represented in the trial, one such example is pregnant women. It is also the case that trials struggle to recruit candidates from certain ethnic populations, as is common for Hispanic communities. With a lack of diverse representation in clinical trials, the results from RCT do not consider all patient characteristics including age, gender, ethnicity, pregnancy, medical history, etc. However, using RWE can fill in some of these gaps.

Why is RWE important?

Real-world evidence can provide insights into how medicines work in certain patient subgroups that may not have been investigated in RCTs. RWE, for example, can allow researchers to investigate how novel medicines function in patients within specific age ranges, or specific socio-demographic groupings. Real-world evidence can also help researchers comprehend what occurs to a patient over the course of his or her life, not only during the RCT period, to assess the treatment’s effectiveness. How people use a product, what symptoms it does improve, side effects — all of that can be different from what is observed in a controlled trial in a clinic. The healthcare industry is constantly working on improving patient outcomes and reducing hospitalization rates. One way to achieve this is to work on more tailored therapies and personalized medicine. Because RWE insights are so valuable, the FDA employs real-world data and RWE to monitor post-market safety and adverse occurrences, and to support regulatory decisions.

RWE also helps improve healthcare decisions and is used in health economics and outcomes research (HEOR). HEOR measures the outcomes of healthcare interventions and the effect they have on patients to provide data and insights for healthcare decision makers. These decision makers include clinicians, governments, payers, health ministries, patients, to name a few. For these stakeholders, real-world data collection, analysis and reporting is essential.

How to collect and use RWE?

With so many varied data sources (clinical data, patient generated data, public health data, cost data) healthcare stakeholders may choose to use clinical trial and data science services. Evident IQ provides researchers with software to collect real-world data and can provide support to analyze RWD to generate and report on RWE.

As part of clinical trial data collection services, EvidentIQ offers eCRF Setup which simplifies electronic data collection forms, design branching questions, develop in-form and cross-form validations and edit checks to maximize data cleaning at the field level. They also provide configuration support for multilingual eCRF forms, and any localization support needed to configure the application workflow to align with the site’s local processes. Their Patient Reported Outcome (PRO) surveys collect a multitude of data points including treatment preference, quality of life, value of health, disease/treatment burden, and unmet needs. This data is then subject to sophisticated methodologies used to generate unique RWE. Data Linkage is one such method. Data Linkage helps match and merge records from different sources to get a richer dataset and more valuable evidence. EvidentIQ uses significant clinical data expertise, data science analysis, and access to Carenity patient platform, to successfully conduct state of the art data linkage studies. Their innovative data linkage capabilities combine unique self-patient reported data with biomarkers collected by a wearable/sensor through continuous monitoring, bringing RWE to the next level.

Real-world evidence is growing in popularity. Before it was only considered in the post-approval period, but now, it is being considered earlier in the clinical trial process. Using RWE at an early stage in treatment development is a critical consideration for healthcare stakeholders and is driving the trend of gathering more RWE and ultimately improving the safety and effectiveness of medical therapies.

Sources: 

https://veradigm.com/real-world-evidence/

https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence

https://www.arbormetrix.com/blog/9-ways-real-world-evidence-is-changing-healthcare/

https://vial.com/blog/articles/what-is-real-world-evidence-in-clinical-trials/?utm_source=organic

https://viseven.com/what-is-rwe-in-pharma/

https://servier.com/en/newsroom/folders/importance-real-world-studies/

https://rwe-navigator.eu/use-real-world-evidence/rwe-importance-in-medicine-development/

https://www.clinicalleader.com/doc/why-all-the-talk-about-real-world-evidence-0003

https://www.statnews.com/sponsor/2017/02/16/real-world-evidence-need-2/

https://www.ispor.org/heor-resources/about-heor

An evidence generation strategy is an advanced plan for generating evidence necessary for all the steps of the product life cycle. Traditionally, research follows consecutive steps which is more of a reactive process. Using an evidence generation strategy allows for proactive planning and execution of a holistic strategy.

What is evidence generation and what is it used for?

Evidence generation is multifaceted and includes various study designs and data sources. Typically, each department involved in the process (e.g., clinical development, medical affairs, and health economics and outcomes research (HEOR)) works in a siloed structure and independent from one another to come up with evidence to support their specific research topics.

Integrated evidence-generation plan (IEP) is a strategy used where the upfront research plan is developed specifically to find evidence to address the concerns of each stakeholder group (payers, clinicians, patients). Beyond simply proving if a therapy is effective and safe, pharmaceutical companies need to prove that the new treatment is addressing market gaps that the current products do not meet.

