A cancer diagnosis is a shock, but adults with the disease can take some comfort in the numerous treatments available to them — both through clinical trials and as drugs that are already on the market. Children cannot. Because they make up only 1% of US patients with cancer, children are a low priority for pharmaceutical companies that want to launch an effective drug quickly. The hassle of a paediatric clinical trial may not seem worth it until after the drug has proved to be safe and effective in adults. This process can take decades, leaving children with therapies that are sometimes almost obsolete.

To access therapies early, parents of these children can turn to compassionate-use programmes, in which companies give experimental drugs to people who are in desperate need. In the United States, firms that agree to provide medicines in this way will ask the Food and Drug Authority for emergency permission, which is almost always granted.

This system, although helpful for some, is rife with complications. Patients and their families report difficulties in applying for such programmes, and say that they rarely receive responses. Companies that withhold a drug — because it is in short supply or not right for a patient — can find themselves on the receiving end of critical social-media campaigns highlighting individual patients. And firms worry that if a person dies or is harmed while taking a drug, it could hurt the drug’s chances of being approved. No one knows how many requests parents make and how often companies approve them, but anecdotally, firms often deny drugs on the grounds that they have not been tested in children.

Proper clinical trials for childhood cancer drugs are scarce. 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.

“Legal loopholes often prevent children with cancer from accessing new drugs.”

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.

Also exempted are drugs for ‘orphan’ diseases that affect fewer than 200,000 people in the United States. The number of orphan designations has skyrocketed in recent years — the improved ability to define the molecular basis of an individual’s cancer means that diagnoses have become increasingly subdivided, and the majority of approved cancer drugs now carry this orphan designation.

Legislation is now attempting to close those loopholes. The Research to Accelerate Cures and Equity (RACE) for Children Act, introduced to the US Congress on 14 July, would require companies to apply the PREA to any therapy with a molecular target that is relevant to both an adult and a childhood disease. It would also end the exemption for orphan diseases. Last July, the European Medicines Agency passed similar rules to make it more difficult for companies to avoid testing drugs in children. This applies when the disease has a common mechanism in adults and children, unless the drug is likely to be unsafe in children.

With Congress now out of session and focused on the upcoming US election, the RACE for Children Act is unlikely to advance before next year. But when lawmakers pick it up, they should also address problems with compassionate-use programmes — and ensure a transparent and useful process for people to gain access to unapproved drugs. They should also encourage companies to make more drugs available through market incentives, and provide increased protection should something go wrong.