United States: Progressing Toward A Cure: House Committee Unveils Revised, Streamlined 21st Century Cures Discussion Draft

On April 29, 2015, the House Energy & Commerce Committee ("E&C Committee") released a revised discussion draft of the medical innovation reform legislation that has been in the works for over a year as part of the 21st Century Cures Initiative. As Ropes & Gray previously reported, the E&C Committee released its initial discussion draft, which included a variety of legislative proposals organized in five titles spanning nearly 400 pages, at the end of January 2015.

The revised discussion draft continues to target the "the complete cycle of cures," organized in three broad titles—discovery, development, and delivery. Nonetheless, the revised discussion draft has been significantly pared down. A number of proposals, including those relating to drug exclusivity and laboratory developed tests, have been removed. The revised draft also includes several new proposals, including a provision proposing several modifications to Section 114 of the Food and Drug Administration Modernization Act (FDAMA). These revisions and the pared down nature of the draft likely are the product of negotiations among E&C Committee members. Significantly, unlike the original discussion draft noting no sponsors, the revised version lists five bipartisan sponsors, including the Chairs and Ranking Members of the full E&C Committee and the Health Subcommittee.

On April 30, 2015, the E&C Committee held a legislative hearing regarding the revised discussion draft at which representatives from FDA's Center for Drug Evaluation and Research and Center for Devices and Radiological Health, as well as the National Institutes of Health (NIH) testified. Committee mark-ups of the discussion draft are expected to occur soon, and E&C Committee chairman Fred Upton and others spearheading the 21st Century Cures Initiative have expressed their intent to enact legislation this year.

The key provisions that have been omitted from, added to, or substantially changed in the revised discussion draft are noted below.

What Has Been Cut?

A number of legislative proposals contained in the initial discussion draft have been omitted from the revised version. Notable omissions include proposals that would have:

  • Created or modified drug exclusivity programs, including a new exclusivity program for "dormant therapies," extended exclusivity for significant improvements and reformulations to existing drugs, extended exclusivity for American-manufactured generic drugs and biosimilars, extended exclusivity for orphan drugs, and transferable exclusivity for qualified infectious disease products
  • Clarified FDA regulation of social media
  • Modernized FDA regulation of diagnostics and laboratory-developed tests
  • Enhanced FDA's ability to approve breakthrough therapies based on early stage clinical data
  • Created an accelerated approval pathway and establishment of CMS coverage for breakthrough devices
  • Addressed supply chain security for medical devices
  • Updated current good manufacturing practice regulations and guidance accounting for modern drug manufacturing technologies
  • Created a program for re-evaluation of required post-approval studies and clinical trials
  • Established a 21st-century data-sharing framework
  • Clarified the regulation of drug-device combination products
  • Established a list of devices for which manufacturers have opted out of Medicare secondary payer payment coverage

What Is New?

The revised discussion draft also includes several proposals that were not offered in the earlier version. Highlights include proposals relating to:

  • Communication of Health Care Economic Information by Drug Manufacturers: The revised draft proposes several modifications to FDAMA 114, the statutory provision that permits drug manufacturers to convey health care economic information (HCEI) to formulary committees and other similar entities so long as the information is directly related to an approved indication and is based on competent and reliable scientific evidence. In particular, the draft (1) clarifies that payers are included within the audience permitted to receive health care economic information; (2) permits sharing HCEI that is "related to" the approved indication, as opposed to "directly related"; and (3) indicates that all components of the economic analysis, including the data, inputs, clinical or other assumptions, methods, results, and other components comprising the analysis, are included with the definition of HCEI (and thereby subject to the competent and reliable scientific evidence standard).
  • Guidance on CLIA Waiver Studies: A provision calling for FDA to revise its 2008 "Recommendations for Clinical Laboratory Improvement Amendments of 1988 (CLIA) Waiver Applications for Manufacturers of In Vitro Diagnostic Devices" guidance document to include information concerning the appropriate use of comparable performance to demonstrate device accuracy.
  • Accelerated Approval Development Plans: A provision allowing for the submission of accelerated approval development plans containing study parameters for the use of a surrogate endpoint intended to serve as the basis for the accelerated approval of drugs beyond the current scope of drugs intended to treat patients with life-threatening illnesses and unmet needs.
  • NIH Funding: The revised draft includes a new provision authorizing nearly $100 billion to be appropriated for NIH for fiscal years 2016 through 2018. Additionally, the draft establishes the NIH Innovation Fund, which would appropriate $2 billion for fiscal years 2016 through 2020 for the purpose of funding "Precision Medicine" and "Young Emerging Scientists" initiatives denoted in the previous draft with placeholders.
  • IT System for Data on the Natural History of Diseases: A provision has been added establishing an FDA public-private partnership to create a publicly available, secure information technology system with a focus on the natural history of rare diseases.
  • Repurposing Drugs: A placeholder for "repurposing drugs for serious and life-threatening diseases and conditions" has been added to the draft. If comments submitted by patient advocacy groups are incorporated into the language of this provision, incentives such as exclusive marketing rights could be included to encourage drug manufacturers to pursue therapies for conditions for which there currently are no treatments.

