United States: The Cure For The Common FDA Reform Legislation? House Committee Releases 21st Century Cures Discussion Draft

On January 27, 2015, the House Energy & Commerce Committee ("E&C Committee") released a long-awaited discussion draft of comprehensive medical innovation reform legislation as part of its 21st Century Cures Initiative. Launched in April 2014 by E&C Committee Chairman Fred Upton (R-MI) and Rep. Diana DeGette (D-CO), the 21st Century Cures Initiative has sought ideas for legislative proposals to accelerate the pace of medical innovation in the United States through a series of committee hearings, roundtables held around the country, and white papers. According to supporting materials released by the E&C Committee, the key goals of the proposals in the discussion draft are to (1) incorporate patient perspectives into the development and approval processes for drugs and medical devices and help address patients' unmet medical needs; (2) build the foundation for 21st century medicine; (3) streamline clinical trials; (4) support continued innovation at our federal public health agencies; and (5) modernize medical product regulation.

Two days after the release of the E&C Committee's discussion draft, Senators Lamar Alexander (R-TN) and Richard Burr (R-NC) of the Senate Committee on Health, Education, Labor & Pensions released a report focused on identifying opportunities for meaningful legislative reforms to medical product discovery and development. The Senate report is intended as a parallel and complementary effort to the draft legislation under the 21st Century Cures Initiative, and it likewise focuses on the need for increased incentives for innovation and greater regulatory efficiency in the drug and device industry.

Both the Senate report and the E&C Committee's discussion draft are early proposals intended to generate further debate, yet the authors of both suggest that they intend to introduce comprehensive legislation that they hope will be passed later this year. Given the far-ranging issues that would need to be considered and debated, the goal of passing such legislation this year is extremely ambitious. If comprehensive legislation does not pass this year, it is possible that ideas generated through these efforts may be further considered as the reauthorization process for drug and device user fee laws ("PDUFA/MDUFA") gets underway.

Key Legislative Proposals to Watch for Drug and Device Manufacturers

The 21st Century Cures Initiative discussion draft contains wide-ranging legislative proposals that, if enacted, would significantly alter Food and Drug Administration ("FDA") oversight of drugs and medical devices.

For both drug and device manufacturers, notable highlights include proposals to

  • Modernize Clinical Research Regulation by clarifying the roles of central and local institutional review boards ("IRBs"), minimizing overlap in the regulatory requirements applicable to human subject research, and establishing a framework for companies to incorporate adaptive trial designs and Bayesian and other statistical methods into clinical trial designs in an effort to improve the efficiency of clinical trials. This provision would also empower the Secretary of Health and Human Services ("HHS") to make any necessary changes to the HHS Human Subject Regulations or vulnerable-populations rules to avoid conflict with or duplication of the new law and its accompanying regulations.
  • Permit Re-evaluation of Post-approval Study Requirements by creating a framework for FDA and sponsors to evaluate whether post-approval studies remain scientifically warranted, and to discontinue the studies or revise their scope where appropriate.
  • Redefine Rules for Communication of Scientific and Medical Developments by Manufacturers Regarding Their Products, including so-called "off label" information. As Ropes & Gray has previously reported here and here, FDA is re-evaluating its regulations and policies in response to citizen petitions filed by the Medical Information Working Group, a coalition of drug and device manufacturers to which Ropes & Gray serves as outside counsel. The draft contains a "placeholder" for this proposal, which, although still being developed by the E&C Committee, is certain to be one of the most closely watched provisions in the bill.
  • Clarify FDA Regulation of Social Media by, among other things, acknowledging that sponsors may present summary information concerning the safety and effectiveness of drugs and medical devices in character-limited applications like Twitter via hyperlinks. FDA has previously issued numerous draft guidance documents related to the regulation of social media promotion by drug and device manufacturers, as Ropes & Gray has previously reported here and here.
  • Require FDA to Issue Guidance on Drug-Device Combination Products, particularly with respect to the premarket review process for such products.
  • Launch the Precision Medicine Initiative. The draft contains a placeholder that may serve as a vehicle to discuss the initiative recently announced by the President, which among other things would increase efforts to identify genomic drivers in cancer and direct FDA to develop a new approach for evaluating tests that rapidly sequence large segments of a person's DNA.

