United States: FDA 2017 Year In Review: Therapeutic Products, Part 2

Yesterday we started off our year-end series of blog posts with the first part of a review of FDA's actions for 2017 in the therapeutic products space. Part 1 recapped Commissioner Gottlieb's initiative to tackle drug competition issues, with the end goal of affecting drug prices, as well as current figures related to this year's new drug and biologic approvals. A large proportion of those new products took advantage of the Breakthrough Therapy program and other expedited development/review pathways. Today we'll begin where we left off and consider how the newest expedited review pathway, the RMAT Designation program, is doing one year after its creation by Congress.

Regenerative Medicine's RMAT Designation Is the New Kid in Town

Because Congress recognized that personalized medicine was trending toward the use of modified autologous immune cells (like CAR-T therapies highlighted in Part 1 of this year-in-review post), stem cells, homologous tissues, and even mini-organs, the Cures Act created a new expedited pathway for what are now called Regenerative Medicine Advanced Therapies (see our previous post here). FDA quickly implemented this new pathway through the RMAT Designation Program, formally announced in March 2017. The RMAT Designation has been extremely popular, with at least 11 designations granted out of 31 reviewed by FDA as of mid-November, when the director of the Center for Biologics Evaluation and Research updated the public on the program at a conference in Washington, D.C. Expanding upon that congressional intent, Commissioner Gottlieb was forthright about the fact that gene therapy products may also be eligible to be treated as RMATs even though they're not specifically mentioned in the Cures Act definition. The Agency has set forth this policy in a draft guidance and Dr. Gottlieb recently testified about it on Capitol Hill during his progress reports on the 1-year anniversary of the Cures Act (see, e.g., here).

We also predicted in our year-end post for 2015 that a watershed moment was coming for the human cells, tissue, and cellular and tissue-based product (HCT/P) industry. While we were wrong that such a moment would happen in 2016, it does seem to have happened this year. In August 2017, Dr. Gottlieb took a strong position about the need to police "unscrupulous" stem cell clinics that may be peddling in snake-oil treatments for Alzheimer's and other debilitating diseases for which few approved therapeutic options are available. The long-awaited policy announcement came with two high-profile enforcement actions and was followed by the release of several important guidance documents for the HCT/P industry in mid-November.

At the same time, FDA also released two draft guidance documents aimed at advancing regenerative medicine as mandated by the Cures Act. The Agency announced the release of this "suite" of regenerative medicine guidance documents in November as ushering in a Comprehensive Policy Framework for the field. Between the administrative and fundraising benefits that can now be tapped using the new RMAT Designation and those RMAT guidance documents – entitled "Expedited Programs for Regenerative Medicine Therapies for Serious Conditions" and "Evaluation of Devices Used with Regenerative Medicine Advanced Therapies" – companies and researchers working in this exciting area had a lot to talk about this year.

And Finally – Some Observations on Enforcement in Drugs and Biologics

We've noted above a very important development for HCT/P developers and researchers working in the regenerative medicine space: FDA is clearly going to take more aggressive enforcement action against rogue stem cell clinics. That was significant enforcement news after many years of inaction by the Agency in the face of a burgeoning marketplace for unapproved and potentially very risky cellular products, many of which are promoted directly to consumers. But when it comes to the Agency's enforcement activities in more traditional drug and biological industries under the Federal Food, Drug, and Cosmetic Act (FFDCA), it was business as usual with upticks in certain areas based on recent Agency priorities, such as in the drug compounding space due to ongoing implementation of and inspections based on the Drug Quality and Security Act (DQSA), now four years old. Indeed, the DQSA/compounding quality did receive significant attention from Commissioner Gottlieb during his first six months, including as a subject of one of his numerous public statements in September.

Some important regulatory enforcement trends worth noting this year include:

  1. Data integrity problems for human drug products, especially at foreign facilities, continued to be a high priority enforcement area for the Agency, as we discussed in our 2016 overview blog post. In a public presentation made on December 6, 2017, Donald Ashley, the director of CDER's Office of Compliance (OCE), reported that the Office of Manufacturing Quality within OCE had issued 57 Warning Letters as of November 1, 2017 and created 47 new Import Alerts. Many of these enforcement actions were the result of serious violations of good manufacturing practices (GMPs), which included lack of data integrity and in some cases, outright falsification or destruction of documents. U.S. firms have also become more of a focus of data integrity deficiencies in recent years: consider the fact that no U.S. drug manufacturers had such violations cited in 2014 GMP-related Warning Letters, whereas now a majority of GMP-related Warning Letters issued to U.S. firms include some form of data integrity violation.
  2. The Office of Prescription Drug Promotion's issuance of enforcement letters related to violative prescription drug advertising and other promotional vehicles hit a remarkable low, with only 3 letters during this calendar year as of December 15, 2017. In comparison, during 2016 OPDP issued a total of 11 warning or untitled letters; a total of 9 in 2015; 10 in 2014; and 24 in 2013. This trend likely can be attributed to several different factors, including drug companies' and their ad agencies' increased understanding of the regulatory requirements for prescription drug promotion. But it also likely signals that OPDP is in a state of flux as the Agency's off-label policies are reexamined under Commissioner Gottlieb's leadership. We would note, however, that many OPDP requirements have nothing to do with off-label speech. Companies should not become lackadaisical when it comes to policies and procedures related to their advertising compliance.
  3. As alluded to above, FDA inspections of traditional compounders and outsourcing facilities continue be very high priority as the Agency seeks to reform the drug compounding industry under the congressional mandate that is the DQSA. Compliance Director Ashley's year-end presentation (see above) reported that, for Fiscal 2017 (year ended Sept. 30, 2017) the Office of Unapproved Drugs and Labeling Compliance conducted 141 compounding inspections; issued 62 Warning Letters to compounders; brought 2 injunctions against compounders; and oversaw 41 recall events involving compounded drugs. These reported data do not differentiate between traditional compounders and outsourcing facilities.

In the coming days, we'll be releasing our FDA 2017 Year in Review posts for digital health and medical devices, including diagnostics. Stay tuned for more as you prepare to usher in 2018!

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 Topics
 
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