Last Wednesday evening (November 30), the House overwhelmingly passed (by a vote of 392-26) a compromise version of the 21st Century Cures Act. House and Senate Republican leaders released a revised draft on November 25, the result of diverse stakeholders' lobbying for or against provisions of an earlier House bill of the same name (which passed the House in July 2015 but then stalled in the Senate). The final version ultimately passed was framed as a House amendment to a Senate amendment to unrelated legislation (H.R. 34), and is slightly shorter but reflects no additional substantive changes to FDA-related provisions.
The 21st Century Cures Act contains provisions that would significantly streamline how the Food and Drug Administration (FDA or the Agency) reviews and approves drugs and medical devices. It would also give FDA US$500 million in new funding with which to help implement various efforts such as improving mental health, fighting opioid addiction, and supporting the Precision Medicine Initiative.
Some of the key medical device FDA-related provisions of the bill that just passed the House, and their regulatory implications, include:
- Expediting the development, and prioritizing FDA review, of "breakthrough" technologies (Section 3051). The bill creates a new accelerated pathway to market for devices that address unmet needs for life-threatening or irreversibly debilitating human diseases or conditions. Importantly, this new pathway builds on FDA's existing Expedited Access Pathway (EAP), but would now be open to more device types – those subject to 510(k) clearance as well as those eligible for PMA approval or de novo requests.
- Expanding the scope of diseases/conditions eligible for a humanitarian device exemption (HDE) (Section 3052). FDA's existing HDE program offers a simplified approval pathway for devices addressing an unmet need for a rarely occurring disease or condition, allowing companies to obtain approval based on safety data and probable benefit. 21st Century Cures makes it possible for companies to obtain an HDE for diseases affecting up to 8,000 people, doubling the ceiling from its prior limit of <4,000. This significant increase would enable more devices to take advantage of this pathway, and could increase patient access to new devices for a greater variety of diseases.
- Encouraging FDA to rely more on real-world evidence to demonstrate device safety and effectiveness (Section 3022). Industry has long pushed for FDA to consider more types of data to help fulfill post-approval requirements or to support premarket clearance/approval of a new indication for a previously cleared/approved device. This past July, the Agency released a draft guidance explaining how and under what circumstances real-world data can support regulatory decision-making in these contexts. By providing a statutory basis for this shift, the 21st Century Cures Act supports FDA recognition of a broader scope of data, which will be particularly beneficial for companies for which the historical gold standard in data collection, prospective clinical trials, is less practical or feasible.
- Requiring additional validation data prior to marketing certain reusable medical devices (Section 3059). Largely fueled by the recent incidents of disease transmission between patients through use of duodenoscopes, 21st Century Cures instructs FDA to specify a list of reusable device types for which 510(k) clearance will require validated instructions for use as well as validation data regarding cleaning, disinfection, and sterilization. This is intended to reduce the likelihood of disease transmission, but it will also require companies whose devices are on the list to provide more comprehensive data for FDA review prior to marketing.
- This section of the bill also obligates FDA to finalize its guidance on when changes to legally marketed devices subject to 510(k) requirements necessitate obtaining a new clearance from FDA by November 2017. The final guidance will supersede the draft guidance issued in August and solidify Agency policy on a topic that is critical for any manufacturer seeking to commercialize a modified or updated version of a previously cleared device.
- Streamlining the review process for combination products (Section 3038). Notably, the bill clarifies the designation of a combination product's "primary mode of action (PMOA)," which is the basis for determining which FDA Center is responsible for its regulation. It significantly reduces ambiguity in this area by specifying that FDA should not deem a combination product to have a drug PMOA based solely on its having a chemical action. Consequently, contrary to FDA's current default position, combination products with device components that have some chemical function but for which that function is not the primary intended purpose1 should be designated as having a device PMOA.
- Making certain types of low-risk software functions not generally subject to regulation as medical devices (Section 3060). The 21st Century Cures Act contains a significant carve-out for medical and certain clinical decision support (CDS) software, namely software functions that are intended (1) for administrative support of a healthcare facility; (2) as non-diagnostic, non-therapeutic mechanisms to encourage a healthy lifestyle; (3) to serve as electronic patient records, except for interpreting patient data for diagnosis/treatment; and (4) for transferring, storing, converting formats, or displaying data/results and associated findings by a healthcare professional (e.g., medical device data systems), unless intended for analysis of data, results, or findings. Still, FDA maintains the authority to regulate any such software function deemed "reasonably likely to have serious adverse health consequences." Notably, this part of the Act would solidify into statute FDA's current regulatory approach, as reflected for example in the Agency's General Wellness and Mobile Medical Applications guidance documents.