Each of these stakeholders have different needs and questions regarding new treatments. Payers, those who pay for the development of the treatment, want to ensure the final product has a value to the healthcare system and will prove to be profitable when launched. Clinicians look for evidence-based research to show how the new treatment is different from existing options. Patients need to know how the new treatment will meet their needs and solve their problems.

An IEP will pinpoint these motivations from each group and strategically set up testing to address each one. Considered in the planning are all functions, geographies, and the entirety of the life cycle to meet the needs of the various stakeholders. Demonstrating the economic, clinical and patient burden of a disease is a key need early in the development process and sets the stage to inform stakeholders regarding the potential value of a product.

Why it is important to have a plan early?

Early evidence-generation planning is key to allowing time to connect all the dots between functions and requirements. It is of vital importance when demonstrating value for the new treatment and it must be demonstrated throughout the lifecycle of the product (early development, clinical research, launch, and post approval) to understand the implications of how the product will address needs at each step along the way.

Impactful data points for each function team (medical, clinical, commercial) can be identified and a roadmap created to ensure the evidence-generation plan includes steps to achieve these data results which will add to the products overall value proposition. A cross-functional and early approach ensures the funding and required expertise is available at all stages.

What are the main steps where you could need evidence?

The ultimate goal of the treatment is to hit the market and provide a solution for the patient, add value to the healthcare community in the form of a differentiated treatment, while being profitable for the developer. Therefore, during the development of the product, it is beneficial to incorporate these factors into each stage of the product lifecycle.

In the early stages of development, there is a need to understand the current state of care for the target patient population. This includes conducting market research, literature reviews and developing a Health Economics and Outcomes Research (HEOR) strategy, identifying possible clinical trial designs, among other tasks that could benefit from evidence-generation. At this point in the product life cycle, the payer stakeholder is interested in the economic burden of disease in order to justify resources required to develop and adopt a new treatment.

During the clinical research and development stage is when patient trials and market access strategies start to be executed. Evidence generation collected from Randomized Clinical Trials (RCT) and patient reported outcomes (PRO) can be paired with Real World Evidence for proof of treatment effectiveness and differentiation of current on-the-market products. Clinicians will be most interested in the data that comes from this stage.

To launch a new product, a medical affairs team may use the evidence to inform their market access strategy and develop communications that target patient needs identified early on in the IEP. The launch stage is where the end prescriber and patient will be convinced of the value of the new treatment based on evidence leveraged through the marketing campaign.

The integrated evidence-generation plan utilizes the research results of all functions: market research, health economics and outcomes research, clinical trials, market access, patient-reported outcomes, literature reviews, etc, and combines them with the strategic objectives and ultimate value proposition. Be proactive with your go-to-market strategy, and plan in advance to address the needs of each stakeholder group. Along with a software suite for clinical trials, EvidentIQ offers the ability to run prospective and direct-to-patient studies with patient-reported outcomes through its Carenity Patient Platform. Carenity part of the EvidentIQ Group is a key partner to consider to support your data generation plan.

Take a look at our RWE offer.

Sources

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5047978/

https://www.openhealthgroup.com/news/17-01-2022/key-elements-of-a-real-world-evidence-generation-plan
https://marimacinsight.com/evidence-generation-strategy/
https://www.futuremedicine.com/doi/10.2217/cer-2017-0073
https://www.evidera.com/wp-content/uploads/2017/05/Strategic-Evidence-Generation-Planning-Product-Positioning.pdf
https://www.mckinsey.com/industries/life-sciences/our-insights/integrated-evidence-generation-a-paradigm-shift-in-biopharma

Patient recruitment has often focused on traditional media methods like TV, newspaper, and radio, which often lead to a low recruitment conversion rate. Digital patient recruitment can target specific communities that are both more likely to be interested in and eligible to participate in the clinical trial which, in turn, can save money and keep the clinical trial on schedule.

What is patient recruitment?

Patient recruitment is the process of identifying, screening, and enlisting eligible patients that are willing to participate in a clinical trial.

