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

Expanded Access Expectations of the 21st Century Cures: 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.

Categories: Expanded Access Policy Pre-Approval Access Strategy Uncategorized

About The Author [ Veronica Lopez ]

Veronica Lopez, MPH, is the strategic associate at VOZ. Veronica helps VOZ develop new ways of building equitable and responsible partnerships between industry and patients, advocates, health care professionals, and policy makers.