United States: House Committee Considers 21st Century Cures Draft, Senate Coordinates Sister Bill

In January 2015, the House Energy and Commerce Committee released a discussion draft of the 21st Century Cures Act, a bipartisan bill aimed at overhauling how FDA regulates drugs and devices, which builds on an effort known as the 21st Century Cures Initiative. The initiative launched in April 2014 and has been the subject of many congressional hearings and roundtables, as well as several white papers.

Corresponding to the release of the 21st Century Cures discussion draft, two top Republicans from the Senate Health, Education, Labor and Pensions ("HELP") Committee released "Innovation for Healthier Americans," a report aimed at "identifying and advancing specific steps to better align public policy to support medical innovation and patient access to new medicines and technologies," focusing primarily on ways to improve FDA and the National Institutes of Health ("NIH"). The report, which calls for stakeholder feedback by February 23, 2015, was a precursor to a February 3, 2015 announcement from Senators Lamar Alexander (R-TN) and Patty Murray (D-WA), the Chairman and Ranking Member of the Senate HELP Committee, that they are launching a bipartisan initiative to examine the process for providing safe treatments, devices, and cures to patients and the roles of the FDA and NIH in that process. Similar to the work done on the 21st Century Cures Initiative, the senators are creating a bipartisan working group and plan to hold a series of hearings. Any resulting legislation will likely complement the House's intended 21st Century Cures Act.

The lengthy and long-anticipated 21st Century Cures Act discussion draft addresses regulation of drugs and biologics with an eye toward speeding up approvals (Title I); enhancing discovery and development of new treatments (Title II); changes to the clinical trial process (Title III); reforms for NIH and FDA related to research, hiring, and succession planning (Title IV); and revamping certain aspects of medical device regulation (Title V). A summary of each title is provided below.

Title I. Title I would require FDA to include patient experience data when assessing the risks and benefits of certain therapies and to issue guidance describing the use of surrogate endpoints in the drug-approval process. In addition, it would permit FDA to give early approval to breakthrough therapies and provide a new pathway for approving antibiotics based on the limited and specific patient populations who need them. Another provision of this title seeks to ease patient access to drugs outside of approved clinical trials, commonly referred to as "compassionate use." In order to accelerate new cures for high-need populations, the bill would provide funds to repurpose already-approved drugs for new indications and "streamline" data review for such applications. Title I would also expand exclusivity for several categories of drugs, including new antibiotics, orphan drugs, and certain new drug products if they have an added patient or public health benefit over a previously approved version of the same drug. Perhaps most controversial, one discussion draft proposal would provide up to 15 years of marketing exclusivity for drugs that petition for and receive designation from the Department of Health and Human Services ("HHS") as a "dormant therapy."

Title II. Title II of the draft bill would create an "Innovative Cures Consortium," broader than, but based on, the EU's Innovative Medicines Initiative, to pool the resources of industry, government, and academia to accelerate cure development and delivery by nongovernment entities. Under this title, FDA would be required to issue guidance on accelerating certain therapies, including regenerative medicine and genetic technologies for rare diseases. Title II also proposes definitions for "health software" and related terms that would be incorporated into the Federal Food, Drug, and Cosmetic Act ("FDCA"), which does not currently define these terms. The discussion draft also contains a notation indicating that the drafters are struggling with how to ensure that the definition does not inadvertently exempt products that should be regulated. Data sharing is another key aspect of the draft bill. Title II would require increased data sharing from entities receiving federal funding (through federally funded health insurance programs or NIH grants, for example) and would establish a data-sharing framework to enhance patient treatment and care. The proposed draft legislation would also call for FDA to issue guidance on the coordination of regulatory activities for combination products. The discussion draft contains placeholders for subtitles that would address regulation of diagnostic devices and health care data interoperability, but no further details are available yet.

Title III. Title III focuses on clinical trials and the challenges of balancing the protection of human subjects and resource efficiency for those conducting the trials. One way the bill addresses these challenges is by encouraging the use of adaptive clinical trials, which use prospectively designed benchmarks as decision points to make study design changes. The draft bill would require HHS to provide assistance to any federal agency trying to improve oversight of human subject research, particularly with regard to vulnerable populations. Provisions in this title would also provide a mechanism for FDA to periodically evaluate post-approval studies, taking into account any recent changes in medical practice. Finally, Title III would establish a global pediatric clinical trial network to engage with foreign regulatory entities and share knowledge on pediatric research.

Title IV. Title IV puts a greater focus on the various agencies involved in the discovery, development, and delivery of therapeutic products. Title IV contains proposals to increase research funding for and decrease the administrative burdens on NIH. The discussion draft seeks to accelerate vaccine approval by proposing a 180-day standard timeline and an expedited review process for the Centers for Disease Control and Prevention ("CDC") to evaluate vaccines. Title IV also contains significant proposals related to FDA. It would create a process for FDA to designate eligible infectious diseases for priority review vouchers at least every five years and calls on FDA to issue final guidance on changes to approved biologics. The discussion draft also includes a succession-planning provision addressing how FDA should handle retirements of critical personnel and would promote professional development throughout FDA. The discussion draft also proposes revisions to the Sunshine Act, namely, exempting certain industry-sponsored educational activities from reporting requirements. A provision addressing telemedicine would improve the delivery of health care to Medicare beneficiaries. Title IV contains several other provisions relating to Medicare, which is not the focus of this publication. Finally, this title proposes to ease restrictions on experimental medical devices, subject to listing and informed consent requirements, among others.

