Search Results: keywords:"rare diseases"

  • H. R. 9938 — To amend the Federal Food, Drug, and Cosmetic Act to establish a time-limited conditional approval pathway, subject to specific obligations, for certain drugs and biological products, and for other purposes.
    Status:Introduced in House
    Words:5,297

    H. R. 9938 proposes amendments to the Federal Food, Drug, and Cosmetic Act to create a new, conditional approval pathway for certain drugs and biological products. This pathway is intended for drugs treating rare, progressive, and serious diseases, allowing them to receive...

    Simple Explanation

    H. R. 9938 is a plan to let special medicines for serious sicknesses be used sooner while they are still being checked. People who take these medicines might have to follow some rules, and health plans have to help pay for them.

  • H. R. 9979 — To amend the Federal Food, Drug, and Cosmetic Act to establish a process for externally led, science-focused drug development meetings, and for other purposes.
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    Status:Introduced in House
    Words:2,041

    H.R. 9979, also known as the "Scientific External Process for Educated Review of Therapeutics Act of 2024," aims to amend the Federal Food, Drug, and Cosmetic Act to create a process for science-focused drug development meetings. These meetings, led by qualified third...

    Simple Explanation

    H.R. 9979 is a new plan to help doctors, scientists, and people talk together about making medicines for rare illnesses. Each year, they want to have at least four big meetings where everyone shares ideas, and they have some money set aside to help pay for these talks.

  • H. RES. 1039 — Recognizing the significance of Charcot-Marie-Tooth disease and the need for robust funding of the National Institute of Neurological Disorders and Stroke at the National Institutes of Health.
    Status:Introduced in House
    Words:231

    H. RES. 1039 is a resolution introduced in the House of Representatives recognizing the importance of Charcot-Marie-Tooth (CMT) disease, which affects the peripheral nerves, impacting thousands in the United States and millions worldwide. It highlights that nearly all CMT cases are inherited,...

    Simple Explanation

    H. RES. 1039 talks about a disease called Charcot-Marie-Tooth (CMT) that hurts people's nerves. The bill says we need more money and research to help people with CMT and other similar diseases at a health institute so that doctors can find ways to make them feel better.

  • H. RES. 1036 — Expressing support for the designation of February 29, 2024, as Rare Disease Day.
    Status:Introduced in House
    Words:273

    H. RES. 1036 is a resolution in the United States House of Representatives that supports the designation of February 29, 2024, as "Rare Disease Day." It highlights how rare diseases affect a small number of people, with over 25 million Americans living with these conditions. The resolution...

    Simple Explanation

    This bill wants everyone to know that February 29, 2024, should be called "Rare Disease Day" to remind people that some illnesses are very uncommon and need more help and attention to find cures and make life easier for those who have them.

  • S. 4905 — To amend the Federal Food, Drug, and Cosmetic Act with respect to molecularly targeted pediatric cancer investigations, and for other purposes.
    Status:Introduced in Senate
    Words:2,469

    The bill, known as the "Innovation in Pediatric Drugs Act of 2024," aims to update the Federal Food, Drug, and Cosmetic Act to improve the investigation of drugs targeting pediatric cancer. It proposes changes to ensure timely completion of pediatric study requirements and outlines penalties...

    Simple Explanation

    The bill aims to make it easier and quicker for kids with cancer to get special medicine by making new rules for testing these medicines. It wants to ensure that companies follow these rules and ask people about their opinions to make sure they work well for everyone.

  • H. RES. 1025 — Expressing support for the designation of April 5, 2024, as Barth Syndrome Awareness Day.
    Status:Introduced in House
    Words:193

    H. RES. 1025 is a resolution that supports designating April 5, 2024, as "Barth Syndrome Awareness Day." It aims to raise awareness about Barth syndrome, a rare genetic disorder that primarily affects males and can be life-threatening due to heart failure or infections. The resolution...

    Simple Explanation

    H. RES. 1025 is a suggestion to make April 5, 2024, "Barth Syndrome Awareness Day" to help people learn about a rare illness that mostly affects boys and can make them very sick. This day is meant to encourage finding better ways to identify and treat the disease and to say thank you to groups that support these efforts.

  • H. R. 7383 — To amend the Federal Food, Drug, and Cosmetic Act to set forth limitations on exclusive approval or licensure of drugs designated for rare diseases or conditions.
    Status:Introduced in House
    Words:542

    H. R. 7383, also known as the "Retaining Access and Restoring Exclusivity Act" or the "RARE Act," proposes changes to the Federal Food, Drug, and Cosmetic Act to limit the exclusive approval or licensure of drugs designated for treating rare diseases or conditions. The bill...

    Simple Explanation

    The bill wants to change the rules for special medicines made for rare diseases, so they can only be used for the specific health problem they were approved for, not the whole disease. It wants these changes to count for all these special medicines, no matter when they were first allowed to be used.

  • S. 4426 — To amend the Federal Food, Drug, and Cosmetic Act to establish a time-limited conditional approval pathway, subject to specific obligations, for certain drugs and biological products, and for other purposes.
    Status:Introduced in Senate
    Words:5,340

    The bill S. 4426, known as the "Promising Pathway Act 2.0," proposes changes to the Federal Food, Drug, and Cosmetic Act. It introduces a temporary conditional approval pathway for certain drugs and biological products that treat rare, progressive, or serious diseases. This...

    Simple Explanation

    The Promising Pathway Act 2.0 is a plan to let some important new medicines for serious diseases become available to people sooner, even though doctors and scientists are still studying them. However, it also wants to make sure that these medicines are safe by making medicine makers check how well they work and keep patients informed.

  • S. RES. 636 — Designating February 29, 2024, as Rare Disease Day.
    Status:Agreed to Senate
    Words:205

    S. RES. 636 is a resolution passed by the U.S. Senate designating February 29, 2024, as "Rare Disease Day." This day recognizes the impact of rare diseases, which affect less than 200,000 people each in the United States, acknowledging the challenges faced by individuals with...

    Simple Explanation

    The U.S. Senate decided to make February 29, 2024, a special day called "Rare Disease Day" to help everyone remember and support people who have rare diseases, which are health problems that not many people have. This day is important for helping doctors figure out these diseases sooner and to encourage finding new ways to help people who have them feel better.