There are different methods of patient recruitment. Traditional advertising media channels include TV, radio, or brochures. However, the use of these methods often results in a majority of interested patients being ineligible to participate due to specific exclusion criteria. Investigators can use existing patient databases to identify possible candidates but only patients who are searching for clinical trials would be registered in these. That leaves a large untapped market. Patients can also be recruited directly by their physician or an advocacy group. This recruitment method generally has a higher conversion rate: the number of potential participants informed about the trial vs. number of patients finally recruited to participate. Patients are more likely to be persuaded by a personal connection than a mass marketing advertisement. Both the physician and advocacy group earn the patient’s trust and act as a liaison informing them of the benefits of the trial and calming any hesitations. Digital marketing is also becoming more prevalent. This includes advertising online using keyword targeting. 1 in 3 adults have researched online for medical information. Specific ads are pushed to people who use particular keywords when discussing their conditions on social media or while searching for treatments using search engines. Digital tools can help target wider audiences who already are engaged and interested in a particular illness. These methods typically offer more of a return on investment of advertising funds.

Challenges of patient recruitment

Bringing new treatments to market as quickly as possible is the goal of every clinical trial team. Patient recruitment is a critical part of the trial schedule and budget. If a trial has trouble with patient recruitment, it can extend the length of the trial timeline and incur additional costs. Also, if the trial fails to get enough participants, and the pre-established sample size is not achieved, it could cause the sponsor to abandon the results. Ultimately, efficient patient recruitment can reduce costs and can ensure the trial completion.

One of the main challenges to patient recruitment is that the number of available patients who meet the trial inclusion criteria is often fewer than expected. Since conversion rates are usually low, around 10%, the size of the initial patient pool will affect the total number of participants. The next challenge is identifying and targeting the groups that meet eligibility requirements. Most people are not familiar with what a clinical trial is or how to participate in one. Therefore, in addition to the challenge of finding eligible patients, it is necessary to also educate and convince them to participate. Finally, patient retention is another challenge to completing clinical trials. Patients volunteer to participate in clinical trials and can choose at any time to quit. Prolonged interest in the trial is a vital characteristic that one should look for during the recruitment phase.

How to conduct proper patient recruitment

After the proper approvals have been processed for the trial, and the research team assembled, then it is time to start patient recruitment.

1. The first step to patient recruitment is to select the eligibility requirements for participation and to establish a target audience. Once an ‘ideal’ candidate has been identified, research into that persona type will help the recruiter understand how best to market to them. This research should include:-motivation to participate: What will it take to convince the patient to volunteer their time and energy to participate? Are they motivated by money? In the search for a cure? Will the trial help their condition? What is the value proposition you are offering the candidate? Clearly articulate the relevance of the study and emphasize the possible benefits in your marketing materials.-condition area:  What condition do they have? The type of condition may affect a patient’s willingness to participate in trials. According to a study, patients with chronic conditions are less likely to participate in trials for new drugs because it can take a long time to find the correct treatment for these conditions. You should tailor the outreach approaches to match the type of condition.-best marketing media: What marketing channel will they most interact with? What age is the ideal candidate for your study? What media type do they want to interact with? Does this person want a brochure, video, blog, pdf to download? The marketing strategy you develop should match these aspects of your candidate.
2. Create a simple process for participation. The intake paperwork should be user-friendly, easy to understand, and accessible. Electronic enrollment is one way to simplify this process and gain more interaction with possible participants. Digital tools include online screening questionnaires, scheduling services, and trial information materials. It should be easy for a participant to contact someone involved in the trial and ask questions; this can also be done online.
3. Next, set goals, milestones, and track the conversion rate– how many people see and interact with your marketing vs how many enroll. This will help determine the number of people that need to see your ad before you get enough for your trial- help determine the type of advertising. You may do this through a pilot study and/or through analysis of past trial recruitment statistics.Once you have one persona for each of your ideal candidates, then you may choose to run a pilot study. A pilot study is a scaled down version of the clinical trial requiring fewer participants which tests the trial’s feasibility. If the pilot study fails to recruit enough patients, then it is likely the trial will also fail, and resources would be wasted. The pilot study will also help the researchers identify potential barriers to patient recruitment. Adjustments to the recruitment strategy can be implemented to avoid the same pitfalls during the patient recruitment. This may include changing screening criteria, adding more test sites, or modifying the marketing mix.   If you choose not to do a pilot study, you may analyze data from your past enrollment performance to help you determine a feasible enrollment estimate. This will allow you to adequately manage the finances and staff resources of the study. Pay attention to the number of patients recruited, how long it took to get the first enrollment, and what percentage of the enrollment goal was achieved. Use this past data to identify what form of marketing worked best for the audience.
4. Create a marketing strategy. Identify the marketing mix for your message (place-where will you advertise, product-how will you position your trial, price-what is it that you will give the volunteers, promotion- how will you tell your audience about the trial, process-what is the process to be involved, people-who is involved to get your trial participants). Make sure the information is clearly written and easy to understand, pay attention to not use complex medical jargon. Patients need to feel comfortable with the trial and understand the process. The marketing elements should have a clear call to action. Offering them a roadmap of what is expected of them and how the results will be used.
5. Start recruiting and stay organized. Execute your carefully planned recruitment. Track all your results and capture, in detail, the contact information from interested patients, even those that may not be eligible because perhaps they will be interested in a future study. Remember to ensure patient privacy and that your data collection/retention is following all privacy laws.

