United States: Senate Passes 21st Century Cures Act, But Can It Cure An Ailing FDA?

On Wednesday, the U.S. Senate overwhelmingly passed the 21st Century Cures Act (the "Act") by a vote of 94 to 5.  Spearheaded by Michigan Representative Frank Upton, the bill now heads to President Obama who has promised to sign it. The Act is ambitious, and will impact a wide swath of the U.S. health care system. The Act provides, among other things:

  • $4.8 billion over 10 years to support NIH research on precision medicine, neuroscience, cancer and regenerative medicine.
  • $1 billion in state grants to increase opioid abuse prevention and treatment services, including prescription drug monitoring programs, training programs and treatment programs.
  • Substantial changes to FDA regulations to accelerate the pace of bringing pharmaceuticals and medical devices to market.
  • New obligations on the part of both FDA and industry stakeholders to implement the research initiatives and regulatory changes mentioned above.
  • Other health care initiatives addressing health information technology, vaccines, national security and health care delivery.

At 996 pages, the Act cannot be summarized in one post. Instead, we plan to analyze the various aspects of the Act in multiple posts over the coming weeks. The remainder of this post will highlight provisions that support one of the Act's primary objectives: the acceleration of drugs and devices to market.

Easing and Accelerating the Pathway to Market

A review of the Act uncovers a number of provisions whose purpose is to accelerate the process of bringing drugs and devices to market. We have summarized these provisions below and have provided context when applicable.

Pharmaceuticals and Biologics:

  • Qualification of Drug Development Tools. In 2012, FDA created the Drug Development Tools (DDTs) Qualification Program that it used to validate biomarkers, clinical outcomes assessments, and animal models. Biomarkers, for example, can be used to show whether a certain biological response has occurred in a patient receiving a pharmaceutical treatment. The Act establishes a formal review pathway at FDA for biomarkers and other drug development tools that can be used to help shorten drug development time and reduce the failure rate in drug development.
  • Targeted Drugs for Rare Diseases. The Act clarifies FDA's authority with respect to genetically targeted drugs for rare diseases. Sponsors of genetically targeted or variant protein targeted drugs will be permitted to rely on data for the same or similar technology from previously approved applications by the same sponsor.
  • Reauthorization of Pediatric Rare Disease Priority Review. The Act reauthorizes the pediatric rare disease priority review voucher program until 2020. The program offers vouchers to companies for gaining approval of new rare pediatric treatments affecting fewer than 200,000 patients under the age of 18. These vouchers enable companies to have any one of their drugs reviewed under FDA's priority review system.
  • Novel Clinical Trial Designs. FDA will be required to hold public meeting and issue guidance documents that would assist sponsors in incorporating adaptive designs and novel statistical modeling into new drug applications. "Adaptive designs" refer to changes in the design or analyses of a trial based on an examination of the accumulated data at an interim point in the trial. For example, adaptive designs can be used to make studies more efficient (e.g. by reducing the duration of the study) or more informative (e.g., by providing broader dose-response information.) The FDA provided guidance on adaptive designs in 2012.
  • Real World Evidence for New Indications of Previously Appro3ed Drugs. FDA will be required to evaluate the use of real world evidence to help support the approval of a new indication of a previously approved drug and to help support or satisfy post-approval study recommendations.
  • Data Summaries to Support Approval for New Indications. FDA will be allowed to rely upon qualified data summaries to support the approval of an application for a new indication of an already approved drug. Sponsors will still be required to submit all of the traditional information to the FDA.
  • Expanded Access Policy. The Act requires pharmaceutical companies to have publicly accessible compassionate use policies for drugs treating serious or life-threatening conditions. Compassionate use (also known as expanded access) allows physicians to request FDA approval of the use of an investigational drug outside of a clinical trial when a patient is terminally ill or has no other treatment options. However, this process cannot begin without cooperation by the pharmaceutical company producing the drug, hence the provision.
  • Accelerated Approval for Regenerative Therapeutic Products. FDA will be allowed to grant accelerated approval for regenerative therapeutic products. FDA is directed to consider the unique characteristics of regenerative therapeutic products and provide a rationale with a determination of whether or not to grant accelerated approval. The Act does not change the standards of evidence or limit any other of the FDA's authorities. Regenerative therapeutic products include stem cell therapies that are derived from a patient's own cells. The magazine Wired recently published an in-depth article on the current state of regenerative therapeutic products.

Medical Devices

  • Creation of Breakthrough Device Pathway for Medical Devices. The Act establishes a breakthrough device pathway, which builds on the existing priority review device pathway.
  • Increase in Humanitarian Device Exemption Cap. The FDA will now have the authority to apply the humanitarian device exemption to devices that treat diseases and conditions that affect up to 8,000 individuals in the U.S. The current cap is 4,000.
  • Updates to Class I and II Devices. FDA is requires to update lists regarding the appropriate regulation of Class I and Class II devices.
  • Institutional Review Board Flexibility. The Act strikes the requirement that a sponsor of a medical device trial always use a local institutional review board. This change will allow the use of centralized IRB models, which FDA has published guidance on in the past.
  • Least Burdensome Device Review. The Act clarifies that FDA reviewers shall consider the least burdensome appropriate means necessary for demonstrating a reasonable assurance of safety and effectiveness when requesting additional information from manufacturers during the pre-market approval (PMA) process. The Act also requires an audit by the FDA ombudsman and an assessment of the measurements used to track the implementation of the least burdensome requirements.
  • Clarifying Medical Software Regulation. The Act identifies 5 specific categories of medical software that, given certain conditions, will not be regulated as a medical device by the FDA based on their low level of risk to patients. It also provides FDA with the authority to regulate software in these categories if there is found to be safety concerns.

Combination Products

  • Classification of Devices Used with Regenerative Advanced Therapies. The Act establishes that devices used with a regenerative therapeutic product will be considered moderate risk devices, unless the Secretary determines that the device or intended use requires a higher risk classification.
  • Dispute Resolution for Combination Products. The Act clarifies how dispute resolution works when the different centers of the FDA do not agree on how to treat a combination product. Combination products are products that contain both a drug and device.

Commentary

Will the Act in fact accelerate the process of bringing drugs and devices to market? The answer depends on numerous factors including the stance of the new administration as well as how FDA approaches the regulations needed to implement the Act.  Further complicating the Act's implementation is the fact that the FDA is either opposed to, or at least conflicted about, some of the provisions. For example, the FDA has previously objected to the Pediatric Rare Disease Priority Review program which essentially allows companies to jump the review line.  And there is always the issue of money. The FDA has been allocated $500 million over 10 years to implement these changes. Whether that will cover these ambitious reforms is certainly an open question.

Future posts will dive deeper into a number of these and other provisions, including those related to:

  • Medical software
  • Real world evidence
  • Intercenter Institutes at FDA
  • Human Subject Protections

Please stay tuned for an announcement on a 21st Century Cures Act webinar series that we are planning for January 2017.

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
 
In association with
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
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.

Disclaimer

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.

Registration

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.

Cookies

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.

Links

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.

Mail-A-Friend

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.

Security

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.