Administration

How Is ARPA-H Shaping Up?

by Matt Gontarchick

The Advanced Research Projects Agency for Health (APRA-H) has come a long way since the Biden administration first proposed a new biomedical research agency back in April 2021.  Since then, Congress and the administration have started laying the foundation for the new agency.  The Fiscal Year (FY) 2022 appropriations omnibus provided $1 billion in funding for the nascent agency and directed the president to appoint an ARPA-H director.  In congressional hearings last spring on the administration’s FY 2023 budget request for the Department of Health and Human Services (HHS), Secretary Xavier Becerra offered a few more details, like the administration’s preference to make ARPA-H a part of the National Institutes of Health (NIH).

There are still more questions than answers about what ARPA-H could one day look like.  However, more details have come into focus in recent weeks as authorizing legislation for ARPA-H progresses through Congress and the administration makes new decisions about ARPA-H personnel.

A New Acting Deputy Director

While the search for a permanent director remains under way, the administration has at least made headway in naming a temporary member of the new agency’s leadership team.  On May 25, HHS Secretary Becerra appointed Adam H. Russell, DPhil, to be ARPA-H’s acting deputy director.  With experience managing research projects at the Defense Advanced Research Projects Agency (DARPA), Russell is tasked with building out the administrative structure of the new agency and hiring initial staff until the president appoints a permanent director.  It remains unclear if Russell is under consideration for a permanent role either as director or deputy director.

The ARPA-H Director: Appointment or Confirmation?

When the administration finally taps someone to permanently lead ARPA-H, will the appointment require Senate confirmation?  Recently, Rep. Anna Eshoo (D-CA) revised her ARPA-H authorizing legislation to require the Senate to confirm the ARPA-H director.  The Senate’s legislation on ARPA-H and previous versions of Eshoo’s bill did not address whether the individual appointed by the president would require confirmation.  The House is set to vote on the bill  during the week of June 20, while a Senate panel already approved its ARPA-H bill in March.

The Continued Debate over Placement within NIH

To date, the question of whether APRA-H should be an independent agency within HHS or a part of the organizational structure of the National Institutes of Health (NIH) remains unsettled.  The Senate’s legislation aligns with the administration’s preference of placing the new agency within NIH, which Becerra explained over multiple congressional hearings would allow ARPA-H to focus on developing breakthroughs from day one by delegating administrative functions to the parent agency.  However, Eshoo and other lawmakers have been firm in their stance that ARPA-H must be independent to successfully carry out its mission, and her bill maintains this position. 

Where Will the ARPA-H Headquarters Be?

While certain lawmakers and the administration have yet to agree on whether the Senate should confirm the ARPA-H director and the placement of e the organization, there is a universal agreement that the new biomedical research agency should not be physically located on the NIH campus in Bethesda, Maryland.  Over the past few months, several states have been jockeying to be selected as the home base for the new agency.   Members of the Texas and Massachusetts congressional delegations have recently sent letters to the administration urging their states to be considered as the location for ARPA-H due to their existing infrastructure in medical research and life sciences. 

In Massachusetts, business and academic leaders have joined public officials on efforts to lure ARPA-H to the Bay State.  Several members of the Ohio congressional delegation are also urging the administration to select Cleveland as the new ARPA-H home, and other states vying for the headquarters include California, North Carolina, and Maryland.  However, the administration has yet to announce a timeline for its headquarters selection process or provide any details on how a location will be chosen. 

When Will We Know More about ARPA-H?

Once the House votes on ARPA-H legislation during the week of June 20, stakeholders will have to keep an eye on the Senate.   While minor differences around the provision of whether the Senate confirms   the ARPA-H director seem reconcilable, other issues like whether to place ARPA-H within NIH will continue to garner significant debate.  Until a final agreement can be reached, further details on ARPA-H will probably be sparse, as the administration will likely hold off on major announcements on items like personnel and a headquarters location until legislation is close to the finish line. 

Congress

FDA User Fee Process: The Clock Is Ticking

by Matt Gontarchick

September 30, 2022.  That’s the date the Food and Drug Administration’s (FDA) user fee programs for drug products and medical devices are set to expire.  Given the “must-pass” nature of user fee reauthorization, lawmakers and FDA stakeholders are using this moment to advance other FDA-related reforms pertaining to the accelerated approval process, promoting clinical trial diversity, competition in drug markets, and other areas.  While the House and Senate user fee packages are fundamentally similar, there are some differences between the two.  For instance, the Senate package includes policies related to dietary supplements, diagnostic labs, and cosmetics; the House does not.  Given the differences between the packages and the pressure to get a signed bill before the August recess, stakeholders would be keen to watch the legislative process unfold.

A Brief History of the FDA User Fee Process

Enacted in 1992, the Prescription Drug User Fee Act (PDUFA) authorized the FDA to collect user fees from companies that submit applications for drug products.  Since then, Congress has added additional user fee programs for other products like medical devices, generic drugs, and biosimilars.  Congress reauthorizes user fee bills every five years, and the reauthorization process marks an important opportunity for lawmakers to consider FDA-related policies as well as overall FDA performance. 