Best practices

Tailor communication strategy. Communication is key to building trust, establishing expectations, explaining processes, and keeping the patients informed. The communication plan starts the first interaction the patient has with the trial. The marketing materials should anticipate and answer the patients’ questions. Do the images and terminology reflect the target population? Take into consideration cultural and language differences of your participants. This will make them feel more at ease and comfortable with the process of the trial.

Follow up with patients. Incorporate digital elements like text messages, automated emails or personalized scheduling reminders. These tools can be used to remind patients to take the next steps. Is there constant contact with the trial participant to keep them involved in the trial? Incorporate a range of outreach methods to reach patients and provide them with the necessary information and useful content to keep their interest.

Answer every question the patient has regarding the trial. Keep the physician or advocacy group involved and address any doubts, concerns and to convey the benefits of the study. Use social media platforms to keep connected with the audience and inform them of the results via mixed media like infographics and video.

Show appreciation for the trial participants. Many are receiving financial compensation but that is not always enough to get a participant to “buy into” the study and continue to give their time. How can this trial benefit them- does it help give them a better understanding of their condition? The trial will also move research forward and can help other patients like them in the future.

Keep it interesting. Recruitment and retention are largely affected by the enthusiasm of the lead investigator. If they are showing interest and providing proper follow up because they believe in the success of the study, the participants will too.

Include digital tools. Online aspects are used for patient recruitment, retention, and for the trial itself. Digital patient recruitment can target more precisely a larger audience. Follow up messages via email, social media, or text messages are a great way to encourage patients to take the next step and keep them interested. Tracking enrollment numbers and keeping an updated database can help keep investigators organized. The clinical trial itself can be performed ‘decentralized,’ where the patients are all virtual. Digital clinical trials can widen the eligibility pool. If the trial is available remotely, then there are more people that can participate as location is no longer a limitation. In-person aspects of the trial should be coordinated in conjunction with local health sites. Direct-to-patient lab services are also available and can be explored when necessary.

Recruiting enough patients that qualify for clinical trials is always a challenge. Traditional marketing methods often fail to recruit enough trial participants. Now, with the use of digital tools, it is possible to more efficiently and cost effectively target patients willing and able to participate in trials. EvidentIQ offers a suite of applications within a single integrated cloud platform for clinical data management, clinical operations, and patient centricity. These digital tools not only help for recruitment but also with patient tracking, communication, retention, and can provide statistics for when planning your next clinical trial.

Take a look at our complete offer here.

Paris (FRANCE), 09.12.2021: Data Linkage by Carenity, part of EvidentIQ demonstrates the group’s unique position to support sponsors better understanding characteristics and needs of the population, with the goal of promoting the development of better tailored health products and services.

Data Linkage is a method that helps match and merge records from different sources to get a richer dataset. This process always uses the highest standards of privacy and safety. The linkage of information enables the construction of chronological sequences of events and when used at the macro level provide valuable information for policy and research into the health and wellbeing of the population.

EvidentIQ uses significant clinical data expertise, data science analysis, and access to Carenity, their patient platform, to successfully conduct state of the art data linkage studies.

Carenity is a social network specially designed for patients with chronic diseases and their caregivers. It has more than 500,000 members in 6 countries (France, Spain, Italy, Germany, United Kingdom and the United States), which gives EvidentIQ a firsthand access to healthcare ad-hoc data if needed.

By working on projects with top 5 pharma, EvidentIQ is showing its proficiency in data generation and is offering its clients a next-level data linkage experience combining data with patients’ insights in a real-world setting using a proactive approach.

Contact us for more information on Data Linkage by EvidentIQ.

