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News and Press: Policy News

Federal, State, and International Advocacy Alert: July 2, 2014

Wednesday, July 2, 2014   (0 Comments)
Posted by: Stephen Rapundalo
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Policymakers at the FDA were extremely busy over the last several weeks. Highlights of the many final and proposed regulations released are described below. A complete list of guidances issued recently can be found both for drugs and medical devices.

Additionally, Michigan state legislators have suddenly introduced a rash of new bills that have implications for the bio-industry, particularly the drugs and pharmaceuticals sector, see the State Policy section below for more information.

As always, contact us with any of your legislative and policy concerns at and visit our advocacy pages for background resources on a wide range of regulatory and policy topics.

Federal Policy

FDA Proposes Social Media Guidelines for Pharma, Medtech  
Two draft guideline documents were issued Tuesday by the FDA requiring pharmaceutical and medtech companies to balance discussion of benefits and risks associated with their products in social media communication. The agency also outlined how companies can address misinformation posted by others about their products. The proposals are open for comment within 90 days.

The FDA’s first draft guidance entitled “Internet/Social Media Platforms with Character Space Limitations—Presenting Risk and Benefit Information for Prescription Drugs and Medical Devices” is geared toward manufacturers, packers, and distributors of prescription human and animal drugs, and medical devices for human use. The guidance specifically addresses how these companies should present the benefit and risk information of their FDA-regulated medical products on electronic/digital platforms with character space limitations such as Twitter, sponsored search engine links, and other social media and internet-based outlets.  

A second draft guidance entitled “Internet/Social Media Platforms: Correcting Independent Third-Party Misinformation About Prescription Drugs and Medical Devices” describes the FDA’s current thinking about how the pharma and medtech industries should respond, if they choose to respond, to misinformation related to a firm’s own FDA-approved or -cleared products. The guidance specifically addresses information that is created or disseminated by independent third-parties on the internet regardless of whether that misinformation appears on a firm’s own forum/website or an independent third-party forum/website. 

FDA Issues Action Plan on Heels of Independent Assessment of Medical Device Review Processes
As part of the Medical Device User Fee Act of 2012 ("MDUFA III") Commitment Letter, the FDA and the medical device industry agreed to participate in a comprehensive outside assessment of the medical device review process. The assessment was performed by Booz Allen Hamilton ("Booz Allen") that issued its final report recently.

The final report makes 11 recommendations, including, among other things, that the FDA’s Center for Medical Device & Radiological Health (CDRH) conduct a retrospective review of withdrawn premarket submissions to identify the cause of the withdrawal. The report cites a fifty percent increase in 510(k) withdrawals from fiscal year 2012 to fiscal year 2013. 

AdvaMed released a statement regarding the report’s findings that was generally supportive of any plans to develop consistency and efficiency in the review process. However, it did adopt a ‘wait-and-see’ approach and hoped that the FDA would follow through on its implementation and monitoring plans. Similarly, MDMA in its comments stated that the focus on “increasing consistency and predictability must be paramount.”

Concomitantly, the CDRH issued its Plan of Action in response to the Booz Allen report. The CDRH Plan of Action, which reads somewhat like a FDA Form 483 response by a device manufacturer, outlines actions with respect to each of the four priority recommendations. With regard to the first recommendation, “develop criteria and establish mechanisms to improve consistency in decision making throughout the review process,” the CDRH will inventory and perform a gap assessment of its existing processes, procedures, policies, and metrics relating to premarket applications (510(k)s, PMAs, 510(k) requests for Additional Information, PMA Major Deficiencies, and IDE approval decisions). Once complete, CDRH plans to identify lessons learned and best practices for decision making from other organizations and then develop new premarket application review processes, procedures, and policies with the intent of streamlining the process. 

Medtech Prices Lag Behind CPI, Other Medical Products
AdvaMed released a new study on industry pricing, arguing that medical devices represent a consistently small share of total healthcare spending in the U.S. The study refutes claims that medical devices are a driver of rising health care costs, and aims to dispel misconceptions about medtech spending and pricing.

Final Phase of Global Unique Device Identification Database Guidance Released
FDA announced the availability of a guidance entitled, “Global Unique Device Identification Database (GUDID).” The guidance finalizes the sections on how device labelers will interface with the GUDID, establish GUDID accounts, begin initial submissions, and finalizes the sections on the Device Identifier (DI) record. 

