PPD rolled out a new hub to oversee all rare disease and pediatric drug development activities, as the world observes Rare Disease Day on the last day of February.
The Rare Disease and Pediatric Center of Excellence is a leadership team of six experts focused on conducting clinical studies in rare diseases and children. It will be responsible for driving the design and execution of clinical trials of PPD in those fields.
In the U.S., as defined by the Rare Diseases Act, rare disease refers to a condition that affects fewer than 200,000 people in the country. As for the EU, the European Commission on Public Health defines the term as a disease affecting fewer than 1 in 2,000 people. A disease can’t just be rare in number, but also must be life-threatening or chronically debilitating to qualify for the designation.
The rarity of these diseases usually requires unique expertise from researchers and poses additional challenges for clinical studies such as patient recruitment.
But on the other hand, the health communities have thus far identified more than 6,000 rare diseases, and they combined are affecting about 60 million individuals in the U.S. ad Europe, about half of whom are children, according to PPD.
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“With a great majority of rare diseases touching children, we believe our approach will enable us to be a more connected and active partner,” said Karen Kaucic, M.D., PPD’s SVP and head of the new center, in a statement. She went on and said that the CRO’s “ability to navigate complex trial logistics” and its “keen understanding of customized patient access strategies in low-prevalence, widely dispersed patient populations” could benefit its clients.
The CRO has a strong portfolio in drug development for rare diseases and pediatrics. It has conducted more than 220 rare disease-related studies with more than 50,000 patients over the past five years, according to the company. It has also conducted more than 400 pediatric clinical trials in that time frame for a wide range of therapeutic indications, including infectious diseases, respiratory, cardiology, oncology and immunology, involving more than 97,000 patients.
To enhance its pediatric clinical trial capabilities, and to provide a platform to share resources and expertise in the field, the CRO established a pediatric investigator network (PIN) back in July 2015. The network initially tapped eight renowned pediatric research organizations and later expanded to 12, encompassing more than 25 sites in six countries.
In the U.S., the Orphan Drug Designation program by the FDA offers orphan status to drugs meant for rare diseases and to those with a small market that won’t return the costs of developing and marketing of the treatment.
Besides, to encourage development of rare disease drugs, the FDA also has a Rare Pediatric Disease Priority Review Voucher program, which grants a drugmaker that receives an approval for a rare pediatric disease a voucher that can be redeemed for priority review of a different product. The most recent example of that program saw Sarepta sell the PRV voucher it obtained with approval of Exondys 51 to Gilead for $125 million.