PatientLive® is a unique solution that allows to quickly survey an online community of 500,000 patients and caregiversstudies

Paris (FRANCE), 25.10.2021. Today EvidentIQ announced that its PatientLive® software is opening its doors to the clinical market. The PatientLive® platform grants access to real-time insights and reliable answers from patients and caregivers through quick polls.

PatientLive® was created by Carenity, part of the EvidentIQ group, in 2016 with the purpose of offering pharma companies a unique and fast way to get access to real-time patient insights. The Carenity team believes that guesswork is no longer enough but knows that time is also valuable. Therefore PatientLive® allows to collect real-time patient insights at a fast pace, in order to help make better strategic decisions regarding clinical patient-centric projects.
Carenity’s mission is to bring patient perspective into clinical projects to accelerate clinical trials, save costs and ensure the success of new products. With this revolutionary software, the life science industry can leverage real-time patient insights at each phase of the clinical development like early R&D, Clinical development, Clinical operations, Patient engagement, and Medical affairs.

PatientLive® can help understand patient experience (burden, Quality of life) to define Target Product Profiles, evaluate patient’s acceptance of protocol evaluation parameters and flow chart, incorporate study endpoints that are valuable from a patient perspective, optimize the design of clinical trials to facilitate patient recruitment and retention, identify preliminary feasibility for clinical projects, ensure study materials are patient-centric and easy to understand (ICF testing), and test concepts of services and tools to increase patient retention on clinical trials.

Michael Chekroun, Chief Strategy and Transformation Officer at the EvidentIQ group and Founder of Carenity, tells us about this offering:

“PatientLive® has always offered quick responses and has been characterized for its simplicity and autonomy of use. We want now to make this available for the clinical markets and keep this virtuous circle of Data for Good going.”

More about PatientLive®

PatientLive® is a unique solution that allows to quickly survey an online community of 500,000 patients and caregivers in the US and Europe with up to 36 conditions available, letting users acquire real-time insights via a five-question poll. Five types of questions including images and videos are available: single choice, multiple choice, 0 to 5 scale, text field, and numeric field. Polls on PatientLive® initiate patients data generation on topics including: disease/treatment burden, unmet needs, quality of life, services and communication message testing, and information and service needs. These early insights are invaluable for PatientLive® users as they enable better patient-centric solutions and materials and may guide the development of further non-observational studies and Real World Evidence projects.

Contact us for more information on PatientLive® by EvidentIQ.

Number of vaccine refusers highest in France, lowest in the UK

Hamburg, 12.05.2021: Data science group EvidentIQ, with its subsidiary company Carenity, has published a survey with 3,353 participants on the situation of chronic patients during the pandemic. The target group included adults from France, the UK, Germany, Italy and the US. Among other findings regarding the number of canceled medical visits and procedures, or the rise of telehealth services, the difference in preparedness for vaccination per country stood out.

The motivation to get vaccinated does not come as a surprise. 48 percent state they are at risk, 46 percent want to protect others and 41 percent simply want to protect themselves from the virus. Only 18 percent indicate it would be easier to travel when vaccinated. The most frequent chronic conditions among the respondents are Type 2 diabetes (24%), asthma (19%), multiple sclerosis (15%) and COPD (14%). Given the severity of these illnesses, it is surprising that a mere 67 percent in France and 47 percent in Germany do not intend to get vaccinated.

Michael Chekroun, Chief Strategy and Transformation Officer of the EvidentIQ group and Founder of Carenity explains these results:

“When it comes to the COVID-19 vaccines, nearly half of our respondents are afraid of side effects. Another 45 percent name the lack of information as reason for their reluctance to be vaccinated. To some extent this is understandable.”

Chekroun refers to the comparably short development and trial phases of the new vaccines. For these highly regulated trial processes imposed by public entities like EMA and FDA – EvidentIQ provides software infrastructure, scientific services and even access to patients to secure and automate testing procedures.

“Many people are not aware that for extraordinary situations like these there have always been Accelerated Approval Programs to shorten time-to-market for a new treatment. This is obviously where chronic patients are understandably nervous, fearing side effects and requiring more information. Our survey shows that only 4 percent of all respondents generally oppose vaccination.”

Also, the survey shows that 46 percent report that medical visits or procedures have been canceled or postponed in the first wave of COVID-19, while 75 percent report the same for wave 2.

This may explain why roughly 67 percent of patients in the US are open to the use of telehealth consultations, in Europe only 47 percent.

Read the the full interview with Michael Chekroun on the outcome of this survey and get a free copy of the complete results.

Contact us for more information.