BioWorld International Correspondent

BRUSSELS, Belgium - "Prices for orphan drugs need to allow companies to recoup their investment and have an incentive to develop products in this area. Without appropriate prices, no orphan medicines would be available, as there would not be any return on investment for the developing companies."

EuropaBio made that comment last week as it welcomed a new study that the European Union is to conduct into how EU member states handle pricing and reimbursement for orphan drugs.

Medicines developed and approved under the EU's three-year-old scheme to support orphan products - many of them based on biotechnology - are still not widely available to patients in need of them, EuropaBio pointed out. The problem is particularly acute for biotech orphan medicines specifically developed for a small group of patients: "Research, development and manufacturing costs for biotech medicines remain high," it claimed.

It said many EU member states' regulatory authorities "are not always informed about the rare disease for which an orphan medicine is developed, or about the needs of the patients suffering from these serious diseases, and may be skeptical about their price." But, EuropaBio went on, in order for the scheme to be successful, "European patients and companies need tax cuts, not price cuts." Many of the biotech medicines are not replaceable by other products, as they are often the first treatments ever available to patients suffering from certain genetic diseases, it argued.

The price of an orphan medicine also needs to be compared to the treatment and social costs without the medicine, it said.

"After the commitment to provide incentives to companies for developing treatments for rare diseases, society should also be committed to bring the resulting medicines to those patients in need," it said.

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