“Catalyst Pharms., Inc. v. Becerra threatens to undermine 40 years of practice and progress enabled by the Orphan Drug Act, which has benefited millions of Americans and their families facing rare disease diagnoses,” said Peter L. Saltonstall, President and CEO, NORD. “Still, there are approximately 25-30 million Americans living with a rare disease and more than 90% of the 7,000 known rare diseases do not yet have an FDA-approved treatment. We are very encouraged by the legislation in both the House and Senate to clarify that orphan drug exclusivity is awarded only for the specific use that is approved by the FDA. This clarification will maintain appropriate incentives to encourage rare disease drug development as intended in the Orphan Drug Act with a goal of addressing the still significant unmet needs of many in the rare disease community.” 

The ODA provides a set of incentives to support research and development into drugs for rare diseases, which had been largely neglected by the pharmaceutical industry before the 1983 law. One of the key incentives is a seven-year term of “exclusivity,” or market protection from competition, for the orphan drug once approved and marketed. The law established a two-part process for obtaining orphan drug exclusivity. First, at an early stage in development, a company can request that FDA “designate” the drug as an orphan drug to prevent, diagnose or treat a rare disease or condition. Once a company receives this designation, the company can access other ODA incentives, including tax credits for the research and clinical testing on the drug. Second, after completing the necessary clinical studies and obtaining FDA approval, the drug is then awarded exclusivity that protects the specific use of the drug that is approved. 

However, the recent 11th Circuit decision in the case of Catalyst Pharms., Inc. v. Becerra, if allowed to stand, would turn this system on its head. The Court rejected FDA’s decades-long interpretation of the ODA that the exclusivity protects the “use or indication” ultimately approved. The Court instead held that the rare disease designated at the outset of the drug development process dictates the scope of the orphan drug exclusivity. NORD believes this is an incorrect interpretation of the statute and is grateful to see Congress working to correct and clarify the law.  

In the absence of a legislative fix, NORD believes there would be fewer orphan drugs approved for fewer special patient populations. That is not the goal of the ODA, and it is not in the best interest of the rare disease community. In most cases, the orphan designation is intentionally broader than the use ultimately approved. For instance, a drug might be designated for the treatment of Fabry’s disease, a rare lysosomal storage disorder. After conducting studies in the disease, the sponsor may have only obtained data sufficient to support a narrower population than the entire patient population with Fabry’s disease and seek approval for the treatment of only adults with the disease. Similarly, many orphan drugs designated for cystic fibrosis are designated for the disease broadly but are only approved for use in patients with a specific mutation.  

If the 11th Circuit decision is left in place, the law would create an incentive for sponsors to seek broader designations for an entire rare disease at the outset, leaving little incentive to continue to study the safety and efficacy that drug in special populations, like children. More than half of people with rare diseases are children, so the implications of this Court ruling have the potential to be significant. 

“NORD is grateful that Energy and Commerce Committee leadership and Senators Baldwin and Cassidy have recognized the threat posed by the 11th Circuit’s interpretation of the Orphan Drug Act,” said Heidi Ross, Vice President, Policy and Regulatory Affairs, NORD. “This issue must be addressed swiftly to ensure access to safe and effective drugs for specific rare disease populations is maintained and future research and development efforts for new treatments for rare diseases are appropriately incentivized.”

To learn more about NORD’s policy work and how to get involved, visit: http://bit.ly/Policy-Issues. 

About the National Organization for Rare Disorders (NORD)
The National Organization for Rare Disorders (NORD) is the leading independent advocacy organization representing all patients and families affected by rare diseases in the United States. NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. Since then, the organization has led the way in voicing the needs of the rare disease community, driving supportive policies, furthering education, advancing medical research, and providing patient and family services for those who need them most. Together with over 300 disease-specific member organizations, more than 17,000 Rare Action Network advocates across all 50 states, and national and global partners, NORD delivers on its mission to improve the lives of those impacted by rare diseases. Visit rarediseases.org. 

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People living with rare diseases must be able to afford the therapies that come to the market. The high cost of prescription drugs has a direct impact on the ability of patients to access lifesaving care. The small patient populations and medical complexity associated with rare diseases can lead to costly therapies, but it is vital that these therapies remain affordable and, therefore, accessible to rare disease patients. Furthermore, these therapies must remain affordable so that the sustainability of the healthcare system as a whole is preserved.

As all levels of government and a variety of stakeholders look for legislative and regulatory tools to find ways to ensure that medicines are affordable, NORD has compiled the following principles to help guide NORD’s engagement with these issues in a deliberate and transparent manner:

1. The policy must maintain FDA’s standards of safe and effective therapies. Rare disease patients in the United States rely on the quality and safety of FDA-approved medicines. Any policy to lower the cost of therapies must not lower the safety and effectiveness of the medicines rare disease patients need.
2. The policy must not raise out-of-pocket costs for rare disease patients. Policies to lower the cost of prescription drugs must not inadvertently raise patients’ out-of-pocket costs.
3. The policy must not decrease patients’ coverage of necessary therapies prescribed by their provider. Some policies may negatively impact insurance coverage for rare disease treatments, making these treatments unaffordable and, therefore, inaccessible for patients with rare diseases. Patients should be able to access the therapy that they and their physician believe is right for them.
4. The policy must not place an undue burden on innovation of new therapies. Rare disease patients rely upon and, in many cases, their lives depend on continued innovation by the pharmaceutical industry. However, innovation is meaningless to patients if they cannot afford the therapies that are ultimately developed. Efforts to improve affordability and lower costs should be balanced with the need to sustain innovation.
5. The policy must be supported by reliable data. Policy interventions must be well-researched and supported by empirical evidence.

