Biomedical Innovation and Evidence Development

Scientific advances in basic disease and systems biology, genomics, proteomics, metabolomics, and immunology are driving a robust drug and therapeutic development pipeline that increasingly includes targeted, more effective and even curative specialty therapies, as well as more advanced medical devices. In 2015 FDA approved 45 novel drugs, 36 percent of which are first-in-class. At the same time, biomedical Innovation is challenged by many of the same forces driving the need to reassess delivery and payment models, including the high prices of many new therapies.

Still, unmet medical need remains. Many chronic diseases and acute conditions lack adequate therapies and cures, forcing policy makers to grapple with practical approaches for encouraging innovation and development while facilitating access to leading-edge medical technologies in ways that maximize value and outcomes. Better approaches are needed for encouraging investment in areas of unmet need, reducing the time and cost of medical product development, and supporting high value evidence-based uses of drugs, devices, and other technologies.

Our Approach

Duke-Margolis is creating an evidence and policy hub aimed at developing policies and approaches to support cost-effective continuous learning to improve safe, high-value uses of drugs, devices, and other technologies. Through collaborations with patients, providers, regulators, payers, and the pharmaceutical and medical device industry, the biomedical evidence and policy hub will support improved decision-making across the healthcare system. The primary focus of this work will be on increasing the value of biomedical innovation to patients – including both better health outcomes and lower overall health care costs.

Our work in this area also explores approaches to modernize the drug development process and advance the adoption of new tools for regulatory decision-making. Policy solutions are needed to gain ground in implementing drug development tools such as biomarkers and clinical outcome assessments, and to adopt methods incorporating the voice of the patient in both drug development and regulatory decision-making. development and evaluation of new payment models and contracting approaches related to innovative biomedical projects that support better outcomes and higher value care to patients.

Current directions include:

  • Economic incentives for addressing antimicrobial resistance
  • Advance a value assessment framework
  • Regulatory and payment uses of real world evidence
  • Utilizing electronic health information to address critical questions about product safety and effectiveness
  • Improving biomarker and surrogate endpoint development and accelerating drug development
  • Innovative solutions to deter opioid abuse
  • Improving regulatory approaches involving medical technologies, such as risk evaluation and mitigation strategies (REMS) and the regulation of “off-label” promotion

In the future, the Center will seek opportunities to evaluate and inform Medicare Part B drug payment reform, explore and pilot value-based payment models, generate evidence for new payment models for curative treatments and to inform regulatory guidance and new approaches to medical product evaluation. One particular focus will be on exploring data and methods that better meet clinician and patient information needs and to improve approaches for collecting patient preferences during drug development.

Our Projects

8th Annual Sentinel Initiative Public Workshop

Supporting the Planning Board for the National Evaluation System for Health Technology (NEST)

Supporting Innovative Sources of Real-World Data (RWD) for Medical Devices

Standardizing and Integrating REMS into the Healthcare System

Economic Incentives for Antimicrobial Innovation

Mapping Global Health Research & Development

Second Panel on Cost-Effectiveness in Health and Medicine

Value-Based Payment for Medical Products