FDA new measures!

On May 10, 2022, the FDA's Center for Drug Evaluation and Research (CDER) announced the launch of a new Accelerated Rare Disease Therapy (ARC) program

The FDA defines a rare disease as any disease that affects fewer than 200,000 people in the United States

• Endpoint selection can be complicated if knowledge of the natural history of the disease is limit.

• Small patient populations can make it difficult to conduct and interpret rare disease clinical tria.

Through participation in scientific and regulatory innovation, CDER's ARC program will help support the development and approval of safe and effective treatments for the more than 30 million rare disease patients in the United Stat.

What does CDER's ARC program do?

The CDER Rare Disease Accelerated Treatment (ARC) program was launched in May 202 The vision of CDER's ARC program is to accelerate and augment the development of effective and safe treatment options that address the unmet needs of patients with rare diseases

The project leverages the expertise and execution of the CDER platform to provide macro strategy and coordination of CDER's rare disease activiti.

The main responsibilities of the rare disease team:

• Coordinate the development of CDER policy, review procedures and training for the treatment of rare diseas.

• Assist externally in the development and maintenance of good science as the basis for developing treatments for rare diseas.

• Collaborate with external and internal rare disease stakeholders to advance the development of rare disease treatmen.

• Maintained a partnership with CDER's review division to promote consistency and innovation in the review of treatments for rare diseas.

• Collaborate with international regulatory bodies to discuss and exchange scientific and regulatory information related to rare diseas.

What new drugs for rare diseases have been approved by CDER recently?

Over the past decade or so, we have seen an upward trend in the proportion of drugs used to treat rare or "rare" diseas.

Just two months into 2022, CDER has approved four new drugs for patients with rare diseases in oncology and hematolo.

• The first treatment for uveal melanoma (a rare cancer that develops in the uvea of ​​the eye) that has spread to other parts of the body or cannot be removed surgical.

• The first therapy to reduce the need for red blood cell transfusions due to hemolysis (destruction of red blood cells) in adults with cold agglutinin disease (a rare form of anemi.

• The first therapy to treat anemia in adults with pyruvate kinase deficiency, a genetic disorder that causes premature destruction of red blood cel.

• A medicine for adults with intermediate or high risk of primary or secondary myelofibrosis, a rare bone marrow disorder in which platelets (thrombocytes) are l.

What is CDER doing for rare diseases?

Despite some progress, the vast majority of rare disease treatments remain unapprov.

To this end, CDER is working to support the development of orphan dru.

CDER believes that data sharing is critical to advancing drug development, especially for rare diseases, and will continue its important collaboration with the Critical Path Institute to develop a rare disease treatment acceleration-data and analytics platfo.

The platform provides a database and hub designed to facilitate the secure sharing of existing patient-level data and encourage standardization of new data collecti.

It allows authorized users to access patient-level clinical data to better understand disease progression and disease heterogeneity (or differences between patients with the same disease) in rare disease patient populatio.

This, in turn, can inform trial design, endpoint selection and other important considerations in drug developme.

Additionally, as part of the Standard Core Clinical Outcomes Assessment funding program, CDER is working with grantees to develop a publicly available core set of clinical outcome assessments that can be used for endpoints in clinical tria.

Some of the assessments developed through this program include physical function, pain in infants and young children, and communication in children with neurodevelopmental disabiliti.

CDER is also working with researchers at the University of Michigan to develop innovative trial designs for small groups of rare diseas.

CDER is optimistic about the future of drug development for rare diseases and looks forward to continuing this important work under the new CDER ARC program, working with patients, caregivers, advocacy groups, academia, industry and other partners to address Serious unmet medical needs of patients with rare diseases and their famili.

Reference:FDA official website