News from NORD

A recent article in the Financial Times looked at the growing impact families have on medical research and highlighted the training and advice that NORD provides to family foundations.

A recent article in the Financial Times looked at the growing impact families have on medical research and highlighted the training and advice that NORD provides to family foundations.

A recent article in the Financial Times looked at the growing impact families have on medical research and highlighted the training and advice that NORD provides to family foundations.

On July 12, the FDA’s Division of Drug Information will host a webinar at 1 pm ET and will offer one hour of continuing education credit. The topic will be the Expanded Access process, through which patients may obtain access to investigational therapies and a new patient application recently published by FDA.

On July 12, the FDA’s Division of Drug Information will host a webinar at 1 pm ET and will offer one hour of continuing education credit. The topic will be the Expanded Access process, through which patients may obtain access to investigational therapies and a new patient application recently published by FDA.

On July 12, the FDA’s Division of Drug Information will host a webinar at 1 pm ET and will offer one hour of continuing education credit. The topic will be the Expanded Access process, through which patients may obtain access to investigational therapies and a new patient application recently published by FDA.

NORD has commended the US Senate for authorizing medical foods coverage for the military in the National Defense Authorization Act (S.2943). The Senate bill now must be harmonized with the House version passed in May. Medical foods are special formulas and foods that are medically necessary for many digestive and inherited metabolic disorders. Until now, coverage has been routinely denied by TRICARE, a health care program of the US Department of Defense. NORD and several of its advocacy partners met with the Armed Services Committee and advocated on behalf of families for whom these special foods are a necessity.  

NORD has commended the US Senate for authorizing medical foods coverage for the military in the National Defense Authorization Act (S.2943). The Senate bill now must be harmonized with the House version passed in May. Medical foods are special formulas and foods that are medically necessary for many digestive and inherited metabolic disorders. Until now, coverage has been routinely denied by TRICARE, a health care program of the US Department of Defense. NORD and several of its advocacy partners met with the Armed Services Committee and advocated on behalf of families for whom these special foods are a necessity.  

NORD has commended the US Senate for authorizing medical foods coverage for the military in the National Defense Authorization Act (S.2943). The Senate bill now must be harmonized with the House version passed in May. Medical foods are special formulas and foods that are medically necessary for many digestive and inherited metabolic disorders. Until now, coverage has been routinely denied by TRICARE, a health care program of the US Department of Defense. NORD and several of its advocacy partners met with the Armed Services Committee and advocated on behalf of families for whom these special foods are a necessity.  

NORD and other members of the Zika Coalition have sent a letter to Congress expressing dismay that federal and state agencies have not yet been provided with the funding needed to combat the Zika virus. The letter urges immediate action to address the issue, which it calls a public health emergency. Other members of the Coalition, in addition to NORD, include the American Public Health Association, American Academy of Pediatrics, March of Dimes, and National Hispanic Medical Association.

NORD and other members of the Zika Coalition have sent a letter to Congress expressing dismay that federal and state agencies have not yet been provided with the funding needed to combat the Zika virus. The letter urges immediate action to address the issue, which it calls a public health emergency. Other members of the Coalition, in addition to NORD, include the American Public Health Association, American Academy of Pediatrics, March of Dimes, and National Hispanic Medical Association.

NORD and other members of the Zika Coalition have sent a letter to Congress expressing dismay that federal and state agencies have not yet been provided with the funding needed to combat the Zika virus. The letter urges immediate action to address the issue, which it calls a public health emergency. Other members of the Coalition, in addition to NORD, include the American Public Health Association, American Academy of Pediatrics, March of Dimes, and National Hispanic Medical Association.

An international expert in inborn errors of metabolism, Marshall L. Summar, MD, has been named chairman of the board of directors of NORD. Dr. Summar is Chief of the Division of Genetics and Metabolism at Children’s National Health System in Washington DC.

An international expert in inborn errors of metabolism, Marshall L. Summar, MD, has been named chairman of the board of directors of NORD. Dr. Summar is Chief of the Division of Genetics and Metabolism at Children’s National Health System in Washington DC.

An international expert in inborn errors of metabolism, Marshall L. Summar, MD, has been named chairman of the board of directors of NORD. Dr. Summar is Chief of the Division of Genetics and Metabolism at Children’s National Health System in Washington DC.

The NORD Rare Diseases and Orphan Products Breakthrough Summit will take place October 16-17 in Arlington, Virginia. This annual event draws approximately 500 thought leaders from the rare disease community. Researchers (both professionals and students) are invited to submit poster abstracts to present their rare disease research. This year’s theme for posters is “Life-Transforming Treatments” and there are four topic areas—innovative research, medical education advancement, patient community building, and other topics.

