Health Policy Report (11/1)

Capitol Hill Update

Both chambers of Congress cleared another short-term funding measure for federal surface transportation programs as Democrats again failed to reach consensus on a path forward for President Joe Biden’s legislative agenda. Despite renewed optimism that the bipartisan infrastructure deal would pass last week, Democratic lawmakers did not fully coalesce behind an updated framework for the President’s Build Back Better Act (BBBA) — a prerequisite for progressives’ support for the Senate-passed infrastructure deal. With a new surface transportation deadline of December 3, Democrats have effectively reset the “shot clock” on floor timing for both the Senate-passed infrastructure deal and reconciliation package — thus providing additional wiggle room for last-minute negotiations and tweaks. 

Despite punting the surface transportation deadline, Democratic leadership is pushing to finalize legislative text of the BBBA as soon as today. Votes on both the reconciliation package and infrastructure deal could occur as early as Tuesday if there is enough buy-in from the totality of the Democratic caucus, although that date appears increasingly in doubt. Additionally, reports out of Capitol Hill suggest that a provision on prescription drug price reform could get added in after House and Senate Democrats spent the weekend deliberating a package that would include drug pricing negotiation, a Medicare Part D redesign, and rebates.

New Reconciliation Framework Includes Pared Back Health Provisions

On Thursday morning, President Joe Biden released a framework (fact sheet; TRP analysis) for the Democrats’ $1.75 trillion reconciliation package. After months of negotiations between progressive and moderate Democrats, the package has been whittled down from $3.5 trillion in an effort to garner support from centrists. The framework notably does not include most of the initial package’s drug pricing provisions, though details on whether any such policies will eventually be included have yet to be revealed. Ultimately, the framework is still subject to negotiation within the party ranks, though the administration has expressed confidence that this outline can pass both the House and Senate. 

In September, House Democrats on the Ways & Means and Energy & Commerce Committees introduced (H.R. 5376) a $3.5 trillion reconciliation bill called the Build Back Better Act (BBBA). The House never brought the bill to the floor for a vote due to intraparty disagreements. Since then, congressional Democrats and the White House have been in fierce negotiations to hammer out a compromise deal. In particular, centrists Sens. Joe Manchin (D-WV) and Kyrsten Sinema (D-AZ) have been spearheading cuts to the House-approved language, clashing with progressive lawmakers who have pushed for a more ambitious reconciliation package.   

It remains to be seen whether there will be buy-in from the totality of the Democratic caucus given some of the omissions in the final package. Notably, the updated framework does not include a paid family and medical leave provision nor drug pricing provisions, which is drawing the ire of progressive Democrats who have pushed for a more ambitious size and scope for many of the reconciliation priorities. The House Rules Committee met later on Thursday and released preliminary text (section-by-section) of the package. 

HHS Set to Cut Trump-Era Oversight Rule

On Thursday, the Department of Health and Human Services (HHS) issued a proposed rule which seeks to repeal the Trump-era final rule entitled, “Securing Updated and Necessary Statutory Evaluations Timely” (SUNSET final rule) published on January 19, 2021. Under the SUNSET final rule, all HHS regulations — subject to certain exceptions — are rendered ineffective: (1) two calendar years after the year that the SUNSET rule first became effective; (2) ten calendar years after the year of the regulation’s publication; or (3) ten calendar years after the last year in which HHS conducted a detailed review or assessment of that regulation. The SUNSET final rule provides that HHS must amend or rescind a regulation within a specified timeframe if necessitated by a detailed review or assessment of that regulation and requires that these reviews and assessments be posted on both the Federal Register and the HHS website with an opportunity for public comment.

On March 19, 2021, HHS postponed implementation of the SUNSET final rule from March 22, 2021, to March 22, 2022, due to pending litigation. Today’s proposed rule comes as a result of concerns raised by stakeholders regarding the validity of its provisions as well as concerns from HHS regarding its implementation.  

