The following is from NORD - National Organization for Rare Disorders - I thought many of you would be interested.
NORD Reports Progress in Appropriations Bill
NORD has been notified that the Senate appropriations bill to establish funding levels for key areas related to rare diseases and orphan products has now been drafted and submitted to the Appropriations Committee. We are delighted to report significant progress, especially on the following two points:
The bill includes the first increase in funding for the Orphan Product Development Grant program since Fiscal Year 2005. A $2 million increase is proposed, which would bring funding for the program to $16,035,000.
It also includes funding of $1 million to hire additional staff with expertise in rare diseases and orphan products for the new office established in FDA’s Center for Drug Evaluation and Research earlier this year. This office will focus on development of treatments for rare diseases, and it was established largely as a result of advocacy by NORD. Dr.Anne Pariser, an FDA staff member with strong experience in the orphan products arena, was appointed to the new position of Associate Director for Rare Diseases in CDER. Since her appointment, NORD has been advocating strongly for funding to hire additional staff to help her. We are very happy that the appropriations bill includes this.
The bill also includes other key features on which NORD, the Kakkis EveryLife Foundation, and other partners have been working very hard. The specific wording of the notification sent to NORD yesterday afternoon by representatives of the U.S. Senate appropriations subcommittee is as follows:
Friends,
Shortly, the Appropriations Committee will take up the FY 2011 Agriculture Appropriations bill. During the drafting of this bill, we worked very closely with Chairman Kohl's staff to include specific items to continue moving FDA forward in the rare and neglected disease area. We are very excited about these improvements and it is our hope that you will be too.
The FY 2011 Senate bill includes the first increase for the Orphan Product Development Grant program since FY 2005. The program is increased by $2,000,000 for a total grant level of $16,035,000.
The FY 2011 Senate bill includes, for the first time, specific funding for the Office of the Associate Director for Rare Diseases in CDER. Funding for this office is increased by $1,000,000 to hire additional staff with specific expertise in facilitating the development and review of products to treat these diseases.
The manager’s package that will be adopted at Committee (hopefully)includes bill language cleared by both FDA and the HELP Committee. This bill language builds on the Brownback/Brown language that was included in the FY 2010 Agriculture Appropriations bill (section 740). The language requires FDA to:
1. increase the use of accelerated approval, as appropriate;
2. work with sponsors to facilitate expanded access to investigational therapies;
3. increase coordination with the WHO and international regulatory agencies;
4. increase collaboration with regulatory authorities in developing countries;
5. develop guidance on clinical development programs for rare diseases;
6. develop guidance on the use of surrogate endpoints for use under the agency's accelerated approval process; and
7. increase coordination among the individual drug, biologic product, and device review divisions to support the development of products to treat rare and neglected diseases.
The agency is required to implement these as a part of the FY 2010 reforms and report back to the Committee on implementation of these items.
Thank you for all your help during the past few months.
U.S. Senate Committee on Appropriations
Subcommittee on Agriculture, Rural Development,
Food and Drug Administration, and Related Agencies
NORD Reports Progress in Appropriations Bill
NORD has been notified that the Senate appropriations bill to establish funding levels for key areas related to rare diseases and orphan products has now been drafted and submitted to the Appropriations Committee. We are delighted to report significant progress, especially on the following two points:
The bill includes the first increase in funding for the Orphan Product Development Grant program since Fiscal Year 2005. A $2 million increase is proposed, which would bring funding for the program to $16,035,000.
It also includes funding of $1 million to hire additional staff with expertise in rare diseases and orphan products for the new office established in FDA’s Center for Drug Evaluation and Research earlier this year. This office will focus on development of treatments for rare diseases, and it was established largely as a result of advocacy by NORD. Dr.Anne Pariser, an FDA staff member with strong experience in the orphan products arena, was appointed to the new position of Associate Director for Rare Diseases in CDER. Since her appointment, NORD has been advocating strongly for funding to hire additional staff to help her. We are very happy that the appropriations bill includes this.
The bill also includes other key features on which NORD, the Kakkis EveryLife Foundation, and other partners have been working very hard. The specific wording of the notification sent to NORD yesterday afternoon by representatives of the U.S. Senate appropriations subcommittee is as follows:
Friends,
Shortly, the Appropriations Committee will take up the FY 2011 Agriculture Appropriations bill. During the drafting of this bill, we worked very closely with Chairman Kohl's staff to include specific items to continue moving FDA forward in the rare and neglected disease area. We are very excited about these improvements and it is our hope that you will be too.
The FY 2011 Senate bill includes the first increase for the Orphan Product Development Grant program since FY 2005. The program is increased by $2,000,000 for a total grant level of $16,035,000.
The FY 2011 Senate bill includes, for the first time, specific funding for the Office of the Associate Director for Rare Diseases in CDER. Funding for this office is increased by $1,000,000 to hire additional staff with specific expertise in facilitating the development and review of products to treat these diseases.
The manager’s package that will be adopted at Committee (hopefully)includes bill language cleared by both FDA and the HELP Committee. This bill language builds on the Brownback/Brown language that was included in the FY 2010 Agriculture Appropriations bill (section 740). The language requires FDA to:
1. increase the use of accelerated approval, as appropriate;
2. work with sponsors to facilitate expanded access to investigational therapies;
3. increase coordination with the WHO and international regulatory agencies;
4. increase collaboration with regulatory authorities in developing countries;
5. develop guidance on clinical development programs for rare diseases;
6. develop guidance on the use of surrogate endpoints for use under the agency's accelerated approval process; and
7. increase coordination among the individual drug, biologic product, and device review divisions to support the development of products to treat rare and neglected diseases.
The agency is required to implement these as a part of the FY 2010 reforms and report back to the Committee on implementation of these items.
Thank you for all your help during the past few months.
U.S. Senate Committee on Appropriations
Subcommittee on Agriculture, Rural Development,
Food and Drug Administration, and Related Agencies
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