Device Firms: What You Should Know About The 21st Century Cures Act

By Mason Weeda

Last month, legislation that would affect, among other things, FDA’s regulation of medical devices and the manufacturers of such devices took a considerable step forward when it was introduced and then unanimously approved by the House Energy and Commerce Committee by a vote of 51-0 on May 21.  Known as the 21st Century Cures Act (“Act”), the stated aim of the legislation is to modernize and personalize health care, encourage innovation, support research, and streamline the U.S. health care system to promote the delivery of better and faster “cures” to more patients.

In support of this goal, the Act would change the review of medical devices determined to be “breakthrough,” establish a third-party option for the inspection of medical device manufacturers, make changes to certain Humanitarian Device Exemption (“HDE“) requirements, institutionalize FDA’s ongoing efforts regarding the regulation of medical software applications, and loosen some clinical investigation requirements.  Significantly, the legislation also indicates an ongoing Congressional interest in the restrictions FDA has placed on the dissemination of truthful and nonmisleading off-label information.

Breakthrough Device Pathway

The Act would support faster “cures” by creating a “priority review” pathway for those devices that meet the definition of a “breakthrough device.”  These “breakthrough devices” include those that “represent breakthrough technologies… for which no approved alternative exist,” offer “significant advantages over existing approved or cleared alternative,” and are “otherwise in the best interest of patients.”

Upon a sponsor’s request, FDA would determine whether a device meets the “breakthrough device” designation using specified criteria.  If a device receives such designation, it would be eligible for expedited review by a team of staff that will interact with the device sponsor.  During this process, the Act would require FDA to “take steps to ensure that the design of clinical trials is as efficient as practicable, such as through adoption of shorter or smaller clinical trials, application of surrogate endpoints and use of adaptive trial designs and Bayesian statistics.”  Likewise, the agency would also be required to “facilitate … expedited and efficient development and review of the device through utilization of postmarket data collection.” Although these are laudable goals, the Act does not impose any specific timelines in which an “expedited review” must be completed or otherwise quantify how much existing review times will be reduced.

Third-Party Inspections of Device Manufacturers

The Act also contains provisions that could allow FDA to conserve its inspectional assets and speed up approval of modified versions of existing devices by allowing for the use of third party inspectors to conduct the necessary establishment inspections.  The theory being that creating a “quick” method to inspect facilities in these circumstances would promote the earlier availability of improved “cures.”

Under the provisions of the Act titled “Medical Device Regulatory Process Improvements,” Congress would require FDA to establish a “third-party quality system assessment” program, where accredited third parties would inspect manufacturers for compliance with the Quality System Regulation (“QSR”) (21 C.F.R. Part 820).  However, use of such third-party inspections would be limited only to QSR inspections necessary as the result of submissions involving “device related changes” and would not be available in other types of establishment inspections.

Changes to Humanitarian Device Exemptions

This section of the Act would double the number of patients that must suffer from a disease in order for FDA to consider it a “rare disease.”    Presently, the HDE pathway is intended to incentivize and encourage the development of devices to treat “rare” diseases or conditions affecting small patient populations when the device manufacturer`s research and development costs would otherwise exceed its market returns.  It does so by significantly reducing the clinical data that would be necessary for the manufacturer to generate to support the efficacy of the device that would otherwise be required by FDA in a traditional marketing application. To qualify for an HDE, the disease or condition must presently affect fewer than 4,000 individuals in the United States per year. The Act seeks to encourage additional development making “cures” more widely available by increasing this number to 8,000 individuals.

Medical Software

In an apparent effort to “modernize” the technology involved in health care, the Act would create a definition of “health software,” which generally would not be regulated unless it:

  • is intended for use to analyze information to provide patient-specific recommended options; or
  • FDA determines that it poses a significant risk to patient safety.

This provision continues to allow FDA some flexibility as to how it may regulate software.  The Act also would require the agency to review existing regulations and guidance regarding software, including the classification of software, standards of verification and validation, review of software, and quality system for software, among others.

Clinical Trials

The Act would also make it easier for sponsors conducting clinical investigations by requiring the Department of Health and Human Services (“HHS”) to harmonize its requirements applicable to clinical investigations with FDA’s own requirements. This supports the Act’s goals by significantly reducing the regulatory burden imposed on sponsors who must presently ensure that their clinical investigations meet the often duplicitous requirements imposed by both HHS and FDA controls.  The Act also would make it easier for sponsors to meet Institutional Review Board (“IRB”) requirements by allowing the use of non-local IRBs to review medical device trials, including Investigational Device Exemptions (“IDE”) and HDEs. Permitting the use of non-local IRBs support the Act’s goal of “quicker cures” by eliminating the “log jam” and delays sometimes associated with the use (and overuse) of local IRBs by giving sponsors additional options that are potentially faster than the traditional ones.