Since FDA depends on user fees to operate, failure to reauthorize user fee programs would catastrophically disrupt FDA operations by triggering massive layoffs of career staff who review new drugs and devices and monitor drug safety.   As a result, the timelines for new drug products to reach patients and providers would increase exponentially.   

Although current authority for the user fee programs expires at the end of Fiscal Year (FY) 2022, Congress typically passes reauthorization bills in the summer preceding the September deadline to avoid disrupting FDA operations. With that in mind, the House and Senate are on the clock to pass a legislative package over the next couple of months before the FDA starts handing out pink slips.

What’s in the House and Senate Bills?

The House Energy and Commerce Committee and the Senate Health, Education, Labor, and Pensions (HELP) Committee have already released their own respective user fee reauthorization bills that would reauthorize fees that drugs, biosimilars, and medical device companies must pay the agency for review of their products.  Both bills appear to move forward with fee levels that were negotiated between FDA and industry groups prior to their release. 

Additionally, both bills set out to reform the FDA’s accelerated approval program, which allow the agency to approve drugs more quickly for serious conditions that fill an unmet medical need.  In exchange, drug manufacturers must complete post-approval trials to confirm those clinical benefits – something the FDA has not consistently ensured. 

Both the House and Senate bills would allow the FDA to require manufacturers to start post-approval studies prior to accelerated approvals, give the FDA more control over study design and deadlines, and create a mechanism for the agency to pull drugs off the market.  The bills also would clarify that post-approval studies could rely on real-world evidence (RWE) to confirm that drugs are effective.  Notably, the Senate bill differs from the House bill in that it would create an interagency coordinating council that would periodically review accelerated approvals.

Additionally, both bills set out to promote drug competition through better access to generic and biosimilar drugs, albeit through different paths.  For instance, the House bill contains provisions that would streamline the generic approval process in certain types of cases, while the Senate bill addresses exclusivity for interchangeable biosimilar products.

Outside of fee proposals, changes to the accelerated approval process, and access to generic and biosimilar drugs, both bills contain several notable differences.  For example, the House bill includes provisions aimed at improving diversity in clinical trials by requiring trial sponsors to submit diversity action plans with details on how the sponsors intend to enroll a diverse group of trial participants.  Other provisions would require the FDA to submit reports on diversity action plans and to hold public workshops on improving trial diversity. While the Senate bill does not contain such a provision, another Senate HELP Committee-approved bipartisan bill, the PREVENT Pandemics Act, does include policy intended to promote clinical trial diversity.

Among the Senate’s user fee package proposals are provisions that would enhance the regulation of laboratory-developed tests (LDTs) by allowing the FDA to regulate LDTs based on the level of risk associated with them, similar to how the agency regulates medical devices.  The Senate bill would also expand the FDA’s ability to regulate dietary products by requiring dietary supplement companies to list their products with the agency, which would theoretically improve the FDA’s ability to enforce consumer protections.  Furthermore, the Senate bill contains a slew of provisions aimed at boosting regulation of cosmetics.  These include requirements for cosmetics manufacturers to register their products with the FDA, new labeling requirements, and the establishment of good manufacturing practice regulations.

What Comes Next?

The House Energy and Commerce Committee voted in favor of its user fee package in the Health Subcommittee on May 11 and the full committee on May 18 with a vote on the House floor scheduled for June 7.   Over in the Senate, the HELP Committee is scheduled to mark up its user fee package on June 14, along with legislation to improve access to mobile health clinics. 

Congress has always addressed the user fee reauthorization legislation ahead of the expiration deadline (September 30), and leaders from both House and Senate committees with jurisdiction over the FDA have publicly stated that they don’t want to hold up the reauthorization process.  However, substantive differences between the House and Senate versions of the reauthorization bill combined with a highly partisan climate have the potential make passing user fee legislation more difficult than in previous years. 

Additionally, the Senate HELP Committee has also voted in favor of its PREVENT Pandemics Act, another top priority of Chair Patty Murray (D-WA) and Ranking Member Richard Burr (R-NC), who is retiring at the end of this year, for enactment.  Likewise, a priority of House Energy and Commerce Health Subcommittee Chair Anna Eshoo (D-CA) will be enactment of her bipartisan House legislation creating the Advanced Research Project Agency for Health (ARPA-H).  It remains unclear if/how these policy priorities and user fee reauthorization packages will come together and form one bill to be signed by the President before the expiration of the current user fee programs. As the summer unfolds, stakeholders would keen to monitor the reconciliation process to see which provisions and changes make it into the final reauthorization bill.

