Each year, we designate the last day in February as Rare Disease Day to raise awareness about the potential impact we can make for this important segment of the world’s population. From policy makers to researchers to health authorities, we all play a part in making a difference for more than an estimated 300 million people in the world suffering from a rare disease.
To mark the event, I would like to reflect on my role to move the needle and make a difference to patients that are awaiting life-changing treatment. My journey started as a clinician where I had the privilege in caring for patients with a variety of rare diseases. At a personal level, I am also the aunt of a very special boy who suffered for 8 years with a rare cardiopulmonary disease. To me, rare diseases are not so rare. Continue reading →
Several European countries have been utilizing big data together with randomized control trials to guide healthcare decision making and to address market access issues. The Region Europe RWE Advisory Board was formulated to explore potential areas of real world evidence (RWE) use in countries like UK, France, Portugal etc. The board engages stakeholders including payers, providers, and patient organizations with the intent of developing a platform and widening the community for RWE. The board has developed a three-year road map of initiatives for the enhanced use of RWE. The contributors are spread across the areas of licensing, commissioning and access, clinical decision-making and patient outcome decision-making to prioritize four key action points.
In our June 2017 blog post, we described advantages and challenges of using syngeneic, GEM, and humanized mouse models for preclinical immuno-oncology (I/O) drug development. In this blog, we expand on this idea and offer thoughts on choosing the most appropriate I/O tumor model for one’s study. While there are benefits and limitations of any model, one can use these considerations, as well as others, as a foundation for preclinical in vivo efficacy study design. Understanding tumor placement, immune composition, response to treatment, and molecular characterization for the model of interest can be invaluable when designing the most appropriate study for your research goals.
Real World Evidence (RWE) helps payers make reimbursement decisions and assists patients in budget management and estimating risk benefit of interventions. The integration of RWE in value assessment frameworks, used for ascertaining drug value, can help payers make informed evidence-based reimbursement decisions. RWE provides insight into real world impact of an intervention; facilitating “performance-based risk-sharing arrangements” between payers and manufacturers. United Health and Merck have collaborated to link payments to drug performance by utilizing RWE. Similarly, Amgen and Humana have partnered to target chronic diseases by generating RWE.
Patient engagement is another core component for RWE generation as well as its dissemination. Prescription Drug User Fee Act (PDUFA) re-authorization and the 21st Century Cures Act emphasize the use of patient data for drug development. Patients can make better-informed health decisions based on information from Patient-Centered Outcomes Research Institute, National Health Council, virtual networks and digital technology like mHealth. Johnson & Johnson collaborated with Aetna to use wearable technology in atrial fibrillation screening for RWE generation. Health PROMISE, a patient reported outcome tool integrated with EHRs, helped IBD patients and providers to visualize real time data; thus improving their quality of life.
Conclusion Real World Evidence can help payers and patients assess cost-benefit analyses of an intervention through real world patient data. Innovative use of technology can show the way forward in enhancing patient engagement and creating opportunities to access, integrate, analyze and effectively utilize RWE.
When a patient reads the label on their medicine bottle, he or she naturally relies on the medicine to contain the correct drug, be safe, work as intended and list the correct dosage. The pharmaceutical companies that produce these medicines similarly must rely on their internal manufacturing processes and quality control testing to generate the medicine responsible for this patient trust.
For the development of biologic medicines, the process of generating a quality product is less straightforward than that of a small-molecule medicine, like pain relievers such as aspirin. Selecting the right partner, such as Covance and its ‘Central GMP Testing Laboratory’ model, can smooth the path to validation and consistent manufacturing quality for your biologic. Continue reading →
Randomized control trials demonstrate drug safety and efficacy and hence are fundamental for regulatory approval. However, real world data (RWD) becomes a progressively more important source of evidence as we move across the development continuum of a drug. RWD can be potentially useful for obtaining regulatory approval as well. Communicating the value of a health technology is critical for supporting the market access of a drug. Favourable comparative clinical profiles and cost effectiveness can influence the position of a drug in the formulary of a payer.
The 21st Century Cures Act (Section 3037) amended the US FDA Modernization Act 114, directing real world evidence (RWE) utilization to support recommendation of a new indication for an approved drug. This allowed manufacturers to communicate wider scope of a product’s value such as comparative patient reported outcome data. The change broadened the audience covered and helped to expedite market access. The FDA has also acknowledged the importance of RWE for closing evidence gaps while making healthcare decisions. Avelumab was granted accelerated approval due to supportive data from an observational study that described poor outcomes in Merkel Cell carcinoma patients treated by cytotoxic chemotherapies.
Conclusion Real World Evidence obtained during pre and post phases of drug development provides an opportunity to accelerate market access and facilitate quicker approvals. RWE can be potentially utilized by regulatory authorities to close the evidence gap associated with real-world effectiveness.
Most sponsors are well aware of the recent revision (R2) of ICH GCP E6, which outlines recommendations to unify standards across the EU, Japan and the U.S. with defined requirements for sponsors regarding the role of monitoring and risk management, there are many factors to consider for a successful implementation.
This article discusses a variety of challenges sponsors may face when preparing to address these recommendations and also examines opportunities to drive greater efficiencies in today’s complex trial ecosystem.
Addressing varying levels of adoption
According to the latest revision, sponsors are tasked with a requirement to employ an adaptable quality management system and are expected to maintain oversight of CROs. But with various tracking systems, vendor logs, CTMS and monitoring platforms, seeing the complete picture can be an overwhelming, error-prone effort. Continue reading →