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Compassionate Use: A necessity not a nicety

rare disease compassionate use family
rare disease compassionate use family

Clearer terms of Compassionate Use are needed to ensure patients with rare diseases can access vital medication safely, fairly and as soon as possible.

Drugs must become more readily available for patients

It’s hard enough knowing you have a disease with no cure, but perhaps it’s even harder to know that there is a drug that could help you, but you can’t access it. François Houÿez is Director of Treatment Information and Access, Policy Advisor at Eurordis, an alliance representing 738 rare disease patient organisations in 65 countries. He is amongst professionals who are advocating for better legislation and a more unified approach with regard to Compassionate Use of treatments for rare diseases.

Enabling patients to access treatment in development

Put very simply, Compassionate Use enables groups of patients, who have a disease with no satisfactory authorised therapies and who cannot enter clinical trials, to access products in development under strict conditions. “It recognises that there is a human side to the development of drugs,” explains Mr Houÿez. “It’s horrible to die knowing that there is a treatment out there that may help you.”

We estimate that 5,000 deaths could have been avoided in Italy alone, had Compassionate Use been granted.

In 1996, a group of patients in France were granted permission under Compassionate Use to access medication to treat HIV in the March even though the product didn’t come to market until the September. “During that time more than 11,000 patients received treatment based on ‘Compassionate Use’,” explains Mr Houÿez. “We saw hospital admissions amongst that group fall by 50% compared to same period in 1995. However, in other countries patients who had to wait until September for the European Marketing Authorisation.”

Safe and fair practice

Whilst Compassionate Use undoubtedly saves lives, legislation varies from country to country, company to company and drug to drug. Mr Houÿez believes that in order to address these issues, pharmaceutical companies need to build terms of Compassionate Use into their drug development plans from the outset.

“We had a situation recently where a patient in Romania with multi-drug resistant tuberculosis wanted to access drugs that were in development elsewhere in Europe,” explains Mr Houÿez. “The patient was too ill to travel to France where a compassionate use programme had started it was difficult to transport the drugs to Romania. There was a rapid exchange of information, but sadly decisions were made too late for him. A programme could start in Romania shortly after. ”

Mr Houÿez believes that more detailed guidelines could be put in place to ensure companies make provision for Compassionate Use.

The European Medicines Agency (EMA) has recently launched a voluntary scheme called PRIME which offers proactive support to medicine developers to enable accelerated assessment of medicine applications. However, the guidelines are not legally binding, so companies are under no obligation to act.

Across the world Compassionate Use of drugs is already saving lives, and whilst there will always be patients for whom the drugs come too late, there are those who can benefit right now from changes in policy and a little more forward planning.

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