Regulatory Radar: 21st Century Cures Act, Subtitle D − Patient Access to Therapies and Information

Facilitating access to investigational new drugs and adding new label indications

July 15, 2017

The 21st Century Cures Act under Section 3032 seeks to facilitate expanded access of patients to investigational new drugs. This type of expanded access is also referred to as compassionate use, and in this context means the ability of an individual patient to try an investigational new drug outside a clinical trial prior to drug approval. It differs from the “right-to-try” laws recently enacted by over 37 states that allow for patient access to investigational drugs without obtaining FDA approval.

Under the current FDA regulations a patient may seek “compassionate use” individual access to investigational products for a serious disease or condition if the following conditions are met:

  • The patient and a licensed physician are both willing to participate.
  • The patient’s physician determines that there is no comparable or satisfactory therapy available to diagnose, monitor or treat the patient’s disease or condition.
  • The probable risk to the person from the investigational product is not greater than the probable risk from the disease or condition.
  • FDA determines that there is sufficient evidence of the safety and effectiveness of the investigational product to support its use in the particular circumstance.
  • FDA determines that providing the investigational product will not interfere with the initiation, conduct or completion of clinical investigations to support marketing approval.
  • The sponsor or the clinical investigator (or the patient’s physician in the case of a single patient expanded access request) submits a clinical protocol that is consistent with FDA’s IND/IDE regulations.
  • The patient is unable to obtain the investigational drug under another IND or to participate in a clinical trial.

Section 3032 of the Cures Act aims to make it easier for patients to understand a drug manufacturer’s policies regarding availability of its investigational new drugs for compassionate use and how to request access. Specifically, the Cures Act requires pharmaceutical companies or distributors to have publicly-accessible compassionate use policies for drugs treating serious or life-threatening conditions available as of February 2017 or with first initiation of Phase 2 or 3 studies. It requires that the policies covering access to a company’s investigational drugs are made readily available, e.g. through websites. The information should include contact information, information on how to make a request, criteria a manufacturer or distributor uses to evaluate a request, how long it will take to acknowledge a request and a link to or other reference to the clinical trial record containing information about the expanded access for such drugs.

Importantly, the provisions in the Cures Act for companies to publicize compassionate use policies are not a guarantee of a patient having access to a specific investigational product. The pharmaceutical company must agree to provide the investigational drug for expanded access use. Also, FDA cannot require a company to provide an investigational drug for expanded access use to proceed. A company may decide to turn down a request if, for example, it is not able or willing to provide access to an investigational drug outside of clinical trials intended to support marketing approval. In some cases, patients may have to pay for using the investigational drug and/or for medical care associated with its use. In other cases, pharmaceutical companies may elect not to charge. Also under the act, a company has the right to change its policies at any time and the act does not contain any provisions regarding charges/payments for the investigational drug.

As of March 2017, approximately 50 percent of drug companies post their compassionate use policies publicly, with the majority of large pharmaceutical companies adhering to the new requirement.

In another effort to reduce review efforts and make drugs available earlier for new indications, Section 3031 of the Cures Act allows FDA to rely upon qualified data summaries to support the approval of an sNDA or sBLA application for a new indication for those drugs that have a large established safety database and with a high benefit-to-risk profile. Qualified data summaries under the act are defined as a summary of clinical data that demonstrates the safety and effectiveness of a drug with respect to a qualified indication. Qualified indication meaning an indication that FDA determines to be appropriate for summary level review. Review of the supplemental application under this path would require that existing data is available and acceptable to the Secretary demonstrating the safety of the drug and that all data used to develop the qualified data summaries are submitted to the Secretary as part of the supplemental application.

This process was successfully piloted in 2015 by FDA prior to the 21st Century Cures Act with well-known oncology drugs. The intent of this provision is to reduce time and agency resources by potentially limiting the extent of re-analysis needed of the primary data sets contained in these supplemental applications and by approving it based on the qualified data summary. The initial application of this provision is expected to be for oncology products where the Oncology Center of Excellence has been tasked to develop policies and procedures for implementation of summary-level review.

The Cures Act also requires HHS to annually post the number of applications reviewed using this path on an FDA website. This website needs to contain information on the time needed to complete the review versus average time needed for review of supplemental applications not covered under this section, and the number of applications that required full data sets in addition to the data summaries.

Please see the FDA’s website for additional information on expanded access and how to request it.

Do you have any questions? Please reach out to Marinka Tellier, NSF’s Director of Regulatory Affairs for Pharma Biotech, at or Andy Papas, NSF’s VP of Regulatory Affairs for Pharma Biotech, at, or visit our website to learn more about NSF's pharma biotech services.

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