Pediatric cancer is the number one killer of American children today and about 35 children are diagnosed every day.  For certain types of pediatric cancers, the survival rates have improved greatly. However, 2,300 children still die every year, and thousands (three out of five children) who are fortunate enough to survive a difficult battle with cancer will suffer from life-altering and/or life-threatening consequences from harsh treatments intended for adult patients.  Since 1980, the FDA has approved only one drug for treatment of childhood cancer, compared to 50 for adults.

Congressmen Michael McCaul (R-TX), G.K. Butterfield (D-NC), Sue Myrick (R-NC) and Chris Van Hollen (D-MD) introduced to legislation the “Creating Hope Act” to encourage drug makers to have a primary focus on rare children’s diseases. This Act would expedite the FDA approval process for pharmaceutical companies that develop drugs for the treatment of rare pediatric diseases. Congress is hoping that this financial incentive of early market approval would encourage companies to target the “lesser needed treatments.”  President Obama
signed this legislation into law on July 9, 2012.

Why Pediatric Clinical Trials Are So Challenging

Conducting clinical trials in children presents two challenges:

1) Safety and efficacy in children vs. an adult – in any drug development, not only does one have to be concerned with safety and efficacy but more importantly, the long- term affect of the drug on the future development of a child.  This is the reason why drugs are contraindicated in pregnant women.  A child is still developing their full immune system and other systems that make a child biologically different from an adult.  For example, when a child greater than 6 months has a temperature of 101-104 degrees, the pediatrician will be concerned if it is accompanied by symptoms and/or the duration of the fever.  For adults having a temperature of 103 degrees or higher is considered dangerous.

2) Recruitment - it is difficult to recruit children into trials particularly in rare disease because the disease may be difficult to diagnose by your average MD who has not seen a case before, and who then refers the child to a specialist.  Depending on the disease, the severity of the disease and the mortality rate, by the time a child is identified to enroll into a trial, the child may be nearing his or her life expectancy.  In some cases the parents may not want their child to be in the control arm knowing that there is a medication that might help their child.

Companies Working on Pediatric Indications

Alexion Pharmaceuticals Inc. has taken on these challenges when they acquired patents and assets from Orphatec Pharmaceuticals GmbHm in Germany to treat a very rare genetic disorder.  The disorder is molybdenum cofactor deficiency (MoCD) Type A, which results in severe brain damage and rapid death in newborns because of buildup of neurotoxic sulfite.

AstraZeneca and Novartis received EU approval for a Nasal Flu Vaccine for Children and FDA approval for Meningitis Vaccine in Children respectively.

At the 2012 American College of Allergy, Asthma & Immunology (ACAAI) meeting in Anaheim, California, Sunovion Pharmaceuticals Inc. announced results of a safety study that evaluated the effect of ZETONNA^® (ciclesonide) Nasal Aerosol compared with placebo on the hypothalamic pituitary adrenal (HPA) axis in pediatric patients 6 to 11 years old with perennial allergic rhinitis (PAR), as well as results from a Phase III pivotal seasonal allergic rhinitis (SAR) trial that assessed the 24-hour efficacy of ZETONNA in patients 12 years of age and older.

Closing Thoughts

Developing a drug with a pediatric indication has many challenges which makes it cost prohibited both in time and money and the main reason the pediatric population has been neglected.  An expedited approval process and financial incentives is the best way to encouraged a greater proportion of companies to focus on rare diseases and rare pediatric diseases.  A number of companies have already taken on the challenge even before President Obama signed the “Creating Hope Act.” 

What is the reaction from industry leaders about targeting rare diseases? “For me, every sign here that more people are getting involved in rare diseases and gene therapy is a great thing,” says Nick Leschly, president and CEO of Bluebird Bio, a biotech company developing gene therapies for rare diseases such as beta thalassemia and childhood cerebral andrenoleukodystrophy reported by Xconomy.

Sylvie Gregoire, president of Shire Human Genetic Therapies said “I think that the whole sector of rare diseases has gained more attention, not just from companies but from the FDA and the NIH.  In a way, that’s good for the science in terms of investment,” reported by Xconomy

Written by Regina Au