Patients, Policy and the IRA: Navigating an Evolving Federal Landscape

Dec 21, 2023

(Photo by John Wilcox)

In recent years, significant shifts in federal policy have had an outsized impact on the biotech and life sciences industries and the patients they serve. Paramount among these is the Inflation Reduction Act (IRA), which has complicated the intricate dance between policymakers, patients, drug developers, and other stakeholders within the healthcare system. The economic implications of the IRA continue to ripple through the industry, influencing everything from affordability to innovation to the relationships between industry and patients.  

MassBio has been at the forefront of these conversations, interacting with agencies like the Centers for Medicare & Medicaid Services (CMS) on the implementation of the drug price negotiation program, working to ensure the life-saving and life-changing work of our member companies isn’t slowed or disincentivized by the IRA’s fine print. 

As part of MassBio’s Patient Advocacy Summit last month, we welcomed a diverse panel of experts across the advocacy, healthcare, and policy spectrums to discuss how our industry can best navigate the challenges between federal policies and patient-driven healthcare. 

Navigating the IRA 

Takeda’s Liz Lewis moderating a panel at the Patient Advocacy Summit. (Photo by John Wilcox)

Our panel, which was moderated by Takeda Oncology Head of Global Oncology, Patient Value, Policy and Access Liz Lewis, and featured patient advocate and mother of a child with a rare disease Kayla Klein, Stoke Therapeutics Head of Global Patient Advocacy Parisa Sanandaji, and EveryLife Foundation Director of Public Policy Jamie Sullivan, MPH, shared their thoughts on the IRA and what its complex provisions mean for patients. 

Klein spoke to the uncertainty many patient advocates feel about some of the law’s unintended consequences.  

“There are going to be positives [out] of it but as a rare disease mom, it’s just another layer of what’s going to happen, how we find our voice, what are we going to have to say to amplify that this affects us and could potentially affect our family,” she said.  

Many policies are enacted without their authors fully understanding the implications, especially on the smaller rare disease communities. People like Klein, whose son has Severe Hemophilia A, are left to wonder what the goal of such a federal policy is.  

“There’s many benefits for patients, especially those on Medicare, those who are eligible for larger subsidies,” said Sullivan. “But what we haven’t done is taken a nuanced approach to avoid the [unintended] consequences.” 

Innovation Is Hope 

Innovation in healthcare is not a luxury; it’s a necessity. As federal policies shape the economic framework, the challenge lies in fostering innovation without exacerbating financial burdens on patients. Our panelists talked about the industry imperative to promote cost-effective, sustainable solutions. This may involve incentivizing research and development that specifically targets affordability, encouraging the adoption of technology that streamlines processes and reduces overhead costs, and fostering partnerships between public and private sectors to drive innovation that aligns with economic constraints. 

“You have more misses than hits,” said Sanandaji. “There is a lot of money and investments that go into developing new drugs and solutions to quickly bring to families. The IRA will be nuanced and there will be provisions that are tricky to navigate, so, we need to think about incentives that will help us approach these issues for R&D.”  

Sullivan called for greater transparency from federal policymakers on why certain decisions are being made and greater discourse between patients and policymakers. 

“There is a call to action,” she said. “We need to know what is happening behind closed doors and why policy makers are making certain decisions. We can’t help or inform the public if we don’t know what’s happening ourselves. Knowing the details will help both patient advocates and industry players advocate for the necessary changes that will have the most significant impact.” 

Sullivan spoke about some of the intermediate technical corrections to the IRA her organization is focused on such as expanding the Optimizing Research Progress Hope and New Cures (ORPHAN Cures) Act and clarifying that the negotiation clock should start for orphan products only once it loses its exemption.  

“There are 18 drugs on the market for hemophilia and unfortunately, none of them worked for my son,” said Klein, highlighting the importance or rare disease research and innovation. “From January to March in 2020, we were in and out of the hospital until they shut down for COVID. Right before our final discharge, there was a new drug that just launched, and it worked. Innovation is key and because of it, my son is now able to be a child first, and a child with hemophilia second.” 

Industry and Patients: A Symbiotic Relationship for Success 

All our panelists agreed the life sciences industry must broaden its tent, increasing the awareness that policymakers have with patients and bridging the gap between research and the patients who benefit from them. Industry professionals may be the experts in technology, but patient advocates are the experts in living with rare diseases. 

The IRA is a massive piece of legislation and, even those who have steadfastly supported it know that a huge piece of legislation often has significant unintended consequences. Our patient advocacy panelists highlight the need for collaboration between those writing the policies and those whose families are impacted most by them. 

“Putting on my mom hat, innovation really is key to provide what’s best for you or your child when it comes to rare diseases,” said Klein. “We just have to continue to drive innovation forward for families like mine.” 

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