Process and Regulatory Changes Making Rare Cancer Drug Development More Efficient

The study of rare cancers poses special challenges for drug developers, who often must draw on their experience in both oncology and rare disease. Careful consideration of clinical trial design and regulatory pathways can help increase the likelihood of success in rare oncology clinical trials.

Researchers studying rare cancers must call on expertise in both oncology and rare disease. Current strategies and processes for general oncology drug development do not always apply to rare oncology, a field that today accounts for over 20 percent of new cancer diagnoses.

In 2014, more than 40 percent of U.S. Food and Drug Administration (FDA) orphan drug designations were for rare cancers. With increasing international cooperation among big pharmaceutical companies, biotechnology companies, and academia, rare oncology research is becoming more efficient, and changes in the regulatory landscape are enabling sponsors to more quickly bring new therapeutic options to patients with rare cancers.

In this white paper, we examine how new processes and regulatory pathways are helping to accelerate development of novel therapies.

About rare oncology

Rare oncology is defined as a disease with an incidence of less than six cases per 100,000 people. Despite the relatively low incidence of each individual type, rare cancers as a whole account for 27 percent of all new cancer diagnoses in the U.S., and 22 percent of all new cancer diagnoses in the EU.3 Over half a million rare cancers are diagnosed in the EU each year, and the incidence of all cases of rare oncology combined is 108 cases per 100,000 people. Currently, there are approximately 4.3 million people living with a rare cancer in the EU.

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