There is no reason for guess work. The pharmaceutical companies are not carrying out pediatric equivalents to their adult treatment drugs.
27 July 2016
...Proper clinical trials for childhood cancer drugs are scarce. (click here) Designing a clinical trial is never simple, but adding children to the picture complicates the process immensely. Children are not just ‘small adults’ — they metabolize drugs in very different ways. It is difficult to predict from adult or animal studies whether a chemotherapy drug will be more or less toxic in a child, and at what dose. The process of obtaining informed consent for children participating in a trial can also be more complicated. And companies fear that the death of a child — even if unrelated to the treatment — could bring bad publicity for a new drug.
Recent years have seen attempts to make more drugs available to treat children. In the United States, a 2003 law known as the Pediatric Research Equity Act (PREA) requires that companies develop a plan for how they will test experimental drugs in children, although many trials are exempted. A second law, called the Best Pharmaceuticals for Children Act, motivates companies to perform paediatric clinical trials by granting an extra six months of market exclusivity for the adult drug.
Overall, these laws have been successful, leading to hundreds of drug labels being updated with information for use in children. But legal loopholes often prevent children with cancer from accessing new drugs. For instance, therapies for conditions that do not affect children — such as Alzheimer’s disease — are exempt from the PREA. And exemptions intended for such diseases have been broadly applied to cancer. For example, therapies that are being trialled in adults with breast cancer are exempted because children do not get that cancer, even if the drug could treat a childhood cancer in a different organ....
27 July 2016
...Proper clinical trials for childhood cancer drugs are scarce. (click here) Designing a clinical trial is never simple, but adding children to the picture complicates the process immensely. Children are not just ‘small adults’ — they metabolize drugs in very different ways. It is difficult to predict from adult or animal studies whether a chemotherapy drug will be more or less toxic in a child, and at what dose. The process of obtaining informed consent for children participating in a trial can also be more complicated. And companies fear that the death of a child — even if unrelated to the treatment — could bring bad publicity for a new drug.
Recent years have seen attempts to make more drugs available to treat children. In the United States, a 2003 law known as the Pediatric Research Equity Act (PREA) requires that companies develop a plan for how they will test experimental drugs in children, although many trials are exempted. A second law, called the Best Pharmaceuticals for Children Act, motivates companies to perform paediatric clinical trials by granting an extra six months of market exclusivity for the adult drug.
Overall, these laws have been successful, leading to hundreds of drug labels being updated with information for use in children. But legal loopholes often prevent children with cancer from accessing new drugs. For instance, therapies for conditions that do not affect children — such as Alzheimer’s disease — are exempt from the PREA. And exemptions intended for such diseases have been broadly applied to cancer. For example, therapies that are being trialled in adults with breast cancer are exempted because children do not get that cancer, even if the drug could treat a childhood cancer in a different organ....