The NORD Rare Diseases and Orphan Products Breakthrough Summit will take place October 16-17 in Arlington, Virginia. This annual event draws approximately 500 thought leaders from the rare disease community. Researchers (both professionals and students) are invited to submit poster abstracts to present their rare disease research. This year’s theme for posters is “Life-Transforming Treatments” and there are four topic areas—innovative research, medical education advancement, patient community building, and other topics.

The NORD Rare Diseases and Orphan Products Breakthrough Summit will take place October 16-17 in Arlington, Virginia. This annual event draws approximately 500 thought leaders from the rare disease community. Researchers (both professionals and students) are invited to submit poster abstracts to present their rare disease research. This year’s theme for posters is “Life-Transforming Treatments” and there are four topic areas—innovative research, medical education advancement, patient community building, and other topics.

On June 2, 2016, the Food and Drug Administration (FDA) issued three final guidance documents related to access to investigational therapies for patients with serious or immediately life-threatening medical conditions who lack therapeutic alternatives.

The first guidance, Individual Patient Expanded Access Applications: Form FDA 3926, describes a streamlined option for licensed physicians to request use of an investigational new drug to treat individual patients who have exhausted other treatment options, including for emergency use. When this form is substituted for the current forms FDA 1571 and 1572, the agency estimates that each submission will take about 45 minutes, which is considerably less time than is required by the current forms.

The second guidance, Expanded Access to Investigational Drugs for Treatment Use—Questions and Answers, responds to frequently asked questions about the implementation of FDA’s regulations on expanded access to investigational drugs.

The third guidance, Charging for Investigational Drugs under an IND—Questions and Answers, provides information for the pharmaceutical industry on frequently asked questions about the implementation of FDA’s regulations related to charging for investigational new drugs in the context of clinical trials or expanded access for treatment.

These documents are intended to simplify and clarify expanded access to help health care professionals, patients, and industry more easily navigate the process and to facilitate patient access to potentially life-saving medications. The FDA has also developed patient and physician fact sheets about expanded access and will host a webinar for health care professionals on July 12th at 1 PM ET to further explain the expanded access process. Information about the webinar will be posted on the FDA website.

On June 2, 2016, the Food and Drug Administration (FDA) issued three final guidance documents related to access to investigational therapies for patients with serious or immediately life-threatening medical conditions who lack therapeutic alternatives.

The first guidance, Individual Patient Expanded Access Applications: Form FDA 3926, describes a streamlined option for licensed physicians to request use of an investigational new drug to treat individual patients who have exhausted other treatment options, including for emergency use. When this form is substituted for the current forms FDA 1571 and 1572, the agency estimates that each submission will take about 45 minutes, which is considerably less time than is required by the current forms.

The second guidance, Expanded Access to Investigational Drugs for Treatment Use—Questions and Answers, responds to frequently asked questions about the implementation of FDA’s regulations on expanded access to investigational drugs.

The third guidance, Charging for Investigational Drugs under an IND—Questions and Answers, provides information for the pharmaceutical industry on frequently asked questions about the implementation of FDA’s regulations related to charging for investigational new drugs in the context of clinical trials or expanded access for treatment.

These documents are intended to simplify and clarify expanded access to help health care professionals, patients, and industry more easily navigate the process and to facilitate patient access to potentially life-saving medications. The FDA has also developed patient and physician fact sheets about expanded access and will host a webinar for health care professionals on July 12th at 1 PM ET to further explain the expanded access process. Information about the webinar will be posted on the FDA website.

On June 2, 2016, the Food and Drug Administration (FDA) issued three final guidance documents related to access to investigational therapies for patients with serious or immediately life-threatening medical conditions who lack therapeutic alternatives.

The first guidance, Individual Patient Expanded Access Applications: Form FDA 3926, describes a streamlined option for licensed physicians to request use of an investigational new drug to treat individual patients who have exhausted other treatment options, including for emergency use. When this form is substituted for the current forms FDA 1571 and 1572, the agency estimates that each submission will take about 45 minutes, which is considerably less time than is required by the current forms.

The second guidance, Expanded Access to Investigational Drugs for Treatment Use—Questions and Answers, responds to frequently asked questions about the implementation of FDA’s regulations on expanded access to investigational drugs.

The third guidance, Charging for Investigational Drugs under an IND—Questions and Answers, provides information for the pharmaceutical industry on frequently asked questions about the implementation of FDA’s regulations related to charging for investigational new drugs in the context of clinical trials or expanded access for treatment.

