Expanded Access Expectations of the 21st Century Cures: Frequently Asked Questions

By: Jane Reese-Coulbourne, MS ChE

Frequently Asked Questions

What is the 21st Century Cures Act and when does it take effect?

The 21st Century Cures Act is a $6.3 billion legislative package designed to speed approval of medical devices and drugs, boost funding for medical research, and provide on-going efforts to better incorporate the patient’s voice into FDA’s decision-making. It is a large bill with many provisions, among them some that require specific actions from drug and device manufacturers regarding “expanded access” policies and communications and how they are to make such information publicly available. It was signed into law on December 13, 2017 and went into effect on February 11, 2017.

Why was the expanded access (EAP) language included?

The language in the bill is in response to complaints that many drug makers make it difficult for patients and their physicians to find information and sort out the process for gaining access to medicines that are not yet approved for use by the US Food and Drug Administration.

What are the EAP provisions in the 21st Century Cures Act?

The 21st Century Cures legislation requires that all manufacturers of one or more investigational drugs for the diagnosis, monitoring, or treatment of one or more serious diseases or conditions shall publicly disclose their policies for how and under what conditions they will or will not make available their investigational medical products. This is to be communicated by posting their policies on a publicly available Internet website, beginning February 11, 2017, 60 calendar days after the date of enactment of the 21st Century Cures Act.

The posted information must include:
  • contact information for the manufacturer or distributor to facilitate communication about requests
  • procedures for making requests
  • general criteria manufacturer will use to evaluate requests for individual patients and for responses to such requests
  • length of time manufacturer or distributor anticipates will be necessary to acknowledge receipt of requests
  • hyperlink or other reference to the clinical trial record containing information about expanded access
  • declaration that this info will not serve as a guarantee of access to any specific investigational drug by any individual patient and that nothing prevents a manufacturer or distributor from revising their policy at any time

Are there penalties for not complying with the EAP provisions of 21st Century Cures Act?

While 21st Century Cures Act does include penalties for non-compliance, it is unclear whether or not penalties apply to the EAP requirements.

What is likely to happen if a manufacturer does not comply with the EAP provisions of 21st Century Cures Act?

The 21st Century Cures Act has been enacted into law so manufacturers are under legal obligation to comply.  Additionally, patients and patient advocacy groups will be actively seeking this information. If it is not publicly available, they are likely to go back to Congress and the FDA to pressure them to enforce the law.


How MK&A can help

Our customized services will help ensure a timely and knowledgeable approach to pre-approval access policies and programs for your company:

1.  Policy creation and implementation
Understanding of relevant laws, regulations, policies; examination of ethical questions and dilemmas; assessment of key stakeholders’ points of view; industry best practices; current regulatory perspectives
2.  Program scoping 
Determination of appropriate goals and best approaches to early access; internal alignment workshops; ethical roundtables; tactical action plans; vetting of program partners, costs, and timelines; crisis management
3.  Feasibility analysis of pre-approval access
In-depth look at current pipeline need for early access; comprehensive examination of stakeholder interests and constraints; analysis for potential detractors; and clinical considerations, impact on ongoing clinical trials
4.  Design of pre-approval access program
Guidance on operational design of program; input from external stakeholders (e.g. patients, regulators); internal and external communications; conversion to commercial product
5.  Engagement plan
Introduction to stakeholders; advisory boards; consultations with experts; connections to regulators and policy makers
6.  Observational data collection in a pre-approval setting
Understand current thinking on uses of observational data collection in early access setting; determination what data to be collected; analysis of Real World Data (RWD) to inform understanding of experimental compound, links to regulatory decision making, use in payer decision making

About the Author:

Jane Reese-Coulbourne, MS ChE

Jane is a senior consultant at MK&A.  Jane brings comprehensive knowledge of evolving regulatory requirements and sensitivity to patients and sponsors’ needed in the conception and design of pre-approval drug access programs. She has helped shape policy and practice in this new paradigm of patient advocacy for early access to experimental treatments.

Most recently, Jane was the founding executive director of the Reagan-Udall Foundation for the FDA. Prior to that, she was an advisor to Dr. Richard Klausner, director of the National Cancer Institute. She served as executive vice president of the National Breast Cancer Coalition and as the board chair of the Lung Cancer Alliance.

Jane holds an MS in chemical engineering from the University of Virginia.

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