The performance of research studies to evaluate drugs in children are critical for determining the safety and efficacy of medications in children. …Without proper drug studies in children, children may not benefit from and may even be harmed by drugs that are available to adults. Also, certain disorders affect children primarily, necessitating drug testing on appropriately aged subjects. It is morally imperative, therefore, to formally study drugs in children so that they can enjoy appropriate access to existing and new therapeutic agents” 1

CRDO form a group of network resources for scientists worldwide working on children’s rare diseases. We can provide free research funding, early research guide, advice of making promising research findings to useful and beneficial drugs to treat the patients.  The experts in the network will provide scientific and research guidance, regulatory and clinical strategy, business advice, and ultimate help to bring promising drugs to the needed children.

Reference:

  1. Robert E. Shaddy, MD, Scott C. Denne, MD and The Committee on Drugs and Committee on Pediatric Research. PEDIATRICS Vol. 125 No. 4 April 2010, pp. 850- 860

Children’s rare diseases and disease search

In the U.S., a condition is considered a rare disease if it affects fewer than 200,000 people nationwide at any given time.  The Office of Rare Diseases Research at the National Institutes of Health reports that there are approximately 7,000 rare diseases known today, and sometimes a diagnosis of a rare disease can take months or even years. Among them are approximately ten million children who are affected by a rare disease. Many rare children’s disease does not have a known treatment at this time for several reasons. Most of the clinical research studies do not address the specific pediatric populations, and children’s rare disease patients are few and located in various geographic areas. Additionally, many rare diseases do not have a clear underlying cause.  This makes research in this year more critical.

The definition of children’s rare disease is that (A) the disease is a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years, including age groups often called neonates, infants, children, and adolescents {and} (B) The disease is a rare disease or condition per US law definition.

Some examples of children’s rare disease are:

•Batten Disease

•Biliary Atresia

•CANDLE Syndrome

•Childhood Interstitial Lung Disease

•Duchenne muscular dystrophy

•Krabbe Disease

•Neuroblastoma

Please follow the following link to search for all the children’s rare diseases.

Browse A-Z | Genetic and Rare Diseases Information Center (GARD) – an NCATS Program (nih.gov)

FDA rare pediatric disease designation and awards

The FDA regulates all the drug clinical development and approval in the U.S, and provide support and incentive to promote the interest in developing rare pediatric diseases. All the sponsors can apply for rare pediatric disease designation and entitled its associated benefits including, may not limited to, clinical development tax credit, potential waive of the marketing application fee, and 7 years of marketing exclusivity.

Most of all, the sponsor of a human drug for a rare pediatric disease may be eligible for a valuable voucher that can be used to obtain a priority review for a subsequent human drug.

 For updated information for incentive for rate pediatric diseases, please check FDA website at

https://www.fda.gov/industry/developing-products-rare-diseases-conditions

 Request for Rare Pediatric Disease designation should be sent to:

The Office of Orphan Products Development (OOPD)

Food and Drug Administration

WO32-5295

10903 New Hampshire Avenue

Silver Spring, MD  20993-0002

Or using email orphan@fda.hhs.gov during the Covid-19 pandemic time.

Priority review voucher

Under Section 529 to the Federal Food, Drug, and Cosmetic Act (FD&C Act), FDA will award priority review vouchers to sponsors of rare pediatric disease product applications that meet certain criteria. Under this program, a sponsor who receives an approval for a drug or biologic for a “rare pediatric disease” may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product. On December 27, 2020, the Rare Pediatric Disease Priority Review Voucher Program was extended. This voucher is transferable and allows the owner to use the voucher to redeem the priority review of any drug marketing application. For details, please check FDA guidance:

https://www.fda.gov/industry/developing-products-rare-diseases-conditions/rare-pediatric-disease-rpd-designation-and-voucher-programs