The potential of RWE
In a recent article on PMLive, Danny Buckland, an health journalist, says: “Using RWE - derived from multiple sources across clinical, commercial and social settings - is seen as a way of improving therapeutic outcomes and opening up vistas of new pricing and reimbursement structures. Data washes around healthcare systems but often evaporates because its power has been difficult to identify and harness. We live in data-rich times. It streams from electronic health records (EHR), claims databases, registries and lifestyle software so it makes sense to use it. But its collection and context is varied with anything from idiosyncratic hospital coding to machine and human input error introducing bias into the formula. A need for caution was highlighted by the US Food and Drug Administration (FDA) in a debate primer published in the New England Journal of Medicine in December that stated: 'If we are to realise the full promise of such evidence, we must be clear about what it is and how it can be used most effectively”
Official Medicine still skeptic
Dr Antonis A Kousoulis, RWE consultant, echoes FDA concerns: “When considering the use of RWE as sources of evidence towards best practice (though), we need to be mindful that Research via Clinical Trials (RCTs) remain the gold standard. Entirely disrupting the status quo of the process of developing guidelines will still take a few decades, especially in developing countries, but with such unmet need and unequal access to care, we should not allow (what is thought to be) 'best' to become an enemy of 'good enough'. RWE could indeed help with the dissemination of key information by bridging the knowledge gap for clinicians and by improving the quantity and quality of evidence used in guidelines. This translational process is already happening. In a recent systematic review we noted that RWE from a UK electronic medical record database has been used inconsistently, but increasingly, in the last decade to inform guidelines published in the country. The increased uptake in recent years shows that this area of R&D in healthcare is changing and we are now working during a phase in this transition.To fully capitalise on the potential value of using RWE, researchers need to ensure they undertake research of translational value to the healthcare community. (..) It will take a structured and collaborative multidisciplinary approach to look to the future and overcome the barriers to making best use of RWE, namely: capacity and cost, skills and resource, and technology and access, all while maintaining public trust. Cross-sector collaboration will be the catalyst to enable global synergies and the positive shift in the reality versus expectations scale.”
Industry more optimistic
Danny Buckland interviewed also industry representatives: ”Michael Seewald, global head of real world evidence at Novartis, which has established a Centre of Excellence, believes RWE has multiple uses. “First and foremost RWE has a key role to play in post-approval settings,” he says. “Regulators are looking for ways to understand benefit-risk. Payers are increasingly challenging the value of new health technologies. RWE can help to demonstrate value vs standard of care. In addition, RWE is uniquely helpful to highlight how care is provided in real life, ie how ill patients are, which medications are provided to them, what the burden of disease is today.” “It is increasingly powerful to characterise patient populations and their outcomes,” he adds. “Clinicians recognise the value of disease registries and databases, as they supplement their strong clinical knowledge with data from routine clinical practice across various geographies. We do see significant value in RWD informing the design of clinical trials, portfolio management of R&D projects and also helping to improve the operational aspects of clinical development”. Novartis has used RWE extensively across its oncology portfolio and Seewald feels that improved data collection should help industry become stronger and improve outcomes for patients. Catherine Reed, of global patient outcomes and real world evidence at the Lilly Research Centre UK, views Europe as a key player in developing RWE. Lilly, which has spent around $3bn on researching Alzheimer's treatment, believes its public-private partnership in an Innovative Medicines Initiative project, led by the University of Oxford, could unlock key factors in combatting the condition. The ROADMAP project is interrogating RWE across Europe with the aim of creating a better ethical, legal and social framework. One of its aims is to 'identify RWE in Alzheimer's to better inform the future regulatory environment - for regulators on matters of efficacy and safety and medicines; for medicines manufacturers on building a sustainable payer environment; and for providers on cost-effectiveness'. Reed sums up the potential: “With the help of real-world evidence, the entire population becomes the decision-making laboratory.” .”Then it appears that the journey of AI could be especially helpful for health care facilities that can't afford a Dr. House”, says Venkat Rajan, who has the great title of global director for the company's Visionary Healthcare Program; places that have, for example, a general cardiologist rather than a team with different subspecialties. This will "democratize" diagnosis and care, he says. Computer-aided diagnosis can weigh more factors than a doctor could on their own, such as reviewing all of a patient's history in an instant and weighing risk factors such as age, previous diseases, and residence (if it's in a heavily polluted area) to come up with a short list of possible diagnoses, even a percent confidence rating that it's disease X or syndrome Y. Much of this involves processing what's called "unstructured data" such as notes from previous exams, scan images or photos. This new era will allow patients, similarly to the introductory example, to have his/her personalized approach with the right medicine and the best treatment”.
Patients (crowd)sourcing: an alternative?
Reports Anthill, in its website, that in the recent Web Summit Lisbon Crowdsourcing is now established in life sciences, as demonstrated by the quality and ingenuity of the new medical devices and technologies at the Patient Innovation Awards. So while experts and companies are debating about how to use the richness of data already available patients, care-givers and inventors have crowdsourced an amazing set of relatively simple but well-designed solutions to problems that have not been addressed directly by medical devices companies.” Can Patient(crowd) sourcing become the accelerator of data use? A patient full health history could be in an electronic format given to the patient who then can decide to participate directly in crowdsource programs that contribute to a better understanding of his/her disease. If we do not democratize the system “entirely disrupting the status quo of the process of developing guidelines Dr Antonis A Kousoulis, states will still take a few decades”....Patients may not be willing to wait that long.
