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Rachel V Thomas

US FDA's New Framework Calls For Speeding Up Approvals For Rare Disease Therapies

US FDA's New Framework Calls For Speeding Up Approvals For Rare Disease Therapies

The US Food and Drug Administration (FDA) today recommended a new framework for accelerating approvals for the development of individualized therapies for rare and ultra-rare diseases.

The Plausible Mechanism Framework, issued by the FDA's Center for Biologics Evaluation and Research and Center for Drug Evaluation and Research, is the first such framework by the regulator in 25 years.

The draft guidance can help drugmakers with cutting-edge personalized treatments for patients with ultra-rare diseases to be used as a basis for FDA approval.

Also Read: <a href="https://www.healthandme.com/health-news/zepbound-outperforms-other-weight-loss-drugs-more-details-inside-article-153680434" data-type="tilCustomLink" target="_blank">Zepbound Outperforms Other Weight Loss Drugs, More Details Inside</a>

The move is expected to drive innovation among drugmakers to develop life-saving treatments for patients with ultra-rare diseases, that are not only safe but also low-cost.

Importantly, with the new framework drugmakers can depend on small, well-controlled studies in cases where traditional randomized controlled trials are not possible due to small patient populations. 

“President Donald Trump promised to accelerate cures for American families -- and we are delivering, especially for children with ultra-rare diseases who cannot afford to wait,” said Health and Human Services Secretary Robert F. Kennedy, Jr.

“We are cutting unnecessary red tape, aligning regulation with modern biology, and clearing a path for breakthrough treatments to reach the patients who need them most,” he added. 

The draft guidance focuses on genome editing and RNA-based therapies such as antisense oligonucleotides. 

The therapies must target a specific genetic, cellular, or molecular abnormality. 

They must also be designed to correct or modify the underlying cause of the disease. The key criteria for therapies include:<ul><li>Finding the disease-causing abnormality</li><li>Demonstrating that the therapy targets the root cause or proximate biological pathway</li><li>Relying on well-characterized natural history data in untreated patients</li><li>Confirming successful target drugging or editing</li></ul>

“This guidance is a critical step the FDA is taking to tailor our regulatory approach to patients with ultra-rare conditions,” said FDA Commissioner Marty Makary.

“It is our priority to remove barriers and exercise regulatory flexibility to encourage scientific advances and deliver more cures and meaningful treatments for patients suffering from rare diseases,” Makary added. 

<h2>What Is A Rare Disease </h2>

The World Health Organization (WHO) defines a rare disease as an often debilitating, chronic, or degenerative condition affecting 1 or fewer per 1,000 population.

Rare diseases lack of epidemiological data, are at high risk of misdiagnosis, and often also lack of effective treatments. 

According to the WHO ICD-11 (International Classification of Diseases), there are over 5,500 rare diseases. It also assigns unique identifiers (URIs) to them for better tracking. 

There are estimated to be over 7,000 distinct rare diseases affecting more than 300 million people globally. 

The FDA hosted Rare Disease Day, a virtual public meeting, on February 23, in global observance of Rare Disease Week.

The theme “Moving Forward. Looking Ahead. An Event for Patients" explored ways to engage and collaborate with patients and their communities to support and accelerate the development of medical products for rare diseases.

Globally the day is observed on February 28 (February 29 in case of a leap year).

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The US Food and Drug Administration (FDA) today recommended a new framework for accelerating approvals for the development of individualized therapies for rare and ultra-rare diseases. The Plausible Mechanism Framework, issued by the FDA's Center for Biologics Evaluation and Research and Center for Drug Evaluation and Research, is the first such framework by the regulator in 25 years. The draft guidance can help drugmakers with cutting-edge personalized treatments for patients with ultra-rare diseases to be used as a basis for FDA approval.Also Read: Zepbound Outperforms Other Weight Loss Drugs, More Details Inside The move is expected to drive innovation among drugmakers to develop life-saving treatments for patients with ultra-rare diseases, that are not only safe but also low-cost.Importantly, with the new framework drugmakers can depend on small, well-controlled studies in cases where traditional randomized controlled trials are not possible due to small patient populations. “President Donald Trump promised to accelerate cures for American families -- and we are delivering, especially for children with ultra-rare diseases who cannot afford to wait,” said Health and Human Services Secretary Robert F. Kennedy, Jr. “We are cutting unnecessary red tape, aligning regulation with modern biology, and clearing a path for breakthrough treatments to reach the patients who need them most,” he added. What Does The Framework Propose The draft guidance focuses on genome editing and RNA-based therapies such as antisense oligonucleotides. The therapies must target a specific genetic, cellular, or molecular abnormality. They must also be designed to correct or modify the underlying cause of the disease. The key criteria for therapies include:Finding the disease-causing abnormalityDemonstrating that the therapy targets the root cause or proximate biological pathwayRelying on well-characterized natural history data in untreated patientsConfirming successful target drugging or editing“This guidance is a critical step the FDA is taking to tailor our regulatory approach to patients with ultra-rare conditions,” said FDA Commissioner Marty Makary. “It is our priority to remove barriers and exercise regulatory flexibility to encourage scientific advances and deliver more cures and meaningful treatments for patients suffering from rare diseases,” Makary added.  What Is A Rare Disease The World Health Organization (WHO) defines a rare disease as an often debilitating, chronic, or degenerative condition affecting 1 or fewer per 1,000 population.  Rare diseases lack of epidemiological data, are at high risk of misdiagnosis, and often also lack of effective treatments. According to the WHO ICD-11 (International Classification of Diseases), there are over 5,500 rare diseases. It also assigns unique identifiers (URIs) to them for better tracking. There are estimated to be over 7,000 distinct rare diseases affecting more than 300 million people globally.  Rare Disease Day 2026 The FDA hosted Rare Disease Day, a virtual public meeting, on February 23, in global observance of Rare Disease Week. The theme “Moving Forward. Looking Ahead. An Event for Patients" explored ways to engage and collaborate with patients and their communities to support and accelerate the development of medical products for rare diseases.Globally the day is observed on February 28 (February 29 in case of a leap year).