Rare Disease Report

NORD News for October 2017 - The Voice of the Community

OCTOBER 20, 2017
New Study Refutes Idea That Orphan Drugs Are Raising Healthcare Costs
An important new Quintiles/IMS report, released at the recent NORD Summit, provides data showing that orphan drugs are not a significant factor in overall healthcare spending. The study, commissioned by NORD, examined the impact of the Orphan Drug Act (ODA) since it was implemented in 1983, and the use and costs of orphan drugs.

The report is important because many people assume that orphan drugs, which tend to be expensive, are driving up the cost of healthcare for all. However, because the volume of usage of these drugs is low, they represent only a small percentage of overall healthcare spending, according to the Quintiles/IMS data.

The ODA was originally needed because pharmaceutical companies couldn’t afford to develop therapies for rare diseases because they couldn’t recover their investment. Incentives in the ODA, such as seven years of marketing exclusivity and a tax credit for clinical trial expenses, make development of these therapies financially feasible. 

The Quintiles/IMS report was released in tandem with a NORD white paper, which provides additional analysis and context. Both documents are available on the NORD website.

Other highlights of the 2017 NORD Summit included keynote addresses by FDA Commissioner Scott Gottlieb, MD, and Mike Porath, founder/CEO of The Mighty. Dr. Gottlieb talked about FDA’s priorities with respect to rare diseases, the impact of scientific advances and FDA’s work to reduce the backlog of orphan designation applications and to make review of orphan products more efficient.

Bioethicist Arthur Caplan PhD of New York University School of Medicine moderated a panel on “Ethical Guidelines for Patient Organizations and Industry to Collaborate.”

Other topics over the two-day summit included the outlook for gene therapy, use of mobile apps in clinical trials, promoting earlier diagnosis, “right-to-try” laws, current policy priorities, developing a global strategy for rare diseases, data-sharing and the challenge of healthcare costs.

On Day 2, a panel of senior staff from FDA drew a standing-room-only crowd for a “Current Topics from FDA” session. Topics included “Leadership Perspectives on Orphan Drug Review”, “Perspectives on Gene Therapy” and “Patient Voice and Engagement with the FDA.”

Nominations Are Open for NORD 2018 Rare Impact Awards
Online nominations are being accepted now for NORD’s 2018 Rare Impact Awards. These awards honor individuals, organizations or companies making a difference in the fight against rare diseases.

January 12 is the deadline to submit nominations. The awards are presented in May at NORD’s annual Rare Impact Awards celebration in Washington DC. Because 2018 will mark the 35th anniversary of NORD and the Orphan Drug Act, the event will have special significance. Awards will be presented in four categories:
  • Advocacy
  • Education
  • Research
  • Patient Assistance
NORD Board Chair to Moderate SSA Compassionate Allowances Panel
On November 7, the Social Security Administration will host a National Disability Forum on Compassionate Allowances and Rare Diseases in Washington DC.

Marshall Summar MD, chairman of NORD’s Board of Directors, will moderate a panel of speakers from the National Institutes of Health (NIH) and rare disease patient advocacy organizations who will talk about the challenges of living with rare diseases.

Patient representatives will suggest specific rare diseases for consideration for the SSA Compassionate Allowances list.

This is a list of diagnoses that qualify for expedited review of applications for Social Security Disability Assistance. The Compassionate Allowances Initiative is a way to quickly identify diseases and medical conditions that, by definition, meet SSA’s standards for disability benefits.

The initiative was established following advocacy by NORD and others to alert SSA that people with rare diseases were often having to wait for long periods of time after applying for disability assistance because little information was available about their medical needs.

Diseases may be submitted online for consideration to be added to the list. Read about the Compassionate Allowances Initiative here. View the current list of diseases. Submit a disease for consideration here.  

FDA to Host Workshop on Effective Advocacy in Rare Disease Drug Development
FDA’s Center for Drug Evaluation and Research (CDER) will host a free public workshop on “Strategies, Tools and Best Practices in Rare Diseases Drug Development on Monday, Oct. 30, at FDA’s White Oak Campus in Silver Spring, MD. Space is limited and advance registration is required.

This workshop is open to advocacy organizations, patients, caregivers, healthcare professionals, and representatives of government and industry. Online registration is available here.


Adrenal Insufficiency United (AIU) Conference
Registration is now open for AIU’s first-ever conference. The event will take place March 23-25 in Kansas City. Leading experts will speak on a wide variety of topics important to the AI community. More.  

American Partnership for Eosinophilic Disorders (APFED) Pilot Grants
APFED is now accepting applications for three 2018 HOPE Pilot Grants. This program was established to allow investigators from various disciplines to initiate new projects related to eosinophilic-associated diseases. Find additional information here.

Also, join APFED for an evening of music and fundraising in New York City on November 10. The event will feature Mike DelGuidice and Big Shot, celebrating the music of Billy Joel. More.

Amyloidosis Foundation Annual Research Benefit
This event will take place on October 27 in Pittsburgh. Since 2005, the Foundation has awarded more than $1.4 million to young researchers pursuing a cure for amyloidosis. More.

Bridge the Gap – SYNGAP Education and Research Foundation
Register today for the first annual STEP UP for SYNGAP1 Walk at the Houston Zoo. Come walk a 5K among the animals and enjoy a day of family fun on Saturday, December 9.

CTMC OVM – Netherlands
On November 4, CTMC OVM – Netherlands will again host its global conference in Leusden. This year will mark the organization’s 20th anniversary.

MDS Foundation Patient and Family/Caregiver Forum
Join the MDS Foundation for free one-day conferences in Atlanta GA and Stanford CA on October 28. These events are for individuals newly diagnosed with a myelodysplastic syndrome, long-term survivors and caregivers. Additional information is available here.

Morgan Leary Vaughan Fund Receives Petit Family Foundation Grant
The Morgan Leary Vaughan Fund has received a grant from the Petit Family Foundation in support of the inaugural “Speaking of NEC: Unplugged” event, a one-day regional conference on identifying practical solutions to reduce the devastating effects of necrotizing enterocolitis on premature infants and their families. The event will take place in Hartford CT in the spring of 2018.

Neuroendocrine Tumor Research Foundation Announces Change of Leadership
After six successful years, Ron Hollander will be stepping down as executive director of the Neuroendocrine Tumor Research Foundation. His successor will be Elyse Gellerman. More.

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