Promotion

Lastly, and without limitation, the Act’s section on “Facilitating Responsible Communication of Scientific and Medical Developments,” provides that FDA “shall, within 18 months, issue draft guidance on facilitating the responsible dissemination of truthful and non-misleading scientific and medical information not included in the approved labeling of drugs and devices.”  This provision appears to be in response to the Coronia Case (U.S. v. Caronia, 703 F.3d 149 (2d Cir. 2012)), which holds that representatives of pharmaceutical manufacturers have a right under the First Amendment to make truthful statements regarding their products, even if such statements indirectly promote drugs for uses not approved by FDA.  The Act does not provide further direction on this topic, but Congress is clearly nudging FDA to update its position on off-label promotion which may affect medical device manufacturers.

It may be an uphill battle for Congress to agree on all topics involved in the 308 pages of 21st Century Cures Act.  However, as reported by the House Energy and Commerce Committee Press Releases, the bill appears to have support from industry and consumer groups, which may help bring Congress together.

John Block: GMOs Under Assault

By John R. Block

We can’t seem to silence or satisfy the loud scream of opposition to GE crops. The reason probably is that up until now we have ignored them. It’s time to stand up and set the record straight.

GE food is safe and even beneficial according to 88% of scientists. We have conducted more than 1,000 studies. We have been eating GE food for more than 20 years – no one has gotten sick.

The fact is that for thousands of years, farmers have been improving crops through selective breeding. That process alters genes. We have found that in the laboratory we can do it faster.

Never mind the facts, the critics are beating the drums. Three states have passed labeling laws. Vermont is in the lead as they begin to implement their law. It sounds simple. Just label the food if it has GMO products in the food.

It’s not that simple. Vermont has a long list of exemptions. Animal products are exempt – beef, pork, chicken, dairy. But keep in mind the animals were fed GMO corn and soybean meal.

Trying to get out ahead of everyone, Chipotle recently announced that it has gone GMO free. But it’s not really free. The soft drinks are made with GMO corn sweetener. The burritos have GMO corn-fed beef, pork, chicken, and GMO sour cream.

The U.S. Congress is considering as many as 30 bills to deal with the GMO debate. You may wonder where all of this noise is coming from. Yes, there are individuals that sincerely are concerned about GMO safety. However, the organic companies (some of them are huge) are helping to push the false argument about risk. If they succeed, they can sell more product and make more money.

This debate is not over. You will soon read about the next step in plant technology – gene editing. Gene editing is a more precise way to alter plant traits.

The leading critics of GMOs are totally inconsistent. First, they support the science on global warming, but ignore the science on GMOs. Next, they pretend to care about the poor, but genetic engineering helps the poor by keeping the cost of food down. The world will not produce enough food without new technology. Finally, if they want to reduce the use of chemicals and energy, GE also does that.

Stay tuned.

John Block was Secretary of the U.S. Department of Agriculture from 1981-1985, where he played a key role in the development of the 1985 Farm Bill.

Clearing Up Confusion: The Net Quantity of Contents Declaration on Food Labels

By Robert A. Hahn

The FDA’s food labeling regulations include some traps for the unwary.  A few of those traps have to do with the net quantity of contents declaration.

Here are a couple of points to keep in mind when declaring a food product’s net contents:

  • When the net contents declaration is expressed in terms of weight, FDA requires that the declaration include the words “Net weight” or the abbreviation “Net Wt.” However, when the net contents is expressed in fluid measure or numerical count, use of the words “Net” or “Net Contents” is optional.  21 C.F.R. § 101.105(j)(3), (n).  NOTE: In 1993, FDA proposed to remove the requirement to include the words “Net Weight” or “Net Wt,” but the proposed rule was withdrawn in 2004.  See 58 Fed. Reg. 29716, 29724 (May 21, 1993) and 69 Fed. Reg. 68831 (Nov. 26, 2004).  FDA’s current position is that the prefatory language “Net weight” or “Net Wt” is required when the net contents is expressed in terms of weight.  The abbreviation “Net Wt” is frequently presented in all upper case letters, e., “NET WT.”
  • Although FDA regulations continue to provide that a separate statement of the net contents in metric measure is optional (21 C.F.R. § 101.105(p)), the fact is that the net contents declaration, with very limited exceptions for random weight packages and for foods packaged in a retail store, must be expressed in both avoirdupois measure (g., ounce, pound, fluid ounce, pint, quart) and metric measure (e.g., milligram, gram, milliliter, liter). The Fair Packaging and Labeling Act (FPLA) was amended in 1992 to require use of metric measure (15 U.S.C. § 1453(a)(2)), but FDA has never amended its regulations to implement this statutory change.  Nevertheless, the FPLA is the law.
  • FDA requires that the net contents declaration use the largest appropriate unit of measure.  For example, 24 oz should be expressed as “1.5 lb,” “1 ½ lb,” or “1 lb 8 oz,” and 1,100 mL should be expressed as “1.1 L.”  Again, the FPLA was amended to require use of the largest appropriate unit of measure, but FDA has not amended its own regulations to reflect the statutory change.  NOTE: “Dual avoirdupois” declaration (i.e., expressing the net contents in avoirdupois measure using both the largest appropriate unit of measure and ounces or fluid ounces, as, for example, “1.5 lb (24 oz) 680 g”) is permitted as an option to facilitate value comparisons by consumers.