“In God we trust, all others bring data.” -W. Edwards Deming
Recently I was a panelist at a medical society meeting where the medical specialists in the audience were expressing concern about being left out of certain provider networks and requests for certain procedures were being denied. After some prodding, with questions such as: “How many re-admissions did you have in the past year within 30 days of discharge?” and “How satisfied were your patients with the care you provided?” and “Which prescription medication provided the best outcome for your patients?,” it became apparent that they didn’t have access to some basic data about their patients and the outcomes of their care. The current fee-for-service reimbursement scheme does not provide a business imperative to encourage providers to share or collect patient level health information, or Real World Data (RWD), across various settings of care. But physicians should be encouraged to use RWD to optimize their practices, evolving as biopharmaceutical companies have in their understanding of its importance. Perhaps these two health care industry participants can join forces to address barriers to access to RWD.
Just as providers using RWD will pave the way for wiser decisions in patient care, the biopharmaceutical industry can apply it to future research and development investment and clinical trial design and recruitment. We also know that we can use RWD to better understand the value of our products in a real world setting and do a better job of monitoring the safety and efficacy of products post regulatory approval. One day RWD may complement randomized clinical trial data in the regulatory approval processes. There is vast potential for us to enable biopharmaceutical innovation through the appropriate use of RWD. Our predicament is that we do not have access to such information due to several factors including laws that prohibit access to government-funded databases for commercial entities. Such policies bar pharmaceutical companies access and harm biopharmaceutical innovation which ultimately harms the patient who is waiting for the next cure for his or her deadly disease.
A major access impediment to real world claims data exists at the federal level. CMS rules have barred parties with “commercial purposes” from directly acquiring Medicare data. This includes pharmaceutical companies as well as device manufactures and other for profit entities. Most recently, several groups including The Healthcare Leadership Council (HLC) submitted comments to CMS arguing that the ability of such organizations to attain RWD data will help improve the quality and efficiency of the healthcare system. There is no question that such information will ultimately help patients gain appropriate access to innovative medicines.
The outstanding question is how do we fix the system for providers and the biopharmaceutical industry to allow such access? Currently, several states are funding the development of the All Payer Claims Databases (APCDs). This is an effort to bring together data from various payers into a single setting as a way to capture utilization information. Such data can be a starting point to better understanding the cost/benefit of a biopharmaceutical product in a real world setting. Thus it will be important for the states to develop policies that will allow pharmaceutical companies access to this data in a manner that preserves provider and patient anonymity.
As for physicians, on par with purchasing the latest medical devices like a new echocardiogram unit, providers need to prioritize data assimilation, analysis and utilization as critical components of managing and delivering healthcare. Policymakers need to establish and implement interoperability standards while rewarding this approach with a payment and delivery system that pays for value and quality rather than volume.
Moving forward, there need to be rational policies for all parties that pave the way for optimal use of RWD in healthcare. For instance, any organization using RWD to make or influence healthcare decisions should follow transparent data selection and analysis practices including validation of results in multiple datasets. The RWD used for comparative effectiveness and practice guideline development ought to be guided by defined standards such as transparency in methodology used in the analysis of the database. In addition, studies utilizing RWD should be publicly registered like a clinical trial if used for healthcare decision making. Most importantly, we need to ensure clear guidelines that protect patient and provider privacy.
Finally, an important issue not to be overlooked is how to capture and assimilate patient experience data. This is a key element to understanding the impact of medical interventions on patients’ quality of life and satisfaction. Recently, the Patient-Centered Outcomes Research Institute (PCORI) has provided support to select patient organizations to develop databases and systems that capture such data. One example of an organization which received PCORI support is the Global Healthy Living Foundation (GHLF). Unlike CMS, PCORI does encourage the recipients of the grants to partner with the pharmaceutical industry to ensure data will help bring new innovative therapies to patients in need.
Ultimately, access and use of RWD not only will pave the way for physicians and biopharmaceutical companies to improve delivery of care and achieve a better understanding of patient outcomes in a real world setting but it will allow innovators to come up with novel breakthrough therapies that will continue to extend human life and improve quality of life. Working together, we can make that a reality.