Increased focus on Orphan Drugs

Orphan drug legislation has been in place since 1983 in the USA and since 2000 in Europe. The objective of introducing the legislation was to stimulate and motivate the pharmaceutical industry to develop treatment for patient with rare diseases. Many companies from small biotech companies to big pharma are today showing a growing interest in developing drugs for rare diseases. Before the legislation was put into place there were only a handful drugs approved for patients with rare diseases. Today almost 450 indications have been approved by FDA in US and about 80 by EMA in Europe.

Challenges related to developing Orphan Drugs

There are several challenges involved in developing orphan drugs. The clinical trial design are often one of the most prominent depending e.g. on;

few available patients.
lack of knowledge of the natural history of the disease and optimal study endpoints.

Many orphan drugs are approved conditionally or under exceptional circumstances with post-marketing obligations for the manufacturer to further establish the safety and long-term benefit of the new treatment and therefore; Real World Data is a fundamental for developing Orphan Drugs.

Typically safety and effectiveness of orphan drug in the post-marketing phase is demonstrated through registries. By collecting data into patient registries not only the post-approval commitments can be fulfilled but also increasing knowledge about the rare disease and its optimal treatment.
With so few available patients as for rare diseases it is important to avoid duplication in data collection and a public private partnership is strongly encouraged within the rare disease community.

The Late Phase Arena

Compared to earlier phases (I-III) there are specific challenges in the Late Phase in relation to collecting complete non biased data:

Patients are to be followed during busy daily clinical practices over several years.
Clinics involved are often research naive in terms of experience from clinical trials procedures.