Compassionate Use Guideline Finalized - Applied Clinical Trials

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Compassionate Use Guideline Finalized Industry and patient groups embrace new rules but acknowledge concern over initiative's impact across EU.

Source: Applied Clinical Trials



Peter O'Donnell
The new European procedure for compassionate use of medicines came into effect during the summer. The European Medicines Agency issued a finalized guideline in July on its role in the procedure, after more than five years of discussion. The move has been welcomed by patient groups and by the pharmaceutical industry—although there is a widespread feeling that the new rules do not go far enough.

The European Cancer Patient Coalition had complained that European Union delay was hurting patients, with compassionate use "an unacceptable lottery" dependent on the measures taken by individual member states. "Many of our cancer patients' lives will depend on whether they can enroll in a clinical trial or are eligible for a compassionate use program," said the Coalition. It acknowledges the merit of the new procedure, but continues to indicate concern over whether it will have sufficient impact.

Similarly, the European Federation of Pharmaceutical Industries and Associations compliments the European authorities on their initiative, but observes: "There is also a need in the longer term for greater harmonization of compassionate use approach and process across member states. The current initiative can hopefully be viewed as a first step towards that long-term objective."

A new possibility

It was the major revision of European pharmaceutical legislation in 20011 that opened up the possibility of compassionate use, as an exception to the general rule that medicines must be authorized before use or limited to use under an approved clinical trial protocol. A compassionate use program with a new treatment still under development became a formal treatment option for patients in the European Union suffering from a disease for which no satisfactory, authorized alternative therapy exists or who cannot enter a clinical trial.

The new guidance makes clear that this facility is in no way intended to replace clinical trials. "From a methodological point of view, clinical trials are practically the only means of obtaining reliable and interpretable efficacy and safety data for a medicinal product," says the Agency firmly. Compassionate use programs may give rise to valuable safety data, but they "cannot replace clinical trials for investigational purposes. Compassionate use is not a substitute for properly conducted trials. Compassionate use should therefore not slow down the implementation or continuation of clinical trials intended to provide essential information relative to the benefit/risk balance of a medicinal product." In addition, "patients should always be considered for inclusion in clinical trials before being offered compassionate use programs."

Although the option is permitted through EU legislation, compassionate use programs are governed by individual member state rules, whether for an individually named patient or for cohorts of patients. The EU role in all of this is to ensure that for medicines covered by the EU's centralized evaluation procedure, a common approach is followed in respect of the criteria and conditions. The European Medicines Agency can issue a non-binding opinion if a member state requests this before making its own decision.

Tight definitions

The rules lay down that the product can be provided only to "patients with a chronically or seriously debilitating disease, or a life threatening disease, and who cannot be treated satisfactorily by an authorized medicinal product" in the European Union. They spell out clearly how these terms should be interpreted. The severity of the disease needs to be justified based on objective and quantifiable medical or epidemiologic data. And while a life-threatening condition "is relatively easily recognizable," defining what conditions are chronic and seriously debilitating must take account of "morbidity that has substantial impact on patients' day-to-day functioning and will progress if left untreated"—such as cancer, HIV/AIDS, neurodegenerative disorders, and auto-immune diseases. "Chronic or serious debilitation or fatal outcome should be a prevalent feature of the target disease," the Agency says.

Even "patients who cannot be treated satisfactorily" is redefined, to designate "patients left without treatment options or patients whose disease does not respond or relapses to available treatments, or for whom the treatments are contraindicated or inadequate." Whether patients can be treated satisfactorily or not will be assessed by the Agency, based on the review of authorized products and on the arguments as to why these are not considered satisfactory.


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