RDLA Update

Our board chair traveled to Washington, DC, in July to attend the Rare Disease Legislative Advocate brief with the Rare Disease Caucus. We learned much about the impact of the Inflation Reduction Act (IRA) signed into law in 2022, and its effect on the Orphan Drug Act 1983.

The Inflation Reduction Act of 2022 (IRA) could be harmful to the Orphan Drug Act because it could discourage the development of new orphan drugs. The IRA allows Medicare to negotiate the prices of prescription drugs, including orphan drugs. This could lead to lower prices for orphan drugs, making it less profitable for drug companies to develop them.

The Orphan Drug Act was designed to encourage the development of drugs for rare diseases by providing financial incentives to drug companies. These incentives include market exclusivity, tax credits, and grants. However, the IRA could make these incentives less valuable, leading to fewer drug companies developing orphan drugs.

In addition, the IRA could make it more difficult for patients with rare diseases to access the necessary drugs. If the prices of orphan drugs are too low, drug companies may not be able to afford to continue producing them. This could mean patients with rare diseases must switch to other, less effective treatments or go without treatment altogether.

The IRA is still a relatively new law, and it is not yet clear how it will impact the development of orphan drugs. However, there are concerns that the law could have a negative impact on patients with rare diseases.

Here are some specific examples of how the IRA could harm the development of orphan drugs:

• Drug companies may be less likely to start development programs for orphan drugs if they know that the prices of these drugs could be negotiated down in the future.

• Drug companies may be less likely to invest in late-stage clinical trials for orphan drugs if they are not confident that they will be able to recoup their investment.

• Drug companies may be more likely to focus on developing orphan drugs that have a large potential market rather than drugs for rare diseases with a smaller patient population.

The impact of the IRA on the development of orphan drugs is still uncertain. However, there are concerns that the law could have a negative impact on patients with rare diseases, and the outcomes of the IRA should be watched closely to see how the IRA is impacting the Orphan Drug Act..