Congress Introduces the PATIENTS’ FDA Act to Increase Transparency, Efficiency, and Predictability

By Jennifer D. Newberger –

On Tuesday, April 17, 2012, Senators Burr and Coburn introduced the Promoting Accountability, Transparency, Innovation, Efficiency, and Timeliness at FDA Act, or the “PATIENTS’ FDA Act.”  This bill is intended to complement the proposed user fee agreements negotiated between FDA and the drug and device industries by “requiring transparency in the review and decision progress against the initiatives and metrics agreed to in the prescription drug, generic drug, biosimilar, and medical device user fee agreements.”

If enacted, this bill could address many of the concerns raised by sponsors about FDA’s review and approval process for medical products, particularly medical devices.  Though the bill applies generally to all medical products, many of the provisions relate specifically to improving the review process for medical devices by making it more predictable, holding FDA more accountable for its decisions, and allowing industry more insight into how FDA reaches a final decision.

Some highlights of the bill include the following: 

Development of “a strategy and implementation plan for advancing regulatory science for medical products in order to promote the public health and advance innovation in regulatory decisionmaking,” and provision of reports to Congress on its progress in implementing the plan.

Documentation of the “scientific and regulatory rationale for any significant decision” made by CDER, CBER, or CDRH.  Perhaps most importantly, FDA would be required to provide the rationale to the sponsor of the submission resulting in the decision if requested.  This is intended to respond to concerns by industry that reviewers often request large amounts of “additional information about a drug or device that is beyond the scope of data needed to meet the FDA’s approval standard.”  By documenting the scientific and regulatory rationale and making it available to sponsors upon request, Congress hopes to increase transparency of the review process.

To assess the safety and effectiveness of a device “from the perspective of a reasonable patient in the intended use population who would assign the most value to the effect the device purports to have or is represented to have under the conditions of use prescribed, recommended, or suggested in the labeling or proposed labeling, and who would be willing to accept the probable risks that may be associated with the use of the device as prescribed, recommended, or suggested in the labeling or proposed labeling.” This language reflects the position taken by FDA in its recently issued guidance, “Factors to Consider When Making Benefit-Risk Determinations in Medical Device Premarket Approval and De Novo Classifications,” on which we posted previously. 

To work with regulatory counterparts in other countries to reduce duplicative clinical or non-clinical studies and facilitate the acceptance of foreign clinical data.

For sponsors intending to investigate the safety or effectiveness of a class II or III device that has a valid marketing authorization in specified countries, and has no record of device-related serious unanticipated adverse event reports, FDA “shall permit an exemption for clinical testing of such device for the purpose of developing data to obtain clearance or approval for the commercial distribution of such device.”  It will be interesting to see whether FDA interprets the word “shall” to mean that it must grant the exemption for clinical testing, or whether it will allow only discretionary exemptions.

FDA’s Office of Chief Counsel (OCC) would be required to review warning and untitled letters before being issued.

The bill would require FDA to assign “a reviewer with prior review experience with that type of device or technology or other relevant expertise.”  Though it seems something this basic would not require statutory authority, this seems intended to address industry concerns that reviewers often do not have enough experience with the particular type of devices they are responsible for reviewing, leading to the request for information beyond what is necessary to make an assessment of safety and effectiveness.

FDA shall publish detailed decision summaries for each 510(k) clearance.  The Office of In Vitro Diagnostics currently does so, and under this requirement, the Office of Device Evaluation would be required to do so as well.  This would provide additional insight to industry about the criteria used to clear a device, and hopefully provide increased transparency about the clearance process.

Undoubtedly in response to industry criticism of a draft guidance FDA released in July 2011 regarding when a company must submit a new 510(k) notification for a modification to an existing device, discussed here, this bill would clarify when a new 510(k) is not required.  Specifically, it states that no 510(k) notification is required if the change is “validated by [the] same method applied to the classified device undergoing modification, or a current validation method that is equivalent to the validation method applied to the device undergoing modification.”  This addresses the concern that the draft guidance essentially states that validation and verification will be insufficient to demonstrate that a modification does not require a new 510(k) notification.

The bill, if implemented (and actually acted on by FDA), could address many industry concerns about the predictability, transparency, and consistency of FDA actions, particularly with regard to medical devices.