Yesterday, the House Energy and Commerce Committee released its much-anticipated discussion draft for its 21st Century Cures initiative. NORD President and CEO, Peter L. Saltonstall, issued the following statement in response.
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.
Soft drink and fast food companies often make children the primary targets of their advertising. These companies know that people frequently establish consumption habits at an impressionable young age that extend well into adulthood. Such a long-term strategy isn’t just smart business. It is essential to the survival of the brand.
As a second-year medical student with two younger brothers who have a rare genetic disease – ataxia telangiectasia – I think the rare disease community can learn and benefit from the strategies of these corporations. read more >
The cost of orphan drugs, and whether it is approaching a level where sustainability may be an issue, is a topic of intense interest in the rare disease community. However, an important new study reported at the recent American Society of Hematology meeting suggests that the overall budget impact of orphan drugs may be smaller than many people would assume. read more >
Today, NORD Vice President Diane Dorman is providing remarks before the FDA Oncologic Drugs Advisory Committee on the importance of distinguishable names for biologics as a fundamental core of maximizing the benefits for patients and minimizing any potential harm from biosimilars. This is part of NORD’s work to provide advocacy on behalf of the entire rare disease community. Read Diane’s remarks here and follow the conversation online under the hashtag, #ODAC.
ESPN anchor Stuart Scott passed away last night at just 49 years old after his 7-plus year battle with appendix cancer. Mostly Scott’s passing makes me feel for his daughters, his family, his friends, and his colleagues. This is their time to reflect and remember him. However, I also think that his public presence makes it seem like we all knew him, and I think he taught us all something by how he handled his illness.
I didn’t know Stuart Scott. But I feel a special kinship with him because the broad outlines of his story and mine are remarkably similar. We were both diagnosed with appendix cancer in November 2007 – I went to the ER on the Friday after Thanksgiving and he had an emergency appendectomy the following Monday (while I was still in the hospital being diagnosed). We both had two recurrences – mine in 2009 and 2010, and his in 2011 and 2013, with multiple major surgeries as our rewards. We both went to work while our bodies absorbed incredible amounts of energy-depriving chemotherapy. We both fought with everything we had to live for our kids. So I didn’t know Stuart Scott, but I know something about what he went through in fighting appendix cancer.
RareDisease Dialog is the official blog for the National Organization for Rare Disorders (NORD). NORD’s staff and friends will share information of interest to the entire rare disease community.
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