May 21st 2015

Rare disease patient advocates have reason to celebrate an important milestone!

The House Energy and Commerce Committee unanimously passed H.R. 6, the 21st Century Cureslegislation yesterday, which is a crucial step in advancing measures that enhance drug discovery and development of treatments and cures for patients.

Global Genes applauds the year-long bipartisan initiative led by Chairman Fred Upton (R-MI) and Rep. Diana DeGette (D-CO) and the engagement of numerous stakeholders in this process, which has resulted in legislation vital to rare disease patients and families. We thank the leaders and members of the Energy and Commerce Committee for their continued commitment to the rare disease community throughout this entire process.

H.R..6 includes provisions that offer greater incentives for orphan drug development, increase much needed funding for the National Institutes of Health (NIH), strengthen patient-focused drug development and streamline clinical trials. It also reauthorizes for four years a critical program for pediatric rare disease drug development. Global Genes looks forward to continuing to support this legislation and the Committee’s commitment to move it through House, as well as efforts to encourage consideration by the Senate.

To see how Global Genes, along with numerous other rare disease advocacy organizations, supported 21st Century Cures, take a look at these two letters of support 1 & 2. Many rare disease patient advocates have engaged in and vocally supported measures contained in the legislation throughout this entire process, and your voices were heard! Patients across the rare community also mobilized last week to advocate for inclusion of the OPEN Act in 21st Century Cures legislation. You were successful, and the OPEN Act is included in the bill voted out of Committee today. For additional background on this legislation including the bill text, managers amendment and briefing memo, please click here. Global Genes will continue to update our network of advocates and alliance members regarding this important bill as it continues to move through the legislative process.

For more information on this important topic, please check out our RARE Daily here.


March 10th 2015

Senate on the 21st Century Cures legislation.

The Senate Health Committee held its first hearing on the topic. From the Senate’s website,here is a link to information on that hearing.

US Senate Committee on Health, Education, Labor, & Pensions: Newsroom – Press Releases


APBDRF Advocates for repurposing of existing drugs for rare disease

The APBDRF has signed on in support of the bipartisan OPEN ACT (Orphan Product Extensions Now Acclerating Cures & Treatments), which aims to incentivize repurposing of existing drugs for rare disease indications.  A copy of the letter is attached.  We have joined nearly 70 other organizations (such as the National Tay-Sachs & Allied Diseases Association and The Association for Glycogen Storage Disease)that have signed-on to support this Act.

The OPEN ACT’s incentive is modeled after the Best Pharmaceuticals for Children Act which resulted in more than 400 drug label changes.  With less than 500 drugs approved to treat rare diseases, the OPEN ACT has the potential to double the number of approved drugs for Rare Disease Patients.

NORD Issues Statement on 21st Century Cures Discussion Draft

WASHINGTON, D.C.—January 28, 2015—The following statement was issued today by Peter L. Saltonstall, President and CEO of NORD, following the release of a discussion document by the House Energy and Commerce Committee for its 21st Century Cures initiative:

The discussion paper issued by the House Energy and Commerce Committee on January 27 contains a number of ideas on how to advance the development of new medical therapies. A few provisions apply specifically to rare diseases, including adding six months to the patent life of orphan drugs. The Committee and its staff deserve praise for its effort in bringing these ideas together. NORD was pleased to have submitted a number of position papers for the Committee to consider.

NORD’s Advocacy Committee will now conduct an evaluation of the discussion paper. Once that process is completed, we will share our position with the Committee. We will continue to work closely with the Committee and members of the House and Senate as legislation moves forward.

Nothing is more important to the rare disease community than providing an environment conducive to the development of new therapies and access to them. We look forward to working with other like-minded advocates on these issues.

For more information about NORD, visit