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 Successfully designing pediatric clinical trials for ultra-orphan indications

"It is difficult to successfully navigate pediatric trials in ultra-orphan indications if the drug is already available in the marketplace," said Dr. Deborah Lee, Senior Medical Director, Clinical Affairs, Lundbeck.  

"It is important to realize that once a drug is on the market, it is not the end of the potential for that drug. Most drugs that come out have a single indication, but feedback from the physician community can lead to clinical trials evaluating additional indications. There is a relationship between practitioners, academia using the drug and the pharmaceutical industry, which determines the next step in the lifecycle management of the drug," Lee, a speaker at the marcus evans 10th Annual Pediatric Clinical Trials Conference, went on to say.  

When Lundbeck was looking at another indication for a drug that had been available in Europe for 30 years and was only in the US for a few years, its biggest challenge was the availability of the drug. "It is difficult to design a clinical trial when a drug can already be prescribed by physicians. The incentive for being in a clinical trial is much less. It takes a lot more care to develop a doable trial for that, especially when it needs to be placebo controlled." 

On top of that, it is important that pharmaceutical companies have experienced staff in place to navigate challenges specific to pediatric clinical trials. "There can be hesitation about doing pediatric clinical trials, especially if a company's medical directors and decision-makers are not pediatricians or pediatric sub-specialists. They might think, 'You are going to do this to a child? You are going to draw blood from a child? It is mean.' It is helpful for pharmaceutical companies to have pediatric specialists on staff, because they have experience which leads to a different philosophy -- they are used to taking care of children."  

With a background in academic medicine, Lee has seen both sides of the pharmaceutical industry and is concerned about difficulties physicians and pharmaceutical companies face when attempting to work together. "Some institutions have established policies whereby their physicians cannot work with industry. That is a real problem. We need investigators to tell us what kinds of drugs they are looking for. We need their advice to design relevant and doable clinical trials. If academia continues to pull back, neither party is going to get any new drugs out in the market for the patients who need them," she concluded.  

The marcus evans 10th Annual Pediatric Clinical Trials Conference will take place in Philadelphia, PA, June 11-12, 2014. For more information, please visit the event website  

For all enquiries contact: Michelle Thomas -

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