This guidance describes key GUDID concepts such as account management, user roles, the device identifier (DI) module, DI record life-cycle, package configurations, and the two GUDID submission options (web interface and HL7 SPL). In addition, two components of the draft GUDID Guidance have been removed from the guidance and posted separately on the FDA UDI website

FDA Draft Proposes no Regulation of Medical Device Data Systems
The FDA issued draft guidance on “Medical Device Data Systems, Medical Image Storage Devices, and Medical Image Communications Devices.” This document is to inform manufacturers, distributors, and other entities that the agency does not intend to enforce compliance with the regulatory controls that apply to MDDS, medical image storage devices, and medical image communications devices, due to the low risk they pose to patients and the importance they play in advancing digital health. The document also proposes edits to the agency’s guidance entitled “Mobile Medical Applications” (September 25, 2013) which would conform to the policy discussed in this draft guidance, when finalized. 

Draft Guidance on Counterfeit Drugs Released
The FDA announced the availability of a draft guidance for industry entitled “Drug Supply Chain Security Act Implementation:  Identification of Suspect Product and Notification.” This draft guidance document is intended to aid certain trading partners (manufacturers, repackagers, wholesale distributors, and dispensers) in identifying a suspect product and terminating notifications regarding illegitimate product.

This draft guidance is the first of many in the FDA’s implementation of the Drug Supply Chain Security Act (DSCSA). The DSCSA tasked the FDA with addressing three issues in this guidance: 1) identifying scenarios that could increase the risk of suspect product entering the supply chain; 2) recommending ways trading partners can identify and determine whether product is suspect; and 3) creating a process for trading partners to terminate illegitimate product notifications. 

The FDA makes clear throughout the draft guidance, it views the portions of the guidance related to termination of illegitimate product notifications as binding upon finalization. 

FDA Issues Draft Guidance on Disseminating Information on Drug Products
The FDA announced the availability of a draft guidance for industry entitled “Distributing Scientific and Medical Publications on Risk Information for Approved Prescription Drugs and Biological Products—Recommended Practices.” This guidance document describes the FDA’s current thinking on recommended practices for drug manufacturers and their representatives when distributing to health care professionals or entities, scientific or medical journal articles that discuss new risk information for approved prescription drugs, and how to address issues specific to the distribution of new information about risks associated with a drug that further characterizes risks identified in the approved labeling.

ANDA Submissions Draft Guidance Released
The FDA issued a draft guidance for industry entitled, “ANDA Submissions - Content and Format of Abbreviated New Drug Applications.” The guidance document is intended to assist applicants in preparing complete and high-quality original abbreviated new drug applications (ANDAs) for submission to the FDA under the Federal Food, Drug, and Cosmetic Act, and summarizes the statutory and regulatory requirements for ANDAs, references existing guidance documents, and incorporates additional recommendations on the content and format of ANDA submissions. 

FDA Outlines Expectations for Human Drug Compounders
The FDA issued several policy documents regarding compounded drug products for human use, as part of the agency’s continuing effort to implement the compounding provisions of the Drug Quality and Security Act (DQSA), enacted in November 2013. The policy documents consist of a draft interim guidance, a proposed rule, a final guidance, and two revised requests for nominations for the bulk drug substances lists:
In addition, two Federal Register Notices were issued stating the FDA is reopening the nomination process for two lists of bulk drug substances (active pharmaceutical ingredients) that may be used to compound drug products. One list is for drug products compounded in accordance with section 503A, and the other list is for drug products compounded in accordance with section 503B of the FD&C Act.

The draft interim guidance and proposed rule are available for public comment for 60 days, and the dockets are open for the public to nominate bulk drug substances for compounding under section 503A or 503B for 90 days. 

Guidance Issued on Use of Nanotech Products
The FDA released three final guidances and one draft guidance regarding nanotechnology, reflecting the department's stance on regulating products that contain nanotechnology rather than nanotechnology itself. 

One final guidance addresses the FDA’s approach for all products that it regulates, while the two additional final guidances and the new draft guidance provide specific guidance for the areas of foods, cosmetics, and food for animals, respectively:

The FDA does not make a categorical judgment that nanotechnology is inherently safe or harmful, and plans to consider the specific characteristics of individual products. All four guidance documents encourage manufacturers to consult with the agency before taking their products to market. A Nanotechnology Fact Sheet can be found here

The public is invited to comment on the draft guidance document beginning on June 27, 2014, and until September 10, 2014. To submit your comments electronically to the docket go to docket number FDA-2013-D-1009 at

To submit your comments to the docket by mail, use the following address. Be sure to include docket number FDA-2013-D-1009 on each page of your written comments.

