Did the administration consider the importance of blood cancer medicines to patients in price setting process

We've discussed how the Inflation Reduction Act's (IRA) price setting policies threaten research and development of new medicines for diabetes, autoimmune diseases and blood clots. Today, we look at the impact of government price setting for patients with blood cancers, since a crucial blood cancer medicine was included in the first round of price setting. The specific form of blood cancer the selected medicine treats is common among Medicare beneficiaries, with an average age of diagnosis of 70 years.

The impact: Blood cancers like leukemia, lymphoma and myeloma are familiar names when it comes to cancer. Together, these blood cancers make up nearly one out of every 10 new cancer cases in the United States. According to the Leukemia & Lymphoma Society, approximately every nine minutes, someone dies from a form of blood cancer.

How far the industry has come: Groundbreaking new medicines are transforming the way we treat many blood cancers. For example, patients today treated for a common form of chronic leukemia can expect to live normal life spans. These treatment advances are due in large part to targeted therapies that help manage this cancer. Additionally, immunotherapies such as CAR-T cell therapies are used across a wide range of blood cancers and have even been shown to cure many children with advanced forms of leukemia.

Post approval R&D is critically important in advancing new treatments for patients with cancer. The majority of cancer R&D is done after initial FDA approval - more than 60% of oncology medicines approved a decade ago received approval for additional indications for other types of cancer, with most subsequent approval occurring seven or more years after initial approval. Post-approval R&D increases treatment options for patients by demonstrating, for example, that an existing medicine can treat a different disease or patient population, or that a new dosage form or formulation is safe and effective.

Access to blood cancer treatments makes a difference:

• One study, looking across the 15 most common tumor types, found that new cancer medicines approved between 2000 and 2016 alone were associated with nearly 1.3 million prevented cancer deaths, including nearly 100,000 for leukemia and lymphoma.
• While the five-year survival rate for the most common types of blood cancers were rarely higher than 34% in 1975, they now reach 92% for some forms of the disease.

IRA threatens the progress on the horizon: America's biopharmaceutical researchers are developing hundreds of new therapies for different blood cancers, including 162 potential treatments for lymphoma, 158 for leukemia and 84 possible new treatments for multiple myeloma. Unfortunately, setting the price of medicines treating blood cancers puts R&D at risk.

The IRA discourages the post-approval R&D that is so critical to realizing the full potential of cancer medicines. The first round of price setting illustrates this disincentive in action:

• The blood cancer therapy that was price set this fall was initially approved for a form of blood cancer. Further R&D, after its initial approval, allowed the medicine to be approved for other forms of blood cancer and also for an entirely different disease in pediatric patients: graft-versus-host disease (GVHD).
• The innovation didn't stop there. This same treatment was also later developed into an oral suspension form to make it easier to take for children with GVHD.
• With the IRA, the government has chosen to consider every medicine with the same "active ingredient or moiety" as one, single drug. So even though these medicines were approved under entirely different drug applications many years after the initial therapy was approved, they are all wrapped up in the same price setting process.
• This signals to manufacturers that investments in post-approval R&D may be too risky to pursue.

The bottom line: Since the government-set price applies to a medicine regardless of what disease it's treating or its form, the law discourages the costly and time-consuming clinical research needed to further research and develop therapies for patients with blood cancers.

Until policymakers fix the flaws in the law, patients with blood cancers and other diseases will unfortunately be the collateral damage of this price-setting scheme. Learn more about how price setting can undercut the immense value new medicines provide.

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