Update on the Use and Cost of Orphan Drugs

If a treatment or cure for Kennedy's Disease (SBMA) is found, the orphan drug act will be helpful. Below is an update from NORD.

Updated Study Analyzes Use and Cost of Orphan Drugs

Median annual cost for top 10 rare disease therapies is less than $10,000 per year

Washington, D.C., October 18, 2018— Despite a record-breaking number of new approvals, orphan drugs remain a nominal part of overall drug spending, accounting for only 9.6% of total sales in the U.S. in 2017, according to a new study released today by the IQVIA Institute. The study was commissioned by the National Organization for Rare Disorders (NORD) for the second year in a row to show trends in orphan drug usage and costs.

The major findings of the report are as follows:

• The Orphan Drug Act remains successful in encouraging the development and approval of orphan drugs. In 2017, the Food and Drug Administration approved 80 orphan indications and 57 just within the first eight months of 2018 -- shattering previous records. 
• The Orphan Drug Act has not been a major driver of drug pricing in the U.S. In 2017, the median price of the top 10 rare disease therapies used by the greatest number of patients was less than $10,000 per year. 
• In 2017, orphan drugs remained a small part of the overall medicine budget. Total drug spending in the United States in 2017 was $451 billion, with almost 56% spent on non-orphan traditional drugs, 34.7% spent on non-orphan specialty drugs and 9.6% spent on orphan indications of approved orphan drugs. That is a 1.7 percentage point increase over 2016, which is in line with increased FDA approvals. 

“2017 was a year of innovation and advancements in rare diseases, with more FDA approvals of treatments for rare diseases than ever before,” said NORD President and CEO Peter L. Saltonstall. “The recent IQVIA Institute study highlights the enduring importance of the Orphan Drug Act in making that progress possible.”

“It is critical to note that orphan drugs remain a sliver of overall drug spending in the U.S.,” Saltonstall said. “NORD remains committed to shining a light on the simple truths that illustrate how the Orphan Drug Act continues to be vital to the future of rare disease drug research and development.”

About 7,000 rare diseases have been identified, affecting approximately 30 million Americans. Many affect only a few hundred or a few thousand individuals. Rare diseases tend to be chronic, serious and life-threatening. More than 80 percent are believed to be genetic.

Information on the report, including a link to the study, is available on the NORD website at www.rarediseases.org/rareinsights.

NORD Issues Statement on President Trump’s Address to Congress

The process for expediting certain drugs has improved dramatically over the last decade. Yet, we need to keep the pressure on our representatives - lest they forget. There is still a lot of work to be done in finding a treatment for Kennedy's Disease.

NORD Issues Statement on President Trump’s Address to Congress

Posted by Jennifer Huron

NORD Issues Statement on President Trump’s Address to Congress

Washington, D.C., March 1, 2017—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, issued the following statement in response to President Trump’s first speech to Congress:

“Yesterday evening, President Trump recognized Rare Disease Day and the 30 million Americans living with a rare disease in his first address to Congress. We are grateful for his recognition of the day that raises awareness for all individuals with rare diseases and their families. We are elated that he is joining us in our efforts.

President Trump also recognized Megan Crowley, and her father John, as shining examples of the untiring, steadfast commitment individuals with rare diseases and their families bring to finding treatments and cures for their disease. The Crowleys are one of many superstar families that deserve recognition, and we are thrilled that their tireless work was recognized on such a large stage.

The President continued, stating, ‘…our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan’s life, from reaching those in need. If we slash the restraints, not just at the FDA but across our Government, then we will be blessed with far more miracles like Megan.’

We agree that FDA review processes can be improved upon to expedite the development and review of orphan drugs. Yet we disagree with the President that restraints must be slashed, or that the approval process at the FDA is preventing advances from reaching those in need.

Between 2008 and 2013, 87 percent of the 113 rare disease treatments reviewed by the FDA received an expedited review, compared to 35 percent of treatments for common diseases.[i] Seventy-eight percent of rare disease treatments were approved using one or more flexible development approaches (generally defined as an approach that does not include two adequate and well-controlled trials or uses novel endpoints).[ii]

For patients with immediately life-threatening illnesses who cannot participate in clinical trials, the FDA approves 99.5 percent of all expanded access requests submitted by physicians and companies. ..."

To read the rest of the statement follow this link: NORD Statement 

Orphan Product Extensions Now, Accelerating Cures and Treatments

Just received this update on the proposed OPEN Act. It is stalled in the Senate and we need to band together to show our support. Consider sending a message to your representatives. It takes less than three minutes to do and it could make a difference, Thank you.

Dear Rare Disease Advocate,

As you may already know, we are working to pass legislation called the OPEN ACT (Orphan Product Extensions Now, Accelerating Cures & Treatments) that could double the number of treatments available to rare disease patients.

The OPEN ACT was passed in the House of Representatives last July as part of the 21st Century Cures Act (HR 6). However, the companion legislation in the Senate has stalled, and we are running out of time to get it enacted into law in an election year.

Please CLICK HERE to send an email to your legislators asking them to co-sponsor this lifesaving legislation.

This bipartisan bill could bring hundreds of safe, effective, and affordable medicines to rare disease patients within the next several years by incentivizing drug makers to repurpose therapies for the treatment of life-threatening rare diseases. We need your help to make this bill a law.

Thanks again for your support.

Update on 21st Century Cures Legislation

This is another important bill that everyone living with a rare disorder (including Kennedy's Disease) needs to follow.