The Division of Dockets Management
Food and Drug Administration
5630 Fishers Lane, Room 1061
Rockville, MD 20852

State Policy

“Right to Try” Legislation Wave Hits Michigan
“Right to Try” legislation was introduced recently in Michigan by Rep. Gail Haines (R-Waterford) and Sen. John Pappageorge (R-Troy). The proposed bills, HB 5651 and SB 0991, respectively, would give patients with terminal illnesses the ability to access drugs that have not earned final approval from the FDA. The drugs would have to have passed Phase 1 clinical trial safety tests but not Phase 2 tests on effectiveness and wider Phase 3 evaluations. 

The nonprofit Goldwater Institute, an extremely conservative think tank, has helped spur the "Right to Try" proposals in states across the country. The organization argues on its website that patients should be free to exercise a basic freedom—attempting to preserve their own lives. "The FDA, however, often stands between the patients and the treatments that may alleviate their symptoms or provide a cure," the Goldwater Institute says. Goldwater and other proponents say that too many patients are dying because of the lengthy wait times associated with drug approval from the FDA. In its template statute, Goldwater doesn’t just liberalize FDA regulations, it removes the agency completely! 

However, serious issues are created with passage of state “Right to Try” laws. First, it could provide patients with a false hope on the impact state legislation could have on access to novel therapies and devices. Such laws are likely to be either wholly unconstitutional or at least unconstitutional in the majority of possible applications. That is because the FDA has exclusive jurisdiction over the regulation of drugs and state laws that impinge on that exclusivity are pre-empted.

Moreover, once the FDA-drug approval process is underway, pharmaceutical/biotech manufacturers are by definition under FDA jurisdiction and would be circumspect in allowing use of any drug still in development that might cause them to run afoul of regulators. The FDA is certain to take a dim view of a manufacturer going off and distributing that same drug to patients outside the trial process where there would be little if any controls and oversight. For an excellent review of the “right to try” issue, read this article by Dr. David Gorski

The Michigan “Right to Try” bills were referred to the House and Senate Health Policy Committees. Consideration and debate of the bills are likely to occur this fall when the Legislature returns in full session.

State Biosimilars Substitution Legislation Introduced 
Michigan is the latest state to have a biosimilars bill introduced as states pre-emptively grapple with how to deal with an expected influx of biosimilar drugs. HB 5958, sponsored by Rep. Ken Yonker (R-Caledonia), would allow pharmacists in Michigan to substitute from a biologic drug to a lower-cost biosimilar if available. However, the bill does not place any further requirements in terms of notifying physicians of the substitution.

A series of biosimilars substitution bills/laws have made their way through other states. Just last week Massachusetts became the 7th state to pass a law allowing pharmacy substitution of interchangeable biosimilars. However, that law along with the one in Indiana and a few other states incorporated policies that safeguard patient safety and the primacy of the physician-patient relationship, recognizing that treating physicians and their patients are in the best position to determine appropriate therapies. 

Such safeguards are encapsulated in the so-called “5 BIO Principles on Patient Safety in the Substitution of Biologic Products.” Essentially the principles include the following:

  • Substitution should occur only when the FDA has designated a biologic product as interchangeable.
  • The prescribing physician should be able to prevent substitution.
  • The prescribing physician should be notified of the substitution.
  • The patient, or the patient’s authorized representative, should, at a minimum, be notified of the substitution.
  • The pharmacist and the physician should keep records of the substitution.
MichBio is working alongside a coalition of stakeholders including various biopharmaceutical companies, BIO, and PhRMA, as well as other state professional organizations, to insure that a sound policy outlining parameters for safe substitution of interchangeable biologics is the best option to ensure patients have access to high-quality, safe, and effective biologic medicines.

International Policy

EMA Issues Reflection Paper on Advanced Therapies
The European Medicines Agency (EMA) announced the availability of draft reflection paper entitled, “Classification of Advanced Therapy Medicines Products.” This paper is intended to provide guidance on the Advanced Therapy Medicinal Products (ATMPs) classification procedure, as well as the interpretation of key concepts of the definition of gene therapy medicinal products that include somatic cell therapy medicinal product, tissue engineered product, and combined advanced therapy medicinal product.

European Agencies Release Reimbursements Report
The European Medicines Agency (EMA) and the European network for Health Technology Assessment (EUnetHTA) have announced the availability of a report entitled “Improving the contribution of regulatory assessment reports to health technology assessments—a collaboration between the European Medicines Agency and the European network for Health Technology Assessment.” The report is part of an EMA initiative to better coordinate with the health technology assessment (HTA) bodies that determine suitable reimbursements.


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