Regulatory Focus™ > News Articles > Pediatric Challenges Focus of Amended EMA Guideline on Lipid Disorders

Pediatric Challenges Focus of Amended EMA Guideline on Lipid Disorders

Posted 03 October 2012 | By Alexander Gaffney, RAC 

A new pediatric addendum from the European Medicines Agency (EMA) is aimed at highlighting unique challenges facing the development of treatments for pediatric patients with lipid disorders.

Primary, or familial, lipid disorders are characterized by an excess amount of lipids or lipoproteins, which can lead to organ-the heart in particular-and tissue damage in the absence of adequate treatment. In children with familial lipid disorders, this damage starts occurring from birth and requires early and aggressive intervention to moderate the disease.

Dietary changes are often useful in mitigating the effects of the disease, but EMA notes pharmacological interventions may be required if dietary changes fail to work, as they often do in patients with inherited versions of the disorder.

Secondary versions of the disease include some fairly common disorders, including diabetes mellitus type 1 and 2, for which obesity is likely a major contributing factor.

As with most pharmacological interventions, EMA recommends the safety and efficacy of any treatment be established in adults before it is tested in pediatric populations. Children, however, may have different variations of a given lipid disorder, may require the use of different surrogate endpoints and may have specific safety needs that must be taken into account, explained EMA.

One of the biggest challenges acknowledged by EMA's pediatric addendum is extrapolating data on cardiovascular outcomes in early interventions. Lowering LDL-cholesterol in children may be beneficial and reverse vascular damage, but beyond a certain point it may affect physical development. Without interventional studies and long-term marketing data, it will be difficult to adequately assess the benefits and risks to early treatment, they explained. Other issues, such as cognitive development and sexual maturity, were also raised as potential issues to be watched.

As a result of this, EMA recommends any children enrolled in clinical trials be given dietary and lifestyle interventions before being enrolled in a pharmacological intervention.

EMA also recommends follow-up monitoring of all patients up to two years after the conclusion of the study.

Regulatory Focus newsletters

All the biggest regulatory news and happenings.