The PIE Act: A Case Study in the Importance of Persistence in Policymaking
In the world of health care innovation, speed matters. Cutting-edge treatments and therapeutics often represent the best chance for patients facing aggressive diseases and chronic conditions with few available options. But when it comes to developing these pharmaceuticals and devices safely, a cautious and methodical approach is vital as well. Safety matters as much as speed.
The good news is that faster doesn’t always mean riskier. For decades, drug manufacturers needed to wait until Food and Drug Administration (FDA) approval to start sharing information about new treatments with payers, which delayed access to coverage. With the passage of the Pre-approval Information Exchange (PIE) Act in December 2022, payers can now review treatments concurrently with the FDA’s review. That means patients across our country will be able to access potentially life-saving treatments more quickly.
The process that culminated in President Biden’s signature involved years of determination and teamwork by a wide range of stakeholders. As a result, we thought it useful to look back and share how the push for PIE started, how the effort evolved, and why we fought so hard to make it the law of the land.
PIE’s story starts in 1997 when Congress passed the FDA Modernization Act (FDAMA), which sought to create a regulatory safe harbor to increase the dissemination of health care economic information (HCEI) to payers responsible for formulary decision-making. Although well-intended, the statute included vague wording that hampered actual information sharing.
Years passed with no progress until a breakthrough in 2016. That year, AMCP hosted two forums with solution-minded professionals that provided the spark needed to advance PIE. In March, AMCP held a Partnership Forum on FDAMA 114 focused on barriers to HCEI exchange. During an unplanned discussion at that session, the idea arose that while it was critical to ensure the exchange of economic information for approved drugs, having this information for products in the approval pipeline could be a game changer. Six months later, AMCP hosted a session focused specifically on this idea, during which the term “Pre-approval Information Exchange” was coined.
AMCP quickly mobilized to engage those who could help make this vision a reality, notably members of Congress and their staffs. In late 2016, AMCP conducted well-attended briefings with House and Senate offices about the importance of proactive manufacturer-payer communications, particularly pre-approval.
In 2017, PIE found a forceful advocate in Representative Brett Guthrie from Kentucky, an influential member of the House Energy and Commerce (E&C) Committee. In April, he introduced H.R. 2026, the Pharmaceutical Information Exchange Act of 2017, and in July, the E&C Committee held a hearing on the issue. The Act passed out of the E&C Health Subcommittee in January 2018, marking the next step in a long, patient journey to becoming law. Rep. Guthrie’s commitment to PIE would prove essential in years to come.
While Congress was warming up to PIE, the FDA tried to clarify some regulatory ambiguity. In January 2017, the FDA issued draft guidance about manufacturer communications with payers and formularies that referenced pre-approval communications, but the draft was not explicit in the details. In June 2018, the FDA finalized its guidance for payer-manufacturer communication and communication consistent with labeling. AMCP tried to encourage broader adoption of PIE by releasing its Format for Formulary Submissions, v. 4.1, which aligned with the 2018 Final Guidance and included recommendations for preapproval dossiers and dossiers for unapproved indications for approved drugs. Unfortunately, confusion remained about what kind of information could be shared.
Conclusive action needed to come through federal legislation — a process that gathered momentum in 2022. AMCP began working closely with Congress and generated support from other dedicated health care organizations to ensure that any proposed legislation would clarify the scope of PIE and resolve inconsistencies between the guidance and relevant statutory law. Rep. Guthrie remained PIE’s steadfast champion, reintroducing the PIE ACT in March 2022 as H.R. 7008.
This is when the diligent groundwork of engagement and education proved crucial in raising awareness and building bipartisan support for PIE. This past March, AMCP led a diverse coalition of stakeholders in American health care in drafting a letter supporting the PIE Act. It laid out in clear, powerful language why the PIE Act was essential, making the case that lives were on the line. This was part of a broader Congressional engagement, including a March 17 hearing entitled “The Future of Medicine: Legislation to Encourage Innovation and Improve Oversight” and a May 11 markup of various pieces of legislation, including PIE. With this push, PIE cleared an important hurdle and found itself on a short list of bills that would be considered for passage by the full House of Representatives.
Indeed, after receiving and incorporating technical assistance from the FDA, the PIE Act was included as Section 810 of the “must-pass” bipartisan Food and Drug Amendments of 2022 (H.R. 7667), which passed the House on June 8th. As part of this process, AMCP assembled a large coalition of health care organizations to sign a pro-PIE Act letter to the leadership of the House E&C Committee. AMCP then followed up with a letter to the leadership of the Senate’s Health, Education, Labor, and Pensions (HELP) Committee, providing feedback in response to the discussion draft of the Food and Drug Administration Safety and Landmark Advancements (FDASLA) Act of 2022. As with our engagement with the House, our messaging consistently focused on the need for a legislative fix that would save patients’ lives and help them get the treatment they need at an affordable price.
Clear and specific messaging mattered. AMCP developed easily accessible repositories of information, including FAQs and Issue Briefs. Through our most engaged-with grassroots advocacy campaign ever, AMCP members called, emailed, and met with their legislators en masse to promote the benefits that PIE would provide to the practice of managed care pharmacy. We also published an op-ed in Health Affairs to reach policy influencers with real-life examples of why concurrent review was crucial for patient outcomes. Nevertheless, as a result of the vicissitudes of congressional politics, the final FDA User Fee Acts that passed in the Fall were stripped of all riders, including the PIE Act.
This setback provided the impetus for a renewed bipartisan push following the midterm elections. Rep. Guthrie was determined and worked closely with Democratic co-lead Representative Anna Eshoo of California to reintroduce the PIE Act on Nov. 15. The respective leaders of the E&C Health Subcommittee — joined by a deep bipartisan bench of supporters including original cosponsors Representatives Scott Peters (D-CA), Morgan Griffith (R-VA), Doris Matsui (D-CA), and Michael Burgess (R-TX) — were critical in advancing this important legislation, which was included in the Consolidated Appropriations Act, 2023 (H.R. 2617). This “omnibus” bill passed Congress on Dec. 23, 2022, and was signed into law on Dec. 29.
PIE’s success was the work of many hands across industries and political parties. It highlighted the importance of collaboration, patience, and determination in the face of setbacks. The PIE Act is now law because when the clock was running out, a committed group of stakeholders stayed engaged and on message.
Although this is a moment for celebration, there’s hard work ahead. It’s now up to manufacturers, payers, and plans to make good on the opportunity — to coordinate and share information that will accelerate patient access to new and novel treatments for serious illnesses.
When it comes to innovation in treatment and therapeutics, speed and safety matter. With the PIE Act finally law, the path is clear.