For drug manufacturers specifically, notable highlights include proposals to

  • Incentivize Development of New Therapies, Dormant Therapies, New Antibiotics, and U.S.-Manufactured Generic Drugs by extending exclusivity for two years for significant improvements to existing molecules; giving developers of "dormant therapies" (i.e., products that address an unmet medical need but have insufficient patent protection to encourage development) an option to choose a 15-year period of exclusivity as an alternative to the exclusivity available under current law; creating a new transferable exclusivity program for antibiotics; and extending exclusivity for generic and biosimilar drugs manufactured in the United States.
  • Facilitate Surrogate Endpoint Qualification and Utilization by establishing a transparent process for qualifying surrogate endpoints, which can expedite drug approval by allowing drug effectiveness to be demonstrated by showing effect on a biomarker rather than clinical morbidity or mortality.
  • Expedite Approval of Breakthrough Therapies by clarifying FDA's authority to approve such drugs based on early-stage clinical data.
  • Streamline the Review of Supplemental New Drug Applications for New Indications by permitting FDA to accept and review data summaries rather than full data packages.
  • Incorporate Real-World Evidence by requiring FDA to issue guidance regarding the ability of drug manufacturers to rely on real-world use evidence to support approval of new indications and to satisfy post-approval study requirements.
  • Increase Transparency Regarding Expanded Access Programs for Unapproved Drugs by requiring drug companies to disclose details on their expanded access programs.
  • Incorporate Patient Perspectives in Drug Approval Decisions by requiring FDA to consider feedback from patients concerning desired benefits and tolerable risks associated with new treatments.
  • Foster 21st Century Manufacturing by requiring FDA to issue regulations and guidance that take into account modern drug manufacturing technologies.
  • Streamline Regulatory Processes for New Vaccines by establishing timelines for review of vaccines by the Centers for Disease Control ("CDC") and creating a process for expedited FDA review of breakthrough therapies and therapies needed during public health emergencies.

For device manufacturers specifically, notable highlights include proposals to

  • Incentivize the Development of Breakthrough Devices by establishing processes for priority review and accelerated approval of devices integrating breakthrough technologies that have the potential to address unmet medical needs, as well as transitional Medicare and Medicaid coverage of such breakthrough devices.
  • Accelerate Innovation and Patient Access by enabling patients to obtain certain medical device treatments earlier than the treatments would typically be available by establishing a list of devices for which manufacturers have opted out of Medicare secondary payer payment coverage.
  • Modernize the Regulation of Diagnostics, especially in light of FDA's 2014 proposal for the regulation of laboratory-developed tests, which was previously reported by Ropes & Gray here. The discussion draft currently contains a placeholder for this proposal.
  • Reform FDA Oversight of Medical Apps and Health IT by, among other things, distinguishing between "medical software" subject to FDA regulation and "health software" not subject to FDA regulation. FDA has previously issued numerous guidance documents related to the regulation of medical apps and health IT, as Ropes & Gray has previously reported here.
  • Secure the Medical Device Supply Chain by establishing a national framework for licensure of medical device wholesalers and third-party logistics providers.
  • Clarify FDA Review Standards for Evaluating Effectiveness of Devices by recognizing that well-documented, real-world evidence from clinical registries and published studies can qualify as "valid scientific evidence."
  • Streamline the Process for Pre-Market Review of Class I Devices by permitting manufacturers to submit a determination by an accredited third party of current good manufacturing practices compliance instead of a traditional 510(k) (to the extent a particular Class I device is not 510(k)-exempt altogether).
  • Provide Coverage for Disposable Medical Technologies by reforming the Medicare coverage and payment framework for certain disposable medical equipment.

Other highlights of interest to the drug and device industry include proposals to

  • Establish a 21st Century Cures Consortium, a public-private partnership that would bring together leaders from FDA, the National Institutes of Health, and the Centers for Medicare & Medicaid Services ("CMS") with leaders from the drug and device industry, academic research institutions, patient groups, health plans, and other stakeholders to accelerate the discovery, development, and delivery of innovative cures through the award of contracts and grants and other means.
  • Establish a Medicare Pharmaceutical and Technology Ombudsman within CMS to field complaints and requests from pharmaceutical and medical device companies.
  • Create a 21st Century Data Sharing Framework to enable, among other things, patients and physicians to better identify ongoing clinical trials and researchers and developers to use Medicare data to improve the quality of patient care.

