Rare diseases: do not pull on orphan drugs

Rare diseases: do not pull on orphan drugs

Montpellier : France | Nov 04, 2011 at 3:43 AM PDT
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Global economic crisis and climate of mistrust vis-à-vis the drug in France are all threats for "orphan", developed for the treatment of rare diseases, the experts were alarmed at the conference "Rare 2011"

Since 2000, orphan drugs are eligible for incentive European legislation: reducing the cost of placing on the market, market exclusivity for 10 years, able to support measures at national level.

This status should it be challenged? Orphan drugs are they necessarily expensive? The notion of public health does apply to orphan drugs?

These questions have triggered virulent reactions at the conference "Rare 2011" held in Montpellier, when the economics professor of Health (Paris-Dauphine) Claude Le Pen took on the role of "prosecutor", pushing the players in their entrenchments.

"Patients who suffer from rare diseases are entitled to treatment as effective and safe as other," insisted Christel Nourissier, Eurordis (European Organisation for Rare Diseases).

For her, no alternative, solidarity shall pay any price, especially since the social cost of the disease may be disproportionate to the cost of treatment.

But this is not a reason for resignation "that the price is inevitable," she said, pleading for a particular involvement of patient associations in the development of clinical trials.

3.5% of sales of reimbursable drugs

In turn, several industry representatives, very angry against the amendments the Senate tightening the regulatory process for the medicinal, stressed the specificity of orphan drugs, the key point is the small number of patients they serve.

Disease is described as "rare" when it affects less than one in 2000, according to the level allowed in Europe. In France, the disease is considered rare if less than 30,000 people are infected. The 5000-8000 rare diseases listed today, "200 are medium rare, others are extremely rare," said Christel Nourissier.

"It's extraordinarily difficult" to conduct a clinical trial for a disease which we know nothing, argued Annick Schwebig, CEO of Actelion Pharmaceuticals France, especially as its clinical manifestations can be extremely different for different patients.

"When developing a new drug for diabetes, it has 30 of experience," she said, while for a rare disease, the development from scratch.

"When we add research studies and they are divided by a small number of patients, it is increasing prices," For its part, launched Anthony Ferry, President laboratories CTRS.

The cost is also increased in terms of production: production and distribution of small lots is more expensive, have highlighted the industry representatives.

Sales in France orphan drugs accounted for 930 million euros in turnover in 2009, according to the Center for Strategic and prospective study - two-thirds done at the hospital - or 3.5% sales of reimbursable drugs.

Development forecasts for future years have reported 8 to 12 new orphan drugs approved each year in Europe: Christel Nourissier, it is far from the "fantasy" explosion feared.


lalag is based in Karāchi, Sind, Pakistan, and is a Reporter on Allvoices.
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