What Else Has Changed?

  • Precision Medicine: A placeholder has been replaced with a detailed provision directing FDA to publish guidance concerning the definition of precision drugs and methods for identifying subpopulations for biological characteristics research. Data obtained from research targeting such subpopulations may, in turn, be used by drug companies to seek orphan drug exclusivity.
  • Surrogate Endpoints and Biomarkers: Provisions related to surrogate endpoint qualification and utilization have been streamlined and modified to address the qualification of biomarkers, including surrogate endpoints that may be used for accelerated product approval. The streamlined provision also calls on FDA to publish guidance documents concerning the biomarker qualification.
  • Clinical Experience Evidence: A provision calling for FDA to incorporate real-world evidence into its review of drugs for new indications and post-approval study requirements has been revised to focus on evidence based on "clinical experience" and to provide FDA with detailed instructions for establishing a framework for the program.
  • Expanded Access Programs: A provision calling for drug manufacturers to disclose details on their expanded access programs for certain limited categories of drugs has been modified to require manufacturers to disclose their expanded access policies for any investigational new drug used in a phase 2 or phase 3 "human safety study."
  • Health Software: Provisions addressing FDA oversight of "medical" and "health" software have been modified to eliminate the term "medical software" altogether. "Health software" such as administrative, mobile fitness, and other software not intended to serve patient-monitoring purposes and not an "integral part of a device" would remain exempt from FDA regulation.
  • Valid Scientific Evidence for Devices: A provision clarifying that well-documented evidence from clinical registries and published studies can constitute "valid scientific evidence" for purposes of FDA's effectiveness evaluation has been modified to permit FDA to request in certain circumstances the underlying data from a study published in a peer-reviewed journal to confirm its validity. The prior provision would have presumed the validity of data from studies published in peer-reviewed journals.
  • Premarket Review of Class I Devices: A provision that would have streamlined the process for premarket review of Class I devices (to the extent such devices are not already 510(k)-exempt) has been replaced with a placeholder.
  • Disposable Medical Technologies: The details of a provision addressing Medicare coverage for disposable medical technologies have been replaced with placeholder text.

Prospects for the Legislation

The prospects of passage for 21st Century Cures legislation remain uncertain. A key issue discussed at the E&C Committee's April 30 hearing was the need for additional FDA funding to support and implement the legislative proposals in the revised discussion draft. FDA officials expressed concern about the potential for unfunded mandates, and some House Republicans have previously indicated that they would be unwilling to support funding increases for FDA.

In addition to the House's efforts with 21st Century Cures, the Senate is separately considering the need for medical innovation legislation. On April 28, 2015, the Senate Health, Education, Labor & Pensions ("HELP") Committee held its own hearing on medical innovation with participation from FDA and NIH officials. Unlike the House E&C Committee, the Senate HELP Committee has not released any legislative proposals, so it remains to be seen how similar or different the Senate and House bills will be, provided the Senate's recent hearings on Precision Medicine and Medical Innovation ultimately result in a bill.

Any legislative proposals that are considered too politically controversial or too technically complex for medical innovation legislation this year could be reassessed as part of the negotiations for the next reauthorization of the drug and device user fee laws (PDUFA and MDUFA, respectively). Those negotiations for the sixth version of PDUFA are slated to begin in June. Acting FDA Commissioner Stephen Ostroff, in public statements made in April, acknowledged that FDA is already advising members of the E&C Committee as to which proposals are appropriate for the 21st Century Cures legislation and which should be deferred for discussion as part of PDUFA-VI negotiations.

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.

Authors
Similar Articles
Relevancy Powered by MondaqAI
McDermott Will & Emery
McDermott Will & Emery
 
In association with
Related Topics
 
Similar Articles
Relevancy Powered by MondaqAI
McDermott Will & Emery
McDermott Will & Emery
Related Articles
 
Related Video
Up-coming Events Search
Tools
Print
Font Size:
Translation
Channels
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
Password
Confirm Password
Position
Mondaq Topics -- Select your Interests
 Accounting
 Anti-trust
 Commercial
 Compliance
 Consumer
 Criminal
 Employment
 Energy
 Environment
 Family
 Finance
 Government
 Healthcare
 Immigration
 Insolvency
 Insurance
 International
 IP
 Law Performance
 Law Practice
 Litigation
 Media & IT
 Privacy
 Real Estate
 Strategy
 Tax
 Technology
 Transport
 Wealth Mgt
Regions
Africa
Asia
Asia Pacific
Australasia
Canada
Caribbean
Europe
European Union
Latin America
Middle East
U.K.
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.

Disclaimer

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.

General

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