Next Steps in the Legislative Process

The E&C Committee has stated that the discussion draft is "neither perfect nor complete." Several proposals are currently designated with placeholders, and the legislation has not yet been formally introduced in the House. The Senate's efforts are even more preliminary.

Thus, despite the aspirations of both the E&C Committee's discussion draft and the report from Senators Alexander and Burr, it remains to be seen what reforms Congress will be able to enact in the near term. Since 1992, when Congress enacted the original Prescription Drug User Fee Act, most FDA reform legislation has been incorporated into user fee reauthorization legislation enacted every five years (e.g., the FDA Modernization Act in 1997, the FDA Amendments Act in 2007, and the FDA Safety and Innovation Act in 2012). Given that the next user fee reauthorization legislation is not due to be enacted until 2017, any reform legislation enacted this year would depart from the typical five-year cycle.

As debate over the 21st Century Cures Initiative and complementary endeavors in the Senate continues in the coming weeks and months, legislative proposals that prove more controversial may be eliminated from the legislation to be enacted this year and may be deferred for consideration by FDA and industry as part of their statutorily mandated discussions over the next user fee reauthorization legislation slated for 2017.

Another key issue that will significantly affect the prospects of Congress enacting comprehensive reform legislation this year is funding. As initial ideas under the 21st Century Cures Initiative were discussed by the House last year, some House Republicans indicated that they would be unwilling to support increases in FDA funding. 

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.

In association with
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
Check to state you have read and
agree to our Terms and Conditions

Terms & Conditions and Privacy Statement

Mondaq.com (the Website) is owned and managed by Mondaq Ltd and as a user you are granted a non-exclusive, revocable license to access the Website under its terms and conditions of use. Your use of the Website constitutes your agreement to the following terms and conditions of use. Mondaq Ltd may terminate your use of the Website if you are in breach of these terms and conditions or if Mondaq Ltd decides to terminate your license of use for whatever reason.

Use of www.mondaq.com

You may use the Website but are required to register as a user if you wish to read the full text of the content and articles available (the Content). 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 & conditions or with the prior written consent of Mondaq Ltd. You may not use electronic or other means to extract details or information about Mondaq.com’s content, users or contributors in order to offer them any services or products which compete directly or indirectly with Mondaq Ltd’s services and products.


Mondaq Ltd and/or its respective suppliers make no representations about the suitability of the information contained in the documents and related graphics published on this server for any purpose. All such documents and related graphics are provided "as is" without warranty of any kind. Mondaq Ltd and/or its respective suppliers hereby disclaim all warranties and conditions with regard to this information, including all implied warranties and conditions of merchantability, fitness for a particular purpose, title and non-infringement. In no event shall Mondaq Ltd 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 or performance of information available from this server.

The documents and related graphics published on this server could include technical inaccuracies or typographical errors. Changes are periodically added to the information herein. Mondaq Ltd and/or its respective suppliers may make improvements and/or changes in the product(s) and/or the program(s) described herein at any time.


Mondaq Ltd requires you to register and provide information that personally identifies you, including what sort of information you are interested in, for three primary purposes:

  • To allow you to personalize the Mondaq websites you are visiting.
  • To enable features such as password reminder, newsletter alerts, email a colleague, and linking from Mondaq (and its affiliate sites) to your website.
  • To produce demographic feedback for our information providers who provide information free for your use.

Mondaq (and its affiliate sites) do not sell or provide your details to third parties other than information providers. The reason we provide our information providers with this information is so that they can measure the response their articles are receiving and provide you with information about their products and services.

If you do not want us to provide your name and email address you may opt out by clicking here .

If you do not wish to receive any future announcements of products and services offered by Mondaq by clicking here .

Information Collection and Use

We require site users to register with Mondaq (and its affiliate sites) to view the free information on the site. We also collect information from our users at several different points on the websites: this is so that we can customise the sites according to individual usage, provide 'session-aware' functionality, and ensure that content is acquired and developed appropriately. This gives us an overall picture of our user profiles, which in turn shows to our Editorial Contributors the type of person they are reaching by posting articles on Mondaq (and its affiliate sites) – meaning more free content for registered users.

