Who Are Orphan Europe

Orphan Europe were one of our sponsors at the Cystinosis UK conference held in Manchester in September this year for whom we were very grateful . This article explains who they are and what their involvement is with the Cystinosis community.

Serving the Unmet Clinical Needs of patients with Rare Disorders

Many of our members will be familiar with the name of Orphan Europe, who provide CYSTAGON© (cysteamine bitartrate) capsules in varying doses, licensed for the treatment of nephropathic cystinosis. But what is the history & mission of this company?

Orphan Europe was founded in 1990 with the aim to provide treatment for patients with unmet medical needs suffering from rare diseases. The UK subsidiary was established in 1996.

Today Orphan Europe provides nine orphan products to patients all over the world with the help of 130 medical, scientific and marketing specialists in over 15 countries, including the United States and the Middle East. Orphan Europe has developed excellent local & international relationships with clinicians, researchers, patient groups, health authorities and partnering companies. The company enjoys an unmatched understanding of the European regulatory process required to gain approval for the marketing of orphan drugs and its entire infrastructure, is adapted to serve the particular requirements of niche patient groups.

Orphan Europe is evidently committed to the improvement of knowledge about rare diseases among health care professionals. This is exemplified by the Orphan Europe Academy that provides unique, independent, high-level education on rare diseases.

Expertise in Orphan Drug Research & Development

Standard clinical trials are not feasible for orphan drug development. Patients exhibit a wide range of heterogeneous symptoms, often needing individually tailored treatment regimens. The use of placebo controlled trials is not ethically possible and standardized treatments are not available for comparison. Since the very few patients are scattered all over the world, it is virtually impossible to bring them together in one trial.

Working closely with physicians, Orphan Europe designs tailor-made clinical programmes that define the key evaluation criteria for each drug, involving a unique external network of experts who support and cooperate closely with the company.

Regulatory Affairs

In 2009, Orphan Europe received its 7th market authorisation in Europe.

Building a marketing authorization file with very low patient numbers requires specialist knowledge and a solid working relationship with regulatory authorities. Orphan drugs undergo the same rigorous review processes as other pharmaceutical products. They therefore need to meet the same high standards of quality, safety and efficacy.

Orphan Europe now has 21 years of worldwide regulatory experience with regard to orphan drug designation & provision, all Orphan Europe products being supported by a comprehensive pharmacovigilance & medical information facility. Orphan Europe personnel are always conscious that patients are waiting for treatment.

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