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By: John Brennan

Executives from AbbVie, AstraZeneca, Bristol-Myers Squibb, Merck and Co., Johnson and Johnson, Pfizer and Sanofi were grilled on Capital Hill in February on topics ranging from drug pricing, reimbursement, rebates and patent extensions to executive compensation. Senators called for new actions to address the high cost of prescription drugs in America.

Costs to the consumer and healthcare systems are immense. The US spent 345 billion USD on prescription drugs in 2018 and growth estimates could reach 500 billion by 2025. Although not discussed at the hearing, a significant portion (15% to 20%) of the pharma revenue stream is used for pharmaceutical R and D. This investment is the lifeblood of the global pharmaceutical industry. However, discovery, development and commercialization of a NCE or biologic could cost as much as 2.0 billion USD when failures and opportunity costs are taken into consideration. Adding to the cost and risk is the complexity of modern drug development. Establishing drug safety, determining efficacy and ensuring product quality are expensive tasks that must be monitored by sponsors and regulatory agencies. Then, there is the issue of reimbursement: market access, value proposition and pricing are the domain of the commercial payers, the new stakeholder with requirements that must be addressed in all development and commercialization scenarios.

Designing and aligning the appropriate regulatory strategy for each development asset is a critical component of program success. Here are a few of the emerging topics in drug development that should be contemplated when constructing the regulatory pathway for a NCE or biologic.

“Big Data” is a term used to describe large complex historical data sets that can be extracted and analyzed using methods that are different from traditional data management procedures. One application of “big data” gaining some traction in clinical trial design is the use of historical information to create a “synthetic” control arm instead of a traditional placebo treatment. The use of synthetic controls will never replace the randomized controlled study design but the new analysis tools for complex “big data” sets could cut control groups in half or replace them altogether, especially when traditional designs become prohibitive or historical data is complete and well-characterized.

Real World Evidence (RWE) and Patient Centricity. RWE is information collected outside of a formal clinical trial. It includes electronic medical records, claims and billing data, patient and disease registries and data gathered through wearable digital devices. In a recent address to the National Academy of Sciences and in a subsequent publication, CDER’s Janet Woodcock advocated for the use of RWE as a way of collecting and using patient data in clinical trials. Woodcock noted there has been little use of RWE in drug regulatory decisions regarding drug effectiveness. A draft guidance on RWE and a framework for its use is in the works at FDA and scheduled for release in 2020. Further, regulatory agencies have been asking for information on how clinical trials can be “fit for purpose” “with the patient population. Sponsors are now checking protocol design and interventions with patients, not just investigators, KOLs and internal experts. Patient centricity will be very relevant in future regulatory dossier reviews and approvals.

The Digital Health Revolution. Ten years ago it was hard to envision that connectable biosensors, wearables, implantables, smartphone applications, artificial intelligence, remote patient monitoring and machine learning would impact data collection and enable the emergence of personalized medicine. Now, many study protocols use digital and mobile technology as an integral part of study execution.

Value Proposition and Reimbursement. The “fee for care” model used in healthcare is shifting to value-based reimbursement. While regulatory approval requires demonstration of patient safety and efficacy, payer access requires a clear demonstration of a value proposition to qualify for reimbursement. Pipeline commercial development centered on pricing, market access and payer acceptance are now built into development programs long before final investment decisions are made at the governance level. Elements of the value proposition could include differentiation over standard-of-care, price, ease of use and innovative packaging.

These are just a few of the issues that impact strategic regulatory drug development right now. There are others: biosimilars, regulatory guidance for cell and gene therapy and precision medicine applications to minimize patient variability and improve response rate. On top of all this, regulators and sponsors will have to guide new product promotion to be consistent with product labeling as drug development and regulatory approval become more complex.

EAS offers a wealth of knowledge, enabling the development of regulatory strategies that best position your products in today’s environment. For more information or to discuss your product’s challenges contact Bryan J. Coleman, Senior Director for Pharmaceuticals and Medical Devices at bcoleman@easconsultinggroup.com. We also invite you to view our industry services sheet or the pharmaceutical tab on the EAS website.

Posted in Drugs, EASeNews, Issue of the Month and tagged .