NORD Issues Statement on Today’s Approval  of the 21st Century Cures Initiative Washington, D.C. – May 21, 2015 – The following statement was issued by Peter L. Saltonstall, President and CEO of NORD, on the approval by the House Energy and Commerce Committee of the 21st Century Cures legislative initiative and the introduction of the OPEN Act in the United States Senate.  NORD congratulates the House Energy and Commerce Committee for  unanimously approving the 21st Century Cures initiative.  As Chairman Fred  Upton (R-MI) said, “This historic day marks a big bipartisan step toward our  path to cures.”  Today also marks the introduction in the Senate of the OPEN  Act, a bill that aims to greater incentivize orphan product development. We look forward to working with the House as it considers the bill approved   today by the Committee, and with the Senate as it continues its Medical  Innovation Initiative and considers the OPEN Act. The bill approved today contains provisions that are critically important for the  rare disease community. It also puts the patient at the center of drug approval,  strengthens the FDA’s ability to streamline clinical trials, reauthorizes a critical   program for rare pediatric disease drug development, and further incentivizes  development of orphan products. We hope the legislation, which includes sorely  needed additional funding for the National Institutes of Health and the Food and  Drug Administration, will foster an environment that is conducive to the  development of new therapies and improve patients’ access to them.   On behalf of the rare disease community, NORD thanks the Committee for its  months of collaboration, thoughtfulness and hard work.  Today’s vote  underscores the bipartisan commitment to move this legislation through the  House and we hope the same spirit of collaboration leads to prompt  consideration by the Senate. Peter L. Saltonstall President and CEO, National Organization for Rare Disorders (NORD)

30th Anniversary of the Orphan Drug Act

Most of you know I am an advocate of NORD (National Organization for Rare Disorders).  They have done a lot of good representing those of us living with rare disorders including Kennedy’s Disease.  This year is the 30th anniversary  of the Orphan Drug Act.  Below is a press release the KDA received today.

NORD Press Release

 

Launching a Year-Long Celebration: 30th Anniversary of the Orphan Drug Act and NORD

Washington DC, Jan. 4, 2013----Thirty years ago today, President Ronald Reagan signed the Orphan Drug Act into law to encourage the development of treatments for the millions of Americans with rare diseases.

The new law represented a triumph of patient advocates working with government partners, the media, and other supporters to address a critically important unmet need.

To get the law passed, an ad hoc coalition of leaders of rare disease patient organizations gave themselves a name -- the National Organization for Rare Disorders (NORD) -- and taught themselves how to be advocates for the desperate patients and families they represented.

Actor Jack Klugman, whose brother was dying of a rare disease, lent his support with an episode of his popular TV show, Quincy, M.E., dedicated to showing the need for treatments for rare disease patients.  Klugman later testified before a Congressional subcommittee and, when the bill became stalled in Congress, aired another Quincy episode that helped get the bill passed.

Exactly four months after President Reagan signed the bill into law, on May 4, 1983, the coalition of patient advocates formally established NORD as a nonprofit organization to provide advocacy, education, research and patient/family services for all Americans affected by rare diseases.

"It was an amazing accomplishment to get this desperately needed law enacted at that time," says NORD President and CEO Peter L. Saltonstall.  "Today and throughout 2013, we will be saluting all the pioneers who helped make it happen, celebrating progress to date, and working with our partners to find ways to accelerate the pace of progress."

Saltonstall added that the recent death of Jack Klugman (on Dec. 24th) added poignancy to the occasion.

NORD's president for its first 25 years, Abbey S. Meyers, is widely considered the primary patient advocate for the Orphan Drug Act.  She retired in 2008 and Saltonstall joined the organization at that time.

Government partners at the National Institutes of Health (NIH) and Food and Drug Administration (FDA) have provided major support and encouragement to the rare disease community through their implementation of the law.

On Monday, the FDA Office of Orphan Products Development will host an event for FDA staff and guests to observe the anniversary of the law.  FDA Commissioner Margaret Hamburg, MD, will speak, as will Saltonstall, with a video message from Meyers.  Special guests will include individuals and representatives of organizations being honored as "rare disease heroes" by FDA during the anniversary year.

NORD's year-long observance of the 30th anniversary will include a special section on its website where rare disease patients, researchers, and others will be invited to share stories about rare disease milestones and achievements.

On Rare Disease Day 2013 (Feb. 28th), which NORD sponsors nationally, there will be special activities to honor the history of the rare disease community.  In addition, NORD's annual gala in May and annual conference in October will have an anniversary theme.  There will be other special activities throughout the year.

The Orphan Drug Act provides financial incentives such as seven years of marketing exclusivity for products developed to treat diseases affecting fewer than 200,000 Americans.  There are 7,000 diseases considered rare in the U.S. affecting nearly 30 million Americans, or about 1 in 10 people.
In the decade before the law was passed, only 10 new drugs were developed by industry for patients with rare diseases.  Since 1983, more than 2,700 potential treatments have entered the research pipeline as "orphan products" and more than 400 have been approved by FDA.

Highlights over the years since the law was enacted have included the Rare Diseases Act of 2002, promoted by NORD, which made possible a Rare Diseases Clinical Research Network at NIH, and most recently the FDA Safety and Innovation Act of 2012, which includes many major provisions related to rare diseases.

"This year, we will be saluting the progress made to date but we'll also be focusing on the fact that much remains to be done," Saltonstall said.  "Most people with rare diseases still have no FDA-approved treatment.  And, even when treatments exist, many patients have trouble accessing them.

"During this anniversary year, NORD will move forward with renewed commitment toward the development of safe, effective treatments for all, and assurance that all patients with serious medical problems will have access to the treatments and services they need."