These documents are intended to simplify and clarify expanded access to help health care professionals, patients, and industry more easily navigate the process and to facilitate patient access to potentially life-saving medications. The FDA has also developed patient and physician fact sheets about expanded access and will host a webinar for health care professionals on July 12th at 1 PM ET to further explain the expanded access process. Information about the webinar will be posted on the FDA website.

The National Organization for Rare Disorders (NORD) has released its State Policy Legislative Tracker for May/June 2016, providing state-by-state information on current legislative initiatives. Read about current activity in your state and learn how to get involved.

The National Organization for Rare Disorders (NORD) has released its State Policy Legislative Tracker for May/June 2016, providing state-by-state information on current legislative initiatives. Read about current activity in your state and learn how to get involved.

The National Organization for Rare Disorders (NORD) has released its State Policy Legislative Tracker for May/June 2016, providing state-by-state information on current legislative initiatives. Read about current activity in your state and learn how to get involved.

The Undiagnosed Diseases Network (UDN) brings together clinical and research experts from across the nation to try to solve the most challenging medical mysteries. Patients with longstanding unexplained symptoms can apply online, with support from their physicians, to receive an in-depth clinical and research evaluation at one of the UDN’s seven medical centers.

The National Organization for Rare Disorders (NORD) and the UDN recently hosted a free webinar for physicians and other medical professionals, patients, and caregivers. The purpose was to promote awareness of this program.

Speakers included the principal investigators from the UDN sites at UCLA and Duke University. In addition, a patient who has participated in the program described her experiences. The program included an overview of the UDN, the application and evaluation process, and financial considerations. NORD administers a patient assistance program to help UDN applicants who qualify on the basis of financial need pay for medical testing required to complete the application process.

The Undiagnosed Diseases Network (UDN) brings together clinical and research experts from across the nation to try to solve the most challenging medical mysteries. Patients with longstanding unexplained symptoms can apply online, with support from their physicians, to receive an in-depth clinical and research evaluation at one of the UDN’s seven medical centers.

The National Organization for Rare Disorders (NORD) and the UDN recently hosted a free webinar for physicians and other medical professionals, patients, and caregivers. The purpose was to promote awareness of this program.

Speakers included the principal investigators from the UDN sites at UCLA and Duke University. In addition, a patient who has participated in the program described her experiences. The program included an overview of the UDN, the application and evaluation process, and financial considerations. NORD administers a patient assistance program to help UDN applicants who qualify on the basis of financial need pay for medical testing required to complete the application process.

The Undiagnosed Diseases Network (UDN) brings together clinical and research experts from across the nation to try to solve the most challenging medical mysteries. Patients with longstanding unexplained symptoms can apply online, with support from their physicians, to receive an in-depth clinical and research evaluation at one of the UDN’s seven medical centers.

The National Organization for Rare Disorders (NORD) and the UDN recently hosted a free webinar for physicians and other medical professionals, patients, and caregivers. The purpose was to promote awareness of this program.

Speakers included the principal investigators from the UDN sites at UCLA and Duke University. In addition, a patient who has participated in the program described her experiences. The program included an overview of the UDN, the application and evaluation process, and financial considerations. NORD administers a patient assistance program to help UDN applicants who qualify on the basis of financial need pay for medical testing required to complete the application process.

Some health care analysts have criticized patient assistance programs (PAPs) as a cause of growth in prescription drug spending and overall health care costs. The National Organization for Rare Disorders (NORD) has published a position statement to address this issue and explain the essential function of charitable assistance programs for patients with rare diseases. Specifically, NORD’s position is that PAPs do not inflate the cost of treatment and that they ensure patient access to lifesaving therapies. Read NORD’s position paper.

Some health care analysts have criticized patient assistance programs (PAPs) as a cause of growth in prescription drug spending and overall health care costs. The National Organization for Rare Disorders (NORD) has published a position statement to address this issue and explain the essential function of charitable assistance programs for patients with rare diseases. Specifically, NORD’s position is that PAPs do not inflate the cost of treatment and that they ensure patient access to lifesaving therapies. Read NORD’s position paper.

Some health care analysts have criticized patient assistance programs (PAPs) as a cause of growth in prescription drug spending and overall health care costs. The National Organization for Rare Disorders (NORD) has published a position statement to address this issue and explain the essential function of charitable assistance programs for patients with rare diseases. Specifically, NORD’s position is that PAPs do not inflate the cost of treatment and that they ensure patient access to lifesaving therapies. Read NORD’s position paper.