Congress

What Lawmakers Talked About during the HHS FY23 Budget Hearings

by Matt Gontarchick

Xavier Becerra made the rounds on Capitol Hill recently in his capacity as Secretary of Health and Human Services (HHS) to testify on the Biden administration’s Fiscal Year (FY) 2023 budget request for HHS.  Beyond the secretary’s submitted testimony – which was virtually the same for every hearing – here is a countdown of the top five health-related topics on lawmakers’ minds that were discussed across seven congressional hearings on the HHS budget request.

5. Ending the PHE

In a few hearings, lawmakers questioned Becerra on when the administration plans to unwind the COVID-19 public health emergency (PHE), which is currently set to expire on July 16, 2022.  Each and every time, Becerra reiterated the administration’s commitment to providing 60 days’ before ending the PHE in order to give states and health care providers time to prepare.  In recent weeks, the administration has given no indication that it will simply let the current PHE expire in two months.  Since May 16, 2022 marked exactly 60 days before the current PHE expiration date, the administration is all but certain to renew the PHE for another 90-day period come July.

4. Telehealth Waivers

One reason why lawmakers are so interested in how long the PHE will last is because several emergency health care flexibilities are tied to the end of the PHE.  These flexibilities include several Medicare telehealth waivers that waive geographic site originating requirements and allow coverage of audio-only services, among other items.  During the hearings, Becerra repeatedly thanked lawmakers extending temporary telehealth waivers for 151 days – 5 months –  beyond the end of the PHE in the Fiscal Year (FY) 2022 omnibus. 

Members were also strongly supportive of extending at least some of the telehealth waivers permanently. Some of the telehealth benefits lawmakers cited include increased access to health care in rural areas and relief for the health care workforce shortage.  Becerra voiced agreements on telehealth’s many benefits during the hearings, and he urged lawmakers to work with the administration on developing legislation to make the temporary telehealth authorities permanent.  Fortunately, members are already hard at work and a bipartisan group of House members have already introduced legislation to permanently expand coverage of audio-only telehealth and remove geographic restrictions on originating sites.

3. No Surprises Act

Members from both parties across multiple committees criticized the secretary for not following congressional intent in implementing the No Surprises Act because the rulemaking process establishes the Qualifying Payment Amount (QPA) as the presumptive out-of-network rate in the independent dispute resolution (IDR) process.  They argued that doing so tips the scale in favor of insurers during the IDR process. During the hearing, authors of the No Surprises Act like House Ways and Means Committee Chairman Richard Neal (D-MA) said members went to great lengths to ensure that the legislation established a level playing for all factors to be considered in the IDR process.   

In February 2022, a district court ruling struck down provisions of the No Surprises Act that gave weight to the QPA.  Becerra assured members during the hearings that the administration’s final rule on the No Surprises Act will heed the court’s ruling, although he declined to provide a timeline  on when  the rule will be released.  The secretary also said that HHS is working with the Department of Justice (DOJ) on whether to appeal or accept the ruling.  Additionally, Becerra was confident that the new rule will protect patients from surprise medical bills. 

2. 988 Suicide Hotline

988 will officially become the new suicide hotline on July 16, 2022, and members were curious to see how the administration is ensuring the hotline will work from day one.  States and territories will be responsible for operating the actual hotline, and Becerra explained that the administration is providing states with funding to make sure they can onboard enough counselors and behavioral health professionals to take incoming calls.  In the event that existing state call centers are overwhelmed, Becerra told lawmakers that HHS is working to set-up back-up call centers.  As the launch date approaches, Becerra assured the congressional committees that HHS is “keeping tabs” with states on call center preparation, although he noted that some states will be in a better position than others to take calls right away once the new hotline goes live this summer.

1. ARPA-H

The FY 2022 omnibus established the Advanced Research Projects Agency for Health (ARPA-H) as the newest biomedical research agency within HHS.  While members were unanimous in their support of the new agency, some including Reps. Rosa DeLauro (D-CT) and Anna Eshoo (D-CA) were critical of the administration’s decision to house ARPA-H within the organizational structure of the National Institutes of Health (NIH).  DeLauro, Eshoo, and others had been advocating for ARPA-H to be an independent agency within HHS because they felt this arrangement would help cultivate an independent culture within the new agency that would best facilitate the development of new cures.  Additionally, some members including Sen. Jerry Moran (R-KS) and Rep. Tom Cole (R-OK) were concerned that the administration’s was proposing additional FY 2023 funding for ARPA-H at the expense of additional funding for existing research projects at NIH.

Becerra assured lawmakers that the placement of ARPA-H was purely administrative, and he explained that having NIH assume functions like accounting and human resources would allow ARPA-H to focus on developing breakthroughs from the get-go.  To further point to the new agency’s independence, he said the ARPA-H director would report to the secretary – not the NIH director – and he added that ARPA-H would not be “physically housed” within the NIH campus. 

Becerra additionally told the congressional panels that ARPA-H will be operational once a director is appointed.  While the secretary said the search for a director is currently underway, he was unable to provide a timeline.