From its inception, NORD has advocated for the development of safe and effective rare disease therapies so that people living with rare diseases can benefit from novel therapeutic development just as patients with common diseases do. NORD supports the 1983 Orphan Drug Act, which is one way to further this goal. In the three and a half decades following the law’s enactment, over 770 therapies indicated to treat a rare disease have come to market, whereas prior to the ODA there were only 34.

NORD will continue its work to educate all stakeholders on the successes and importance of the Orphan Drug Act. These efforts will include the distribution of robust data collected from trustworthy sources, such as its recently commissioned IQVIA reports, with the goal of ensuring that lawmakers are making decisions pertaining to rare diseases and orphan therapies based upon evidence and facts.

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Now in its 36th year, the ODA has successfully encouraged the pharmaceutical industry to develop therapies for those with rare diseases. Prior to the ODA’s enactment in 1983, only 34 therapies for rare diseases existed. Now, there are over 770 therapies on the market today to treat rare diseases. In addition, a majority of the novel drugs approved by the Food and Drug Administration (FDA) in 2018 were for rare diseases, the first time this has ever occurred. This incredible progress can be largely credited to the incentives within the ODA.

There’s more work to be done. Over 90 percent of rare diseases are still without a single treatment approved by the FDA. Consequently, the ODA must remain strong, and Congress must keep this critical law for our community in place.

“At NORD, we are very excited that Representatives Butterfield and Bilirakis have introduced this Resolution,” said Peter L. Saltonstall, President and CEO of NORD. “We are eager to work with them and their supportive colleagues to not only build support for this important resolution, but ensure the Orphan Drug Act itself remains strong.”

We are asking the rare disease patient community to ask all Representatives to become a cosponsor of this Resolution and show their support of the ODA! Please consider reaching out to your representative today.

For more information on the ODA, check out NORD’s RareInsights webpage here.

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This year marks the 35th anniversary for the ODA. That means 35 years of increased hope and treatments for the rare disease community. Prior to the ODA, there were only 34 therapies indicated to treat a rare disease. Now, there are over 700 and counting!

There’s more work to be done, though. Approximately 95 percent of rare diseases are still without a single treatment approved by the Food and Drug Administration (FDA). Consequently, the ODA must remain strong!

Last year, Congress cut one of the incentives for companies to manufacture orphan drugs contained within the ODA in half. We cannot let that happen again! That’s why we are so excited that Senator Hatch and Representatives Lance and Butterfield have introduced this Resolution. We hope you will join us in asking all members of Congress to become a cosponsor of this Resolution and show their support of the ODA!

For more information on the Resolution, click here. Additionally, for more information on the ODA generally, check out NORD’s new one-pager here.

Support the Orphan Drug  Act Resolution!

Email your Senators and Representative and ask them to demonstrate their support for the ODA by cosponsoring H.Res.1154 and S.Res.690!

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NORD President and CEO Peter L. Saltonstall stated, “We respectfully request the U.S. Court of Appeals to reverse the lower court’s decision, which used the principle of ‘willful blindness’ to erroneously support the approval of a generic drug that utilized the dosage form of a second orphan-protected indication of the same drug. Approval should have been limited to the dosage form of the first now-unprotected indication.”

He continued: “Patient groups, parents, caregivers, researchers, and doctors work tirelessly to improve life and health for people with rare diseases, and we need policies at the highest level, as reflected in the Orphan Drug Act (ODA), to encourage drug development for this large unmet medical need. Preserving the integrity of the ODA is one of NORD’s most important responsibilities on behalf of our members.”

In its amicus brief in the case of Spectrum vs. Burwell, NORD said that “where FDA policy, information in its files, or decisions the agency has made, either confirm or imply intent to usurp an exclusive indication, then FDA should take it into consideration what they know.  They should not be able to engage in ‘willful blindness’ and ignore some distinguishing characteristics that separate a protected second indication from an unprotected first indication.  In this case, an ANDA should be limited to the original ‘no longer protected’ labeling and the physical characteristics of the original orphan product.  Allowing marketing beyond that undercuts the ODA.”

NORD’s founders played an active role in the 1983 passage of the Orphan Drug Act (ODA) under which there are three major provisions to spur the development of orphan drugs: the Orphan Drug Grant Program, the Orphan Drug Tax Credit, and market exclusivity.

Citing the Office of Inspector General of the Department of Health & Human Services, NORD said in its amicus brief that the 7-year market exclusivity has been the most effective of the incentives for spurring orphan drug development, providing the opportunity for manufacturers to recover their initial investments in research and development (estimated to be between $1.5 and $2.6 billion) for new orphan therapies. In this case, the approval of the generic drug was contrary to the intent of the law by undermining the exclusivity.

According to the National Institutes of Health, there are approximately 7,000 rare diseases that affect 30 million Americans, or one in 10 people in the U.S.  The vast majority of rare diseases have no FDA-approved treatment.  Two-thirds of rare disease patients are children.

“NORD’s goal is to help rare disease patients by facilitating and accelerating the development of safe, effective treatments whenever possible,” added Saltonstall.  “While we disagree with the FDA in this situation, we look forward to continuing our constructive relationship with the FDA.”

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About National Organization for Rare Disorders (NORD)

The National Organization for Rare Disorders (NORD), established in 1983 by parents and families of rare disease patients, is the leading, independent nonprofit organization representing all patients and families affected by rare diseases in the U.S.  NORD provides programs of advocacy, education, research and patient/family services to improve the lives of all people living with rare diseases. NORD represents more than 230 disease-specific member organizations and partners with many other organizations in specific causes of importance to the rare disease patient community. Follow NORD at www.rarediseases.org/ and on Twitter at @RareDiseases.

Media Contact:

Jennifer Huron, jhuron@rarediseases.org, 203.304.7258

Attachments: Amicus Brief

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