Earlier this week, the Senate Health, Education, Labor, and Pensions (HELP) Committee held a hearing on a package of legislative measures that are targeted at facilitating medical innovation. This hearing, the second in a set of three, is the Senate's response to the House-passed 21st Century Cures Act (H.R. 6). We wrote about the first hearing, and a recap can be found here.
While the second meeting featured much partisan debate and fanfare, the Committee advanced all seven medical innovation measures before them, with six passing by voice and one measure passing by roll call vote of 20-2.
In opening statements, Senator Lamar Alexander, Committee Chairman, acknowledged the anticipated contention on the panel over what he referred to as a "surge" of additional mandatory funding requested by Democrats for the Food and Drug Administration (FDA) and the National Institutes of Health (NIH). Even expecting contention, the Chairman expressed optimism about the Committee's capacity for compromise, stating that if the Committee successfully creates an innovation agenda, it may be the "most important legislation passed this year."
As may be expected, Ranking Member Senator Patty Murray and Senator Elizabeth Warren both showed strong support for increasing mandatory funding for the FDA and the NIH, each introducing an amendment to S. 1878: one to create a medical device evaluation system and the other to create a biomedical innovation fund. Senator Murray also expressed concern that some of these bills may increase FDA responsibilities and not compensate the FDA for that. While both senators started out firmly believing that any agreement needed to include increased funding, they both withdrew their amendments, including any requests for additional funding, in order to find other consensus points and come to an agreement.
The measures approved in the Committee are as follows:
This bill amends the Federal Food, Drug, and Cosmetic Act (FDCA) to expand the priority review voucher program for rare pediatric diseases to including treatments for both sickle cell disease and pediatric cancers. The voucher program will be extended by removing the provision that terminates the program one year after the FDA's issuance of three rare pediatric disease vouchers. However, a voucher may not be issued for a rare pediatric disease product, if a voucher was ever issued for the product as a tropical disease product. Additionally, a drug sponsor that intends to request a voucher for a rare pediatric disease drug must notify the FDA of their intent to do so upon submission of their new drug application.
The bill's sponsor, Senator Robert Casey explained that this program is integral to fostering innovation and incentivizing private companies to invest in rare children's diseases. Such an incentive is necessary because private companies tend to not see a return on research investment in this area.
This is the one measure passed via a roll call, following adoption of the following amendments: (1) specify the criteria for "rare pediatric disease," (2) insert a section related to rare pediatric disease product applications, (3) require the comptroller to conduct a study on the GAO report on the effectiveness of awarding priority review vouchers for the sponsorship of rare pediatric disease product application, and (4) require the results of the study to be reported to congress.
This bill amends the FDCA to expand the FDA's priority review of breakthrough medical devise to include all classes of devices. Currently, only new or highest risk (Class III) devices are eligible. However, this bill would make it so that upon a sponsor's request, the FDA would be forced to determine whether a device meets the criteria for priority review as a breakthrough device.
To expedite the development and review of designated medical devices, the FDA must: "assign a team of staff for each device, adopt an efficient process for dispute resolution, provide for interactive and timely communication with the device sponsor, expedite review of manufacturing and quality systems compliance, disclose to the sponsor in advance the topics of any consultation between the FDA and external experts or an advisory committee and provide the sponsor the opportunity to recommend external experts, [and] assign staff to address questions by institutional review committees concerning investigational use of the device."
Senator Richard Burr emphasized the idea that this measure would not change the standards that devices are required to meet in order to reach the market, but instead simply changes the approach by which device approvals can be accelerated.
This bill encourages the development of medical countermeasures in case of biological attacks and naturally occurring outbreaks. This bill is intended to build upon the Pandemic and All-Hazards Preparedness Act, by leveraging the innovation of the private sector through priority review vouchers. The urgency of this problem has been exemplified with the Ebola and Zika outbreaks over the past year.
This bill amends the FCPA to exclude several classes of devices from FDA-regulation as a medical device. Some of the included classes are: administrative, operational, or financial records software used in health care settings; software for maintaining or encouraging a healthy lifestyle unrelated to medical treatment; electronic patient records, excluding software for interpreting or analyzing medical image data; software for clinical laboratory testing, excluding software for interpreting or analyzing test data; and software that provides medical recommendations and the basis for those recommendations to health care professionals, excluding software for acquiring, processing, or analyzing medical images or signals.
The bill also states that the FDA must classify a medical device accessory according to its intended function, not the classification of the medical device with which it is used.
This measure was adopted without any discussion.
This bill attempts to address regulations on combination drug and device products. There was little discussion accompanying this bill, but Senator Casey did briefly speak out in favor of the measure, noting that it will allow for improved communication with the FDA and manufacturers.
This bill amends the FDCA to require the package of information published by the FDA upon approval of a new drug to include documentation of efforts to assess patient engagement. The required documentation must include identification of patient-focused drug development tools and an explanation of whether certain information was reviewed or examined, including any patient preferences and patient-reported or caregiver-reported outcomes. The FDA is also required to annually summarize, and publish, the data that is collected in this documentation.
Senator Susan Collins, a cosponsor of the bill, believes that patient input is a valuable resource and should play a larger role in FDA decision-making.
This bill also amends the FDCA, to add the Zika virus to the list of tropical diseases under the priority review voucher program. The priority review voucher program awards a voucher to the sponsor of a new drug or a new biological product that is approved to prevent or treat a tropical disease, which allows the holder of the voucher to have a future new drug or biological product application acted upon by the FDA within six months.
Chairman Alexander mentioned at the outset of this meeting that the Zika virus is a top concern and priority for the HELP panel.
The final HELP Committee on medical innovation is slated for April 6, 2016, at which time Chairman Alexander hopes to have a final package ready for floor consideration.