As Eros Papademetriou at SmartAnalyst explains, real-world evidence is central to this understanding, providing drug developers with a true picture of their therapies’ performance.
The total number of confirmed COVID-19 infections worldwide recently surpassed 148 million people, with some three million deaths since the start of the pandemic.1 This is a sad milestone in what has been a year of suffering and disruption.
Although mass vaccination programmes are accelerating across the globe, giving some hope that the world will soon be able to return to something approaching “normal” life, many health experts are advising that we are still some way off seeing the end of the pandemic.
No vaccine can completely eliminate virus transmission under trial conditions. Unanticipated circumstances in the real world may further affect infection rates even among vaccinated populations.
Differences between individual immune systems, combined with variations in people’s behaviour, may affect how well they respond to vaccination.
The persistently high rates of infection around the world may also increase the likelihood of new strains emerging that are resistant to existing vaccines.
For those currently hospitalised with COVID-19, it may be the case that existing treatments for the most serious symptoms do not work for them. They may even suffer from adverse side-effects from the therapies currently in use.
For millions of COVID-19 survivors, virus symptoms are persisting long after their initial illness has come to an end (so-called long COVID). These people will likely require long-term treatment and new therapies may have to be pioneered to support them back to health or help them to manage a life-long chronic condition. Only time will tell.
No time to relax
With all of this in mind, it is imperative that the pharma industry doesn’t rest on its laurels. Drug developers need to continue to build on the work they have done during the past year, such as identifying effective treatments for patients hospitalised with COVID-19 and pioneering this first generation of vaccines.
The industry needs to work with healthcare providers (HCPs) to learn from current patient outcomes and identify how drugs can be improved or where there are treatment gaps that need to be addressed. They also need to be able to demonstrate globally the value of novel treatments that have been shown to be effective in some markets.
None of this can be achieved through clinical trials alone. The limited pool of individuals, combined with the restrictions of test conditions, means that we cannot gain a complete picture of the performance of vaccines and other therapies. To overcome these issues, it is vital that trial data is supplemented with the gathering and analysis of real-world evidence (RWE).
Understanding RWE
However rigorous the clinical trial, its goal is to evaluate the efficacy of the therapy: how well does the vaccine reduce disease transmission under ideal lab-like conditions? By contrast, the answer to how well a COVID-19 treatment or vaccine works in non-lab conditions (effectiveness) will come from real-world evidence, based on post-launch/real-world data (RWD).
According to the FDA’s Framework for RWE Program, RWE is defined as “data regarding the usage, or the potential benefits or risks, of a drug derived from sources other than traditional clinical trials.”2 This essentially means that RWE provides the clinical evidence about the usage and potential benefits or risks of a medical product derived from the analysis of RWD.
The potential of RWE
Randomised clinical trials have a crucial and necessary part to play in determining whether a new treatment works and whether it is safe. However, clinical trials have narrow inclusion criteria, which often exclude patients under concomitant treatments, with comorbidities or organ dysfunctions, or who don’t match an age profile.
The purpose of these restrictions is to reduce confounding factors and to produce data that is applicable to the average patient.
The downside of this approach is that an important part of the population will be excluded from any study. This means that the data can only tell us so much about a therapy’s true potential.
Another issue is actual adherence to the therapy. During trials, participants tend to be more compliant with instructions. Patients at home, however, may take and manage their medications quite differently, forgetting to take their dose on time or making minor errors in dosage size.
Additionally, the disconnect between a physician’s and a patient’s perception of various aspects of treatment, such as the meaningful impact on symptoms and quality of life, are important and can’t always be addressed in a trial. All these factors can create significant differences between a trial’s findings and the actual effectiveness of a treatment.
Filling the gap between trial and real world
RWE is increasingly being used in all lifecycle stages in a wide range of medical treatments; it’s filling the knowledge gap between trial findings and effectiveness by giving drug developers access to a more holistic data set from a wider sample of the population for a longer time period.
Real world datasets include information regarding choices made between doctors and their patients, diagnoses, prescriptions and reports about treatment progress or adverse side-effects under genuine day-to-day conditions.
It can come from a wide range of sources, from medical databases kept by state healthcare systems or private HCPs to records of health insurance claims held by medical insurers. Patient-generated data from wearables or medical devices used at home can also be harnessed to generate real insight into how patients respond to treatments, whether for COVID-19 or other conditions.
How to benefit from RWE
Not only can RWE be used to identify how therapies need to be improved to maximise their effect in all target populations, it can also help to demonstrate the effectiveness of novel treatments to HCPs in new markets. It can also highlight the benefits of therapies to HCPs and encourage take-up and adoption.
However, gathering all the available data and analysing it in a meaningful way is an enormous undertaking. There is no single, convenient repository of RWE that covers all populations and demographics.
Furthermore, each country has its own privacy regulations relating to medical records, which can limit access by drug developers to data about specific target populations. These must be carefully navigated to gain the required insight.
In addition, the sheer scale of RWD, combined with the complexity of navigating different databases (each with their own layout and structure) and analysing the information contained within each one is a significant logistical challenge.
Few pharmaceutical companies have the expertise or resource to obtain RWD and study it for insight and RWE on their own.
However, there are strategic partners available who specialise in running global RWE studies on behalf of drug developers and other health organisations. They can support pharma and health companies to create customised investigations to help them understand the real-world performance of the treatments they provide.
Expert support to see the bigger picture
COVID-19 has necessarily accelerated the evolution of healthcare and the pharmaceutical space, leading to incredible advances in vaccine and drug product technology in a very short space of time.
However, there is a lot more work to be done to eliminate the grave threat posed by COVID-19 to global health services. The novelty of treatments and vaccines, as well as the emergence of new virus strains, means that we do not know for sure that they will be effective in the long-term.
However, by harnessing the power of RWE, drug developers can gain access to the comprehensive, long-term information they need to see the bigger picture when it comes to COVID-19 or, indeed, any other health threat.
Working with such strategic advisory partners, pharma companies can be confident that they have access to the data and expert analysis they need to truly understand and demonstrate their drug’s effectiveness in the real world.
References
- www.who.int/emergencies/diseases/novel-coronavirus-2019.
- www.fda.gov/media/120060/download.