To help protect access to medicines for cancer and rare disease patients, AstraZeneca  has filed a legal challenge to critical aspects of the drug price negotiation provisions of the Inflation Reduction Act (IRA). The drug price negotiation provisions of the IRA run headlong into the goals of the Orphan Drug Act (ODA), a federal statute designed to encourage manufacturers to invest in new therapies for rare diseases.

Over the last forty years, American patients have benefited from the highly effective incentives the ODA put in place, which spurred companies to focus research and development investment on small populations of patients with significant unmet need. The passage of the ODA accelerated innovation leading to more than 600 medicines treating 1,000 rare conditions today, including cancers. Yet, the vast majority of the 7,000 known rare conditions still have no approved treatments, and continued innovation for small indications is at risk under the IRA.

Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: “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.”

Recently, an article in Journal of the American Medical Association (JAMA) Network Open stated similar concerns.

Many cancer medicines in the US launch first in an orphan indication and broaden use over time to additional populations. One example is LYNPARZA^® (olaparib), a small-molecule cancer medicine approved in 2014 in the US for a small group of late-line ovarian cancer patients. Additional trials added small groups of breast and pancreatic cancer patients, with the most recent indication in prostate cancer approved just this year – nine years later. If the IRA had been in place, significant disincentives would have existed for pursuing the late-line ovarian cancer approval in the US, an indication which has benefited patients in great need of this unique medicine for their rare condition.

Another example is SOLIRIS^® (eculizumab) which has received approval to treat four rare diseases, including debilitating and potentially life-threatening neuromuscular and hematological diseases. First approved in 2007 in paroxysmal nocturnal haemoglobinuria (PNH), a rare chronic blood disorder, historic dynamics enabled continued research investment to support further innovation, resulting in a US approval more than a decade later in neuromyelitis optica spectrum disorder (NMOSD), a rare, autoimmune disease that affects the central nervous system. The IRA would have deterred the continued development of this life-changing medicine for patients with rare diseases beyond its initial indication.

Correcting these issues and protecting medicines like LYNPARZA and SOLIRIS from price-setting will have minimal impact on the overall cost to the US healthcare system but a tremendous impact on patients. As a result, AstraZeneca has taken the step of filing a legal challenge to aspects of the IRA, to protect timely access to medicines for orphan indications in the US