Title V. Title V concentrates primarily on medical devices but also contains provisions related to drugs. Certain drugs and biosimilars designated as "American-Manufactured" would be granted an exclusivity extension of undetermined length. Title V would also require FDA to update its Good Manufacturing Practices guidance for drug products. Concerning medical devices, the discussion draft would extend FDA's use of third-party device reviews to two new areas: facility inspections and the assessment and certification of "a facility's capability to evaluate and implement" modifications to approved or cleared devices. Title V also creates premarket notification requirements for Class I devices, relaxes requirements for predicate device indications and humanitarian device exemptions, proposes changes to the classification review process, and sets out supply chain security measures for medical devices similar to those enacted for drugs in 2013. Finally, the draft bill would define "valid scientific evidence" in the medical device context, would require FDA to decide whether to recognize medical device standards developed by outside organizations within 60 days of publication, and would direct FDA to train medical device regulators to use the "least burdensome approach," including publishing updated guidance on the subject.

Although the discussion draft from the 21st Century Cures Initiative is not a formal legislative proposal, the 350-page document has the appearance of a bill in most respects, save a few placeholders and notes. Going forward, the committee will collect comments on the draft and will likely take up the legislation later this year.

The content of this article is intended to provide a general guide to the subject matter. Specialist advice should be sought about your specific circumstances.

To print this article, all you need is to be registered on Mondaq.com.

Click to Login as an existing user or Register so you can print this article.

Colleen Heisey
Christian Fulda
Similar Articles
Relevancy Powered by MondaqAI
In association with
Related Topics
Similar Articles
Relevancy Powered by MondaqAI
Related Articles
Related Video
Up-coming Events Search
Font Size:
Mondaq on Twitter
Register for Access and our Free Biweekly Alert for
This service is completely free. Access 250,000 archived articles from 100+ countries and get a personalised email twice a week covering developments (and yes, our lawyers like to think you’ve read our Disclaimer).
Email Address
Company Name
Confirm Password
Mondaq Topics -- Select your Interests
 Law Performance
 Law Practice
 Media & IT
 Real Estate
 Wealth Mgt
Asia Pacific
European Union
Latin America
Middle East
United States
Worldwide Updates
Registration (you must scroll down to set your data preferences)

Mondaq Ltd requires you to register and provide information that personally identifies you, including your content preferences, for three primary purposes (full details of Mondaq’s use of your personal data can be found in our Privacy and Cookies Notice):

  • To allow you to personalize the Mondaq websites you are visiting to show content ("Content") relevant to your interests.
  • To enable features such as password reminder, news alerts, email a colleague, and linking from Mondaq (and its affiliate sites) to your website.
  • To produce demographic feedback for our content providers ("Contributors") who contribute Content for free for your use.

Mondaq hopes that our registered users will support us in maintaining our free to view business model by consenting to our use of your personal data as described below.

Mondaq has a "free to view" business model. Our services are paid for by Contributors in exchange for Mondaq providing them with access to information about who accesses their content. Once personal data is transferred to our Contributors they become a data controller of this personal data. They use it to measure the response that their articles are receiving, as a form of market research. They may also use it to provide Mondaq users with information about their products and services.

Details of each Contributor to which your personal data will be transferred is clearly stated within the Content that you access. For full details of how this Contributor will use your personal data, you should review the Contributor’s own Privacy Notice.

Please indicate your preference below:

Yes, I am happy to support Mondaq in maintaining its free to view business model by agreeing to allow Mondaq to share my personal data with Contributors whose Content I access
No, I do not want Mondaq to share my personal data with Contributors

Also please let us know whether you are happy to receive communications promoting products and services offered by Mondaq:

Yes, I am happy to received promotional communications from Mondaq
No, please do not send me promotional communications from Mondaq
Terms & Conditions

Mondaq.com (the Website) is owned and managed by Mondaq Ltd (Mondaq). Mondaq grants you a non-exclusive, revocable licence to access the Website and associated services, such as the Mondaq News Alerts (Services), subject to and in consideration of your compliance with the following terms and conditions of use (Terms). Your use of the Website and/or Services constitutes your agreement to the Terms. Mondaq may terminate your use of the Website and Services if you are in breach of these Terms or if Mondaq decides to terminate the licence granted hereunder for any reason whatsoever.

Use of www.mondaq.com

To Use Mondaq.com you must be: eighteen (18) years old or over; legally capable of entering into binding contracts; and not in any way prohibited by the applicable law to enter into these Terms in the jurisdiction which you are currently located.

You may use the Website as an unregistered user, however, you are required to register as a user if you wish to read the full text of the Content or to receive the Services.

