Observe Rare Disease Day by Facilitating Greater Access to Drugs
In observation of Rare Disease Day, the House Energy and Commerce Health Subcommittee is scheduled to hold a hearing today to discuss important bills impacting the rare disease community. During this hearing, several pieces of legislation will be addressed, including the Inflation Reduction Act, the ORPHAN Cures Act, and the MINI Act.
As TAPP has written in the past, the Inflation Reduction Act and its price controls damage the pharmaceutical industry as they limit innovation and patient access and diminish the standing of the United States in the global pharmaceutical industry.
The drugs that treat the rarest of diseases (diseases that affect fewer than 200,000 people in the United States) are known as orphan drugs. Because there is such a limited patient population, the development of these drugs does not make sense from an economic perspective and thus requires incentives and assurances.
As things stand, the Inflation Reduction Act excludes from its price controls only drugs that treat a single rare disease. Thus, orphan drugs that are effective against more than one disease are subject to price controls under the Inflation Reduction Act. This disincentivizes innovators from conducting costly follow-on research to find additional uses for orphan drugs.
This is where the ORPHAN Cures Act comes in. The ORPHAN Cures Act would amend the Inflation Reduction Act and protect orphan drugs that are effective against more than one rare disease from price controls. This would result in better access for patients and continued research for important cures.
The Inflation Reduction Act enforces price controls on drugs with two different timelines. Small molecule drugs that are approved as New Drug Applications (NDAs) become eligible after seven years. Large molecule drugs, known as biologics, become eligible after 11 years.
A problem occurs with drugs that incorporate or utilize genetically targeted technologies that have similarly complex manufacturing requirements and development timelines as biologics. These drugs are often classified as small drugs, despite costing the time and money of large drugs. Seven years is too short a grace period for these genetically targeted technologies, meaning innovators will not invest research into such a costly process because there is no hope of recouping the costs.
The Maintaining Investments in New Innovations (MINI) Act aims to realign the timeline for genetically targeted technology price controls with that of large drugs, giving greater incentive for research and development. While genetically targeted technologies are less common than NDAs and biologics, their effect on patients is not.
Rare diseases require the most research and development, and the United States needs to be the world’s leader when it comes to this R&D. Yet adopting socialist policies like those that exist in most of the rest of the world’s pharmaceutical industry and like those that reside in the Inflation Reduction Act has a chilling effect and threatens to diminish U.S. pharmaceutical innovation. America should not be satisfied with policies that abandon our global standing and push our drug innovators toward a least common global denominator.
For these reasons, TAPP is calling on Congress to mitigate at least some of the negative impact of the Inflation Reduction Act by passing the MINI Act and the ORPHAN Cures Act.