As the clock ticks down to the eagerly awaited unveiling of the Centers for Medicare and Medicaid Services’ (CMS) list of ten drugs earmarked for inaugural pricing negotiations, AstraZeneca takes a bold stride into the heart of the legal fray encircling the Inflation Reduction Act (IRA). However, diverging from the constitutional arguments that other pharmaceutical giants have wielded, AstraZeneca’s legal gambit finds its anchor in the corridors of history—specifically, the 1983 Orphan Drug Act (ODA), and the unforeseen consequences ushered in by the new IRA.
In a nuanced legal dance, AstraZeneca contends that the Medicare price negotiation measures introduced by the IRA have embarked on a collision course with the aspirations of the ODA. In a carefully worded statement, the pharmaceutical powerhouse articulated that the IRA’s directives clash head-on with the noble objectives enshrined within the Orphan Drug Act.
The ODA, conceived as a harbinger of progress, granted a seven-year window of market exclusivity, fostering the development of therapies aimed at combating rare and orphan diseases. AstraZeneca underscored the impressive strides made since the ODA’s inception, highlighting the emergence of over 600 medications targeting 1,000 rare diseases, a testament to the transformative power of innovation.
“Rare disease and cancer patients depend upon high-risk, low-probability drug development that takes many years to develop and aims for cure. If today’s version of the law stands, patients in the United States with rare conditions, who have benefited from the Orphan Drug Act, will get delayed access to scientific breakthroughs relative to other parts of the world.”
– Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca
Yet, within this symphony of achievement, gaps persist. The shadows still shroud most of the 7,000 identified rare conditions, languishing without approved remedies. It is within this context that AstraZeneca’s legal protest finds its resonance.
The crux of AstraZeneca’s argument lies within the tale of Lynparza, a cancer drug borne from the partnership with Merck. Lynparza’s journey from its initial FDA approval in 2014 for a select group of ovarian cancer patients to its recent expansion into prostate cancer exemplifies the narrative arc. However, AstraZeneca contends that if the IRA’s framework had governed Lynparza’s trajectory from the outset, the very existence of these subsequent indications could have been jeopardized due to “significant disincentives.”
Another star in AstraZeneca’s constellation, Soliris, takes center stage. This drug, a product of AstraZeneca’s colossal $39 billion acquisition of Alexion, stands as a beacon of hope for four rare diseases. With its initial approval in 2007 for paroxysmal nocturnal hemoglobinuria, a rare chronic blood disorder, Soliris continued its journey, adding the rare autoimmune disease neuromyelitis optica spectrum disorder to its repertoire in 2019. AstraZeneca contends that under the IRA’s sway, this ongoing evolution would have been stifled.
AstraZeneca joins the ranks of its peers, becoming the sixth participant in the legal pageantry surrounding the IRA. Names like Merck, Bristol Myers Squibb, Johnson & Johnson, Astellas, and most recently Boehringer Ingelheim have also stepped onto the stage. Beyond the pharmaceutical realm, the symphony of dissent resonates further—trade group Pharmaceutical Research and Manufacturers of America and the US Chamber of Commerce lend their voices to the chorus challenging this law.
Amidst these legal harmonies, CMS prepares to unveil its inaugural list of drugs poised for Medicare drug price negotiations. Set against the backdrop of 2026, this watershed moment holds promises of change, reverberating through the corridors of pharmaceutical innovation and policy reform alike.