In Thursday’s proposed rule to rescind the SUNSET final rule, HHS claims that implementation of the SUNSET final rule would result in an administrative burden on the Department and its related agencies, thus diverting resources away from critical public health matters. HHS stipulated that the automatic expiration of its regulations holds the potential for public harm and that the sudden expiration of regulations would also potentially harm small entities by creating an undue and disproportionate burden. Further, HHS claimed that the definitions and exceptions in the final rule are ambiguous, therefore risking the automatic expiration of many regulations as a result. Stakeholder comments raised questions surrounding the final rule and its consistency with the Administrative Procedure Act (APA), which HHS claimed merit further consideration. 

HHS determined that repealing the SUNSET final rule would generate cost savings for both the Department and the public due to a reduction in administrative burden. Further, the proposed rule would reduce any regulatory uncertainty from the potential automatic expiration of rules. HHS determined that the SUNSET final rule should be entirely withdrawn, but also requested comment on whether it should consider modifying the final rule, instead. Stakeholders will have 60 days to submit comments on the proposed rule once it is published in the Federal Register, which is expected to take place on October 29, 2021. 

NIH, FDA Build Coalition to Target Treatments for Rare Diseases

On Wednesday, the Food and Drug Administration (FDA) and National Institutes of Health (NIH) joined forces with ten pharmaceutical companies and five non-profits in an effort to expedite gene therapies for rare disease patients. The new Bespoke Gene Therapy Consortium (BGTC) is part of the NIH’s Accelerating Medicines Partnership (AMP) program and will receive $76 million from NIH and its private partners over five years to execute its mission. Roughly $39.5 million will come from NIH and its partner agencies, and the National Center for Advancing Translational Sciences (NCATS) will contribute $8 million. NCATS is the lead NIH partner on the project, and it developed the Platform Vector Gene Therapy program.

The NIH highlighted the necessity of the program, citing that only two heritable diseases have FDA-approved gene therapies, out of about 7,000 rare diseases. The agency asserts that cultivating a standard therapeutic development model to include gene delivery technology can save time and money throughout the development process, as developing rare diseases therapies is notoriously cumbersome and expensive. Additionally, patchwork regulatory standards and limited access to tools and technology create additional hurdles for developers. These roadblocks compound inherent problems with incentivizing rare disease treatment initiatives, such as the small market for the products. The BGTC will also work to streamline the approval process for promising therapies, including clarification of preclinical testing guidelines. 

A standardized development model with a common gene delivery vector can aid in alleviating many development issues. One of BGTC’s key goals will be to break down the adeno-associated virus (AAV), a common gene vector, to help developers understand how to produce AAV and enhance its clinical benefit. On the clinical side of the project, research will fund about five clinical trials focused on different rare diseases that already have a strong foundation to initiate clinical studies but do not have therapies in development.

HHS Unveils Plan to Address Drug Overdoses

The Department of Health and Human Services (HHS) announced a new drug overdose prevention plan last week. The Overdose Prevention Strategy consists of a four-pronged approach to tackle: (1) primary prevention; (2) harm reduction; (3) evidence-based treatment; and (4) recovery support. The timing of this unveiling comes on the heels of a report which found that more than 840,000 people died of an overdose between 1999 and 2019. Notably, 93,000 of these deaths took place during the COVID-19 pandemic. HHS noted that addressing overdoses is a “top priority” for the agency. 

To improve harm reduction, the administration plans to focus on developing testing strips that detect fentanyl, as well as facilitating safe syringe practices and increasing access to naloxone — an overdose reversal drug. HHS’s plans for prevention include targeted advancements in pain management procedures to mitigate “clinically inappropriate” treatment regimens. So far, these tools include a focus on the Center for Disease Control’s (CDC’s) opioid prescribing guidelines and Substance Abuse and Mental Health Services Administration’s (SAMHSA’s) new Strategic Prevention Framework for Prescription Drugs (SPF Rx). Improving access to treatment and recovery is also at the forefront of the program’s goals. Parallel to HHS’s efforts, the CDC announced a new educational campaign to reduce opioid deaths that will target 18-to 34-year-olds.