We are only able to provide the material on the Mondaq (and its affiliate sites) site free to site visitors because we can pass on information about the pages that users are viewing and the personal information users provide to us (e.g. email addresses) to reputable contributing firms such as law firms who author those pages. We do not sell or rent information to anyone else other than the authors of those pages, who may change from time to time. Should you wish us not to disclose your details to any of these parties, please tick the box above or tick the box marked "Opt out of Registration Information Disclosure" on the Your Profile page. We and our author organisations may only contact you via email or other means if you allow us to do so. Users can opt out of contact when they register on the site, or send an email to unsubscribe@mondaq.com with “no disclosure” in the subject heading

Mondaq News Alerts

In order to receive Mondaq News Alerts, users have to complete a separate registration form. This is a personalised service where users choose regions and topics of interest and we send it only to those users who have requested it. Users can stop receiving these Alerts by going to the Mondaq News Alerts page and deselecting all interest areas. In the same way users can amend their personal preferences to add or remove subject areas.


A cookie is a small text file written to a user’s hard drive that contains an identifying user number. The cookies do not contain any personal information about users. We use the cookie so users do not have to log in every time they use the service and the cookie will automatically expire if you do not visit the Mondaq website (or its affiliate sites) for 12 months. We also use the cookie to personalise a user's experience of the site (for example to show information specific to a user's region). As the Mondaq sites are fully personalised and cookies are essential to its core technology the site will function unpredictably with browsers that do not support cookies - or where cookies are disabled (in these circumstances we advise you to attempt to locate the information you require elsewhere on the web). However if you are concerned about the presence of a Mondaq cookie on your machine you can also choose to expire the cookie immediately (remove it) by selecting the 'Log Off' menu option as the last thing you do when you use the site.

Some of our business partners may use cookies on our site (for example, advertisers). However, we have no access to or control over these cookies and we are not aware of any at present that do so.

Log Files

We use IP addresses to analyse trends, administer the site, track movement, and gather broad demographic information for aggregate use. IP addresses are not linked to personally identifiable information.


This web site contains links to other sites. Please be aware that Mondaq (or its affiliate sites) are not responsible for the privacy practices of such other sites. We encourage our users to be aware when they leave our site and to read the privacy statements of these third party sites. This privacy statement applies solely to information collected by this Web site.

Surveys & Contests

From time-to-time our site requests information from users via surveys or contests. Participation in these surveys or contests is completely voluntary and the user therefore has a choice whether or not to disclose any information requested. Information requested may include contact information (such as name and delivery address), and demographic information (such as postcode, age level). Contact information will be used to notify the winners and award prizes. Survey information will be used for purposes of monitoring or improving the functionality of the site.


If a user elects to use our referral service for informing a friend about our site, we ask them for the friend’s name and email address. Mondaq stores this information and may contact the friend to invite them to register with Mondaq, but they will not be contacted more than once. The friend may contact Mondaq to request the removal of this information from our database.


From time to time Mondaq may send you emails promoting Mondaq services including new services. You may opt out of receiving such emails by clicking below.

*** If you do not wish to receive any future announcements of services offered by Mondaq you may opt out by clicking here .


This website takes every reasonable precaution to protect our users’ information. When users submit sensitive information via the website, your information is protected using firewalls and other security technology. If you have any questions about the security at our website, you can send an email to webmaster@mondaq.com.

Correcting/Updating Personal Information

If a user’s personally identifiable information changes (such as postcode), or if a user no longer desires our service, we will endeavour to provide a way to correct, update or remove that user’s personal data provided to us. This can usually be done at the “Your Profile” page or by sending an email to EditorialAdvisor@mondaq.com.

Notification of Changes

If we decide to change our Terms & Conditions or Privacy Policy, we will post those changes on our site so our users are always aware of what information we collect, how we use it, and under what circumstances, if any, we disclose it. If at any point we decide to use personally identifiable information in a manner different from that stated at the time it was collected, we will notify users by way of an email. Users will have a choice as to whether or not we use their information in this different manner. We will use information in accordance with the privacy policy under which the information was collected.

How to contact Mondaq

You can contact us with comments or queries at enquiries@mondaq.com.

If for some reason you believe Mondaq Ltd. has not adhered to these principles, please notify us by e-mail at problems@mondaq.com and we will use commercially reasonable efforts to determine and correct the problem promptly.