News and Views: Budget and Real World Evidence, Inclusion/Exclusion in Clinical Trials, Gene Therapies, Balancing Opioid Access, Information and Usage Labeling, Mobile Medical Apps Regulation, Drug Shortages Task Force – Drug and Device Digest

CaptureFDA Budget Matters: A Cross-Cutting Data Enterprise for Real World Evidence 

FDA committed to new tools to collect data from routine medical care and develop valid scientific evidence  appropriate for regulatory decision making

  • Leverage “real world data” relating to patient health status and/or the delivery of health care obtained at the point of care
  • Enable more efficient medical product development by integrating safety and benefit information from clinical care
  • Can overcome limitations of traditional randomized clinical trials with defined inclusion and exclusion criteria

FDA’s Sentinel System and the National Evaluation System for health Technology (NEST) analyzes real world data for ‘real world evidence’

  • Need to govern responsible use of data and provide timely access through creation of national resource
  • Maintain strict data security and privacy of personal information
  • $100M medical data enterprise proposal budget for modern system for electronic health records from about 10 million lives

Current initiatives

  • Post-Market Data Sources: Claims Data vs. EHRs and access to clinical medical information in de-identified electronic health records and real-time information
  • Establishing a System that can Leverage All Data Sources by full interoperability
  • Improving Clinical Trials by using real world data for efficient recruitment, integration across clinical care settings, innovative statistical approaches to reduce  size and duration
  • Investing in Tools to More Wisely Use Data to Improve Health for data standards and data quality



Evaluating Inclusion and Exclusion Criteria in Clinical Trials

Eligibility criteria define patient population under investigation

  • Inclusion criteria identify population in which it is expected that the effect of the drug can be shown
  • Exclusion criteria specify characteristics that disqualify patients from participation
  • Both exclude patient subgroups who may eventually receive drug once approved

Strategies to Support Better Development of Eligibility Criteria and Increase Enrollment

  • Improving transparency and increasing patient involvement in clinical trial design
  • Re-examining exclusion and inclusion Practices
  • Increasing the use of innovative and alternative trial designs and methods to support inclusion



FDA’s Efforts to Advance Development of Gene Therapies

For novel technologies like gene therapy, FDA tailoring regulatory path for assuring safety and efficacy

  • Six scientific guidance documents for modern, comprehensive framework
  • Clear recommendations to support innovation

Disease Specific Gene Therapy Guidances

Human Gene Therapy for Rare Diseases

Guidances on Manufacturing Gene Therapies



Balancing access to appropriate treatment for patients with chronic and end-of-life pain with need to take steps to stem misuse and abuse of opioids

New policy to strike right balance between

  • reducing new addiction rate by decreasing exposure to opioids prescribing  – and –
  • make sure that patients with pain have access to appropriate, evidence-based care
  • Mostly, opioid treatment for acute pain and prescribed for short durations
  • Patient-Focused Drug Development meeting  for additional patient viewpoints

New steps to aggressively confront the addiction epidemic

  • Revised Blueprint for opioid drug manufacturers required to make available to prescribers
  • Required training on non-opioid alternatives
  • Develop evidence-based guidelines on appropriate prescribing
  • lnnovation challenge to spur development of medical devices ‒ including digital health and diagnostics – to provide novel solutions to treating pain
  • New series of guidance documents on development of new drugs targeted to the treatment of various types of pain
  • Collaboration with NIH public-private partnership to advance pharmacological treatments for pain and addiction




Steps to encourage more informative labeling on prescription drug and biological products’ indications and usage

General Principle: To enable health care practitioners to readily identify appropriate therapies for patients by clearly communicating drug’s approved indication(s)

  • Scope of an Indication Relative to Population Studied
  • Age Groups in Indication
  • Distribution of Information Among Labeling Sections

Content and Format

  • Indication: Disease, Condition, or Manifestation Being Treated, Prevented, Mitigated, Cured, or Diagnosed
  • Other Information Necessary To Describe the Approved Indication
  • Limitations of Use : Appropriate and Inappropriate situations

Other considerations

  • Identification of Outcomes, Endpoints, and Benefit(s)
  • Accelerated Approval
  • Required or Recommended Language
  • Preferred Wording and Wording Generally To avoid



FDA Regulation of Mobile Medical Apps

Efficient regulation of mobile medical apps – Software as Medical Device (SaMD) – tailored to potential benefits and risks

  • Traditional FDA regulatory framework can stifle the development/access
  • New framework being developed to  recognize distinctive aspects of digital health technology, clinical benefit, unique user interface, and compressed commercial cycles

FDA initiatives

  • Working with International Medical Device Regulators Forum (IMDRF) on internationally harmonized regulatory framework
  • Digital Health Innovation Action Plan to implement the software provisions of the Cures Act
  • Precertification program to qualify for a more streamlined premarket review process
  • New guidance on Mobile Medical Apps

Firm-based approach based on culture of excellence and leveraging

  • Postmarket data collection
  • Clinical data from device registries, EHRs
  • Other electronic health information sources through the National Evaluation System for health Technology (NEST)



Formation of a new drug shortages task force and FDA’s efforts to advance long-term solutions to prevent shortages

New Drug Shortages Task Force – including CMS and VA

  • Understand root causes – e.g. low margins with low incentives, reimbursement issues, limited manufacturing capacity
  • Consider regulations coupled with financial incentives to market critical access drugs
  • Conduct a risk assessment and mitigation plans  to proactively address shortage
  • Emerging technology program  to prevent shortages and new quality metrics initiatives


Image credits: FDA, HHS

Scroll to Top