S. 1862: Optimizing Research Progress Hope And New Cures Act

This bill, titled the Optimizing Research Progress Hope And New Cures Act, aims to modify certain provisions related to orphan drugs under the Drug Price Negotiation Program. Below are the key components of what this bill would accomplish:

1. Definition and Purpose

The primary goal of the bill is to streamline and expand the way orphan drugs are handled in terms of pricing negotiations within Medicare, encouraging the development of treatments for rare diseases.

2. Expanding the Orphan Drug Definition

• The bill proposes to amend the definition of orphan drugs under the Social Security Act. Specifically, it changes the language from referring to "only one rare disease or condition" to "one or more rare diseases or conditions." This expansion aims to encompass a broader range of conditions and encourage more drug applications for rare diseases.
• Additionally, the bill clarifies the definition of orphan drugs by specifying that it includes "one or more rare diseases or conditions" as defined in the Federal Food, Drug, and Cosmetic Act, which provides precise criteria for what constitutes a rare disease.

3. Treatment of Former Orphan Drugs

The bill introduces a new provision regarding the treatment of drugs that were previously classified as orphan drugs. When considering the timeframe for price negotiations of these drugs, any period during which the drug was classified as an orphan drug will not be considered. This aims to ensure that companies are not penalized for the time that a drug spent as an orphan drug when transitioning to a new classification or negotiation phase.

4. Implications for Drug Development

By making it easier to classify drugs for multiple rare diseases and providing more favorable conditions for former orphan drugs, the bill seeks to incentivize pharmaceutical companies to invest in the research and development of treatments for rare conditions. The expectation is that these changes could lead to an increase in the availability of new therapies for patients with rare diseases.

5. Legislative Process

This bill was introduced to the Senate and is currently being reviewed by the Committee on Finance, where it will undergo further discussion and potential amendments before any voting takes place.

Relevant Companies

• AMGN (Amgen Inc.): As a biotechnology company involved in the development of orphan drugs, changes in the regulatory environment could alter the company's strategy in research and pricing.
• BIIB (Biogen Inc.): Known for its treatments for rare neurological disorders, this company may benefit from the expansion of orphan drug definitions.