Fairness in Orphan Drug Exclusivity

Floor Speech

BREAK IN TRANSCRIPT

Ms. DEAN. Madam Speaker, I thank Ranking Member Rodgers and Chairman Pallone for their leadership on this bill.

I, once again, rise in support of H.R. 1629, the Fairness in Orphan Drug Exclusivity Act.

This crucial legislation would close a current loophole that is used to block competition in the pharmaceutical marketplace.

The Orphan Drug Act of 1983 provided incentives for prescription drug manufacturers to develop products to treat rare diseases. This includes an exclusive 7-year marketing right for therapies that receive an orphan drug designation.

For a drug to qualify, it must either be a treatment for a disease or condition that affects fewer than 200,000 people in the United States or a drug intended for diseases that there is no reasonable expectation to recoup research and development costs.

It would require all drug manufacturers who obtain orphan drug status to prove that they have no reasonable expectation that they will recover R&D costs.

This legislation works to prevent companies from continuing to use orphan drug exclusivity status for a newly approved drug with an identical ingredient to the former version without having to prove the inability to recoup costs.

This exact circumstance, as the chairman described, happened when a manufacturer of a buprenorphine product tweaked an older product that had received orphan drug status and subsequently was given a renewed orphan drug exclusivity.

Buprenorphine is used as a treatment for opioid use disorder to help those recovering from addiction. Unfortunately, at the time, the opioid epidemic was raging, as it is today, and was by no means a rare disease. And the drug was by no means a market loser. It was a moneymaker.

Closing this loophole would ensure that products do not receive an unfair market advantage and will get more affordable drugs to patients. It ensures consistency with the spirit and intent of the Orphan Drug Act.

Know that we want to encourage and support the development of rare disease treatments and therapeutics. It is critically important. But we cannot allow this important incentive to be co-opted to allow for unfair market competition for drugs that are not a commercial loss.

This bill was supported unanimously last Congress and has broad support among patient advocates. This bill is about ensuring market competition, reducing barriers to the development of new treatments, and, ultimately, supporting patients.

Madam Speaker, I include in the Record a letter from 16 patient advocacy organizations in support of this legislation.
March 9, 2021. Re H.R. 1629--The Fairness in Orphan Drug Exclusivity Act. Hon. Madeleine Dean, Washington, DC. Hon. Marc Veasey, Washington, DC.

Dear Representatives Dean and Veasey:

Thank you for your leadership on the reintroduction of H.R. 1629, The Fairness in Orphan Drug Exclusivity Act. Our nation is in crisis. Each day, 130 Americans die from an opioid overdose, and according to recent provisional data from the Centers for Disease Control and Prevention, between June 2019 and May 2020, the number of people who died of an opioid overdose increased by 38.4%.

We have a responsibility to ensure that all treatment options are made available to those living with opioid use disorder {OUD). Last year, this important legislation was approved by the House of Representatives unanimously. Unfortunately, the Senate was unable to also act on the measure before the 116th Congress adjourned. The new Congress now has an opportunity to pass this legislation swiftly and improve access to life-saving medication assisted treatments and prevent more lives from being lost. We, the undersigned organizations, support H.R. 1629 and urge quick passage of this important bill that will help ensure the availability of vital treatment options for OUD that can save American lives.

The Orphan Drug Act, enacted in 1983, allows the Food and Drug Administration (FDA) to grant manufacturers Orphan Drug Designation (ODD) and Orphan Drug Exclusivity (ODE) to a product developed to treat rare diseases and conditions affecting less than 200,000 patients. In addition, a manufacturer also can qualify for ODD and ODE if more than 200,000 patients are affected, but there is ``no reasonable expectation'' of recovering development costs. Few know about this option, and as a result it is exceedingly rare with only three uses to date.

In such cases, if a newly approved product has the same active ingredient as a previous product that received orphan designation and exclusivity because the FDA determined the original drug could not recoup its development costs, the newly approved product does not have to demonstrate the inability to recoup its development costs. It does not matter how much time has passed, or how much money the newly approved drug is predicted to make. This loophole creates the potential for abuses within the system, and it is not consistent with intent of the Orphan Drug Act.

Enacting H.R. 1629 will: fix this loophole in a narrow and targeted manner to prevent this abuse of the Orphan Drug Act; preserve incentives for development of products treating rare diseases and conditions; and ensure new treatment options come to market to treat OUD.

During this public health emergency, we need all the tools we can muster to fight this crisis. We should not tie one hand behind our back as the exploitation of this loophole would do.

More than 2.1 million Americans live with OUD, making this disease far from rare. This disease is a national public health emergency, and we need to close this loophole to ensure that all safe and effective treatments are available to fight this disease. Thank you again for your leadership on this issue, and we look forward to working with you to help pass H.R. 1629. Sincerely, Advocates for Opioid Recovery, AIDS United, Aimed Alliance, Daniel's story, Center for U.S. Policy, Cover2 Resources, FORCE, Healthy Women, Maryland Heroin Awareness, Mother's Addiction Journey, No More OD's, Inc., NOPE Task Force, Prevention Action Alliance, Shatterproof, Tyler's Light, Young People in Recovery.

BREAK IN TRANSCRIPT

Ms. DEAN. Madam Speaker, I include in the Record the Statement of Administration Policy issued by the Office of Management and Budget on May 17 of this year in support of this bill. Statement of Administration Policy H.R. 1629--Fairness in Orphan Drug Exclusivity Act--Rep. Dean, D- Pennsylvania, and two cosponsors

The Administration supports House passage of H.R. 1629, the Fairness in Orphan Drug Exclusivity Act. Orphan drug status is intended to encourage companies to develop promising drugs for rare diseases. Current law provides market exclusivity for drugs that treat any disease or condition which (A) affects fewer than 200,000 persons in the United States, or (B) affects more than 200,000 in the United States and for which there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will be recovered from that drug's sales in the United States. H.R. 1629 affects only drugs that qualify under the latter provision. Current law allows market exclusivity to be extended for a new version of the same drug without the drug developer having to show a lack of profitability for that new version as well. This legislation would close that loophole, requiring all drugs that obtain seven years of market exclusivity for conditions affecting 200,000 or more people to illustrate that they have no reasonable expectation of recovering R&D costs through U.S. sales.

The Administration applauds these steps to ensure Americans have access to high quality, affordable treatments.

BREAK IN TRANSCRIPT

Ms. DEAN. Madam Speaker, I thank Chairman Pallone for his support on this bill, and I urge all members to support its passage.

BREAK IN TRANSCRIPT

Source