- This section of the bill also amends the Federal Food, Drug, and Cosmetic Act to say that accessories are to be classified based on their intended use, notwithstanding the classification of any other device with which they are meant to be used (in line with FDA's January 2015 draft guidance).
- Requiring publication and regular updates on 510(k) exemption (Section 3054). The bill instructs FDA to publish in the Federal Register a notice that lists each type of Class I and Class II device that is to be exempted from the requirement for clearance/approval. The Agency has 90 days after enactment of the legislation to release the first such list for Class I devices, and 120 days to issue the first such list for Class II devices; it is then expected to renew these lists at least every 5 years thereafter. Under this provision, FDA may act more quickly on its proposals to make certain products 510(k)-exempt, which would allow manufacturers of those types of devices to get to market much more quickly.
- Enhancing Institutional Review Board (IRB) flexibility (Section 3056).2 The bill modifies requirements for sponsors of medical device trials related to IRB review to align more with the approach taken for drug trials, specifically acknowledging the possibility of allowing one IRB to oversee a multicenter clinical trial. This change is likely to make clinical trial oversight more efficient by consolidating all IRB functions in one centralized entity when appropriate, as well as to reduce unnecessary expenses and duplication of effort in the conduct of multicenter clinical trials, which have become significantly more common.
- Emphasizing the least burdensome standard for device review (Section 3058). 21st Century Cures clarifies the expectation that FDA reviewers will be trained on, and required to consider, the least burdensome approach for demonstrating a reasonable assurance of safety and effectiveness when requesting additional information from device manufacturers during review of premarket submissions. This includes consideration of the role that post-market information can play in this process. The Act also requires the FDA Ombudsman to audit its implementation. This update, while not dramatic, should reassure industry that the Agency will be held more accountable for performing consistent and reasonable premarket reviews.
- Expanding CLIA Waivers (Section 3057). The bill requires FDA to revise its 2008 guidance on Clinical Laboratory Improvement Amendment (CLIA) waivers for in vitro diagnostic devices to replace a gold-standard-based accuracy requirement with a demonstration of accuracy through comparable performance between waived and moderate-complexity laboratory users. This provision will make it easier to show that certain IVD tests present a low risk of error and thus can be appropriately exempted from routine inspections and most requirements under CLIA.
- Accelerating approval of regenerative advanced therapies (Section 3034). In line with recent pressure to enable making the benefits of stem cell therapies available to more patients, 21st Century Cures calls for devices used in the recovery, isolation, or delivery of a stem cell product to be deemed moderate risk unless the Agency determines that the device or its use should be classified as higher risk.
- In addition, while not specific to FDA, the following additional pieces of the 21st Century Cures Act may also be of interest to the medical device industry:
- The bill establishes a new Medicare Pharmaceutical and Technology ombudsman within CMS whose task will be to address complaints/requests raised by drug and device manufacturers related to the coverage, coding, and reimbursement of medical technologies.
- To help fund the bill, Medicaid reimbursement to states for durable medical equipment (DME) will be limited to Medicare competitively bid rates by January 1, 2018 (instead of 2019).
- Payments for infusion drugs provided through DME will be reduced starting in 2017, pursuant to a finding that such payments have often been excessive.
- Medicare coverage will be expanded to include support for telehealth services, including remote monitoring.
Following House passage, the Senate voted overwhelmingly (85-13) on Monday afternoon to end debate on the bill and proceed to a vote on its substance. The Senate is expected to pass the legislation tomorrow. The bill has faced a more uphill battle there than it did in the House; among its most vocal opponents is Senator Elizabeth Warren (D-MA), who maintains that it will effectively result in lower standards for drug/device safety and effectiveness. Nevertheless, the Act is now expected to pass in the Senate as well and then to be sent to President Obama to be signed into law later this week.
President-elect Donald Trump has said he will work to eliminate "red tape" at the FDA but has not commented on the 21st Century Cures Act specifically. It remains to be seen how the Trump administration will interpret the review and clearance/approval procedures for medical devices as modified by this legislation and, more broadly, how much it will seek to influence FDA policy.