You may not modify, publish, transmit, transfer or sell, reproduce, create derivative works from, distribute, perform, link, display, or in any way exploit any of the Content, in whole or in part, except as expressly permitted in these Terms or with the prior written consent of Mondaq. You may not use electronic or other means to extract details or information from the Content. Nor shall you extract information about users or Contributors in order to offer them any services or products.

In your use of the Website and/or Services you shall: comply with all applicable laws, regulations, directives and legislations which apply to your Use of the Website and/or Services in whatever country you are physically located including without limitation any and all consumer law, export control laws and regulations; provide to us true, correct and accurate information and promptly inform us in the event that any information that you have provided to us changes or becomes inaccurate; notify Mondaq immediately of any circumstances where you have reason to believe that any Intellectual Property Rights or any other rights of any third party may have been infringed; co-operate with reasonable security or other checks or requests for information made by Mondaq from time to time; and at all times be fully liable for the breach of any of these Terms by a third party using your login details to access the Website and/or Services

however, you shall not: do anything likely to impair, interfere with or damage or cause harm or distress to any persons, or the network; do anything that will infringe any Intellectual Property Rights or other rights of Mondaq or any third party; or use the Website, Services and/or Content otherwise than in accordance with these Terms; use any trade marks or service marks of Mondaq or the Contributors, or do anything which may be seen to take unfair advantage of the reputation and goodwill of Mondaq or the Contributors, or the Website, Services and/or Content.

Mondaq reserves the right, in its sole discretion, to take any action that it deems necessary and appropriate in the event it considers that there is a breach or threatened breach of the Terms.

Mondaq’s Rights and Obligations

Unless otherwise expressly set out to the contrary, nothing in these Terms shall serve to transfer from Mondaq to you, any Intellectual Property Rights owned by and/or licensed to Mondaq and all rights, title and interest in and to such Intellectual Property Rights will remain exclusively with Mondaq and/or its licensors.

Mondaq shall use its reasonable endeavours to make the Website and Services available to you at all times, but we cannot guarantee an uninterrupted and fault free service.

Mondaq reserves the right to make changes to the services and/or the Website or part thereof, from time to time, and we may add, remove, modify and/or vary any elements of features and functionalities of the Website or the services.

Mondaq also reserves the right from time to time to monitor your Use of the Website and/or services.


The Content is general information only. It is not intended to constitute legal advice or seek to be the complete and comprehensive statement of the law, nor is it intended to address your specific requirements or provide advice on which reliance should be placed. Mondaq and/or its Contributors and other suppliers make no representations about the suitability of the information contained in the Content for any purpose. All Content provided "as is" without warranty of any kind. Mondaq and/or its Contributors and other suppliers hereby exclude and disclaim all representations, warranties or guarantees with regard to the Content, including all implied warranties and conditions of merchantability, fitness for a particular purpose, title and non-infringement. To the maximum extent permitted by law, Mondaq expressly excludes all representations, warranties, obligations, and liabilities arising out of or in connection with all Content. In no event shall Mondaq and/or its respective suppliers be liable for any special, indirect or consequential damages or any damages whatsoever resulting from loss of use, data or profits, whether in an action of contract, negligence or other tortious action, arising out of or in connection with the use of the Content or performance of Mondaq’s Services.


Mondaq may alter or amend these Terms by amending them on the Website. By continuing to Use the Services and/or the Website after such amendment, you will be deemed to have accepted any amendment to these Terms.

These Terms shall be governed by and construed in accordance with the laws of England and Wales and you irrevocably submit to the exclusive jurisdiction of the courts of England and Wales to settle any dispute which may arise out of or in connection with these Terms. If you live outside the United Kingdom, English law shall apply only to the extent that English law shall not deprive you of any legal protection accorded in accordance with the law of the place where you are habitually resident ("Local Law"). In the event English law deprives you of any legal protection which is accorded to you under Local Law, then these terms shall be governed by Local Law and any dispute or claim arising out of or in connection with these Terms shall be subject to the non-exclusive jurisdiction of the courts where you are habitually resident.

You may print and keep a copy of these Terms, which form the entire agreement between you and Mondaq and supersede any other communications or advertising in respect of the Service and/or the Website.

No delay in exercising or non-exercise by you and/or Mondaq of any of its rights under or in connection with these Terms shall operate as a waiver or release of each of your or Mondaq’s right. Rather, any such waiver or release must be specifically granted in writing signed by the party granting it.

If any part of these Terms is held unenforceable, that part shall be enforced to the maximum extent permissible so as to give effect to the intent of the parties, and the Terms shall continue in full force and effect.

Mondaq shall not incur any liability to you on account of any loss or damage resulting from any delay or failure to perform all or any part of these Terms if such delay or failure is caused, in whole or in part, by events, occurrences, or causes beyond the control of Mondaq. Such events, occurrences or causes will include, without limitation, acts of God, strikes, lockouts, server and network failure, riots, acts of war, earthquakes, fire and explosions.

By clicking Register you state you have read and agree to our Terms and Conditions