Are there unlimited supplies of a medicine that can be used for compassionate use?

A company developing a new treatment first produces necessary quantities of the product for its clinical trials (a few hundred to a few thousands of patients, pilot production). When the product is authorised, the company needs to manufacture enough product to satisfy the demand in a larger population (commercial batches). Between these two stages, the size of a Compassionate Use Programme may vary.

The manufacturing capacities for a medicinal product vary with time. When the product is first tested in the laboratory or in animals, only small quantities are needed (usually a few grammes to a few kilogrammes).

When the product is then tested in humans, the first clinical trials are limited in size (typically, a few dozens to a few hundred patients). Thus, quantities of product are still limited. When early results are obtained, the company analyses them and may decide the chances that the medicinal product is safe and effective are high; therefore it elects to  launch larger confirmatory trials and prepare to submit a marketing authorisation application. This is called the Go/No go decision. At this point, the company needs to produce larger quantities of the medicine, as confirmatory clinical trials may enrol thousands of patients.

As a consequence, there is a phase where larger quantities of product becomes available. Before this, the company may be very limited if the demand for compassionate use is high. After this, production can increase and when authorities grant authorisation for a compassionate use, the company should be able to satisfy the demand. But there is a period beginning with the Go/No go decision where it is not certain all demands can be satisfied, as it takes time to build a manufacturing site and to validate the production quality.

 

When the demand is too high

 

In January 1996, people living with HIV knew a new class of anti-HIV medicines was more effective than existing treatments, and by combining different medicines together, survival could then increase, for the first time since the beginning of the epidemic. Patients’ representatives worldwide immediately asked for a Compassionate Use Programme , and tens of thousands of patients expected to receive the so-called Highly Active Anti-Retroviral Treatment in a few weeks.

But the manufacturers could not satisfy the demand immediately: in France, for example, there was only enough ritonavir to treat 100 of 18,000 potential patients. It became clear that during the three to six months it would take for the product to arrive in sufficient quantities, many patients would die.

The National AIDS Council’s advice was to select patients via a random process. "Since patients will be selected randomly by computer, there will be no conscious or unconscious emotional preference or pressure. Drawing lots will relieve doctors of the responsibility of choice and preserve patients' trust in their attending physicians. Lots will be drawn each time supplementary drug doses are made available, with the aim of including all eligible patients.”

 

Page created: 30/10/2013
Page last updated: 19/01/2017
 
 
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