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Think Global, Act Local: Supporting Orphan Drug Development in NJ

You could say that I’m a Jersey Girl. Having lived all my life in the Garden State, it’s important to me to help give back to the New Jersey community that is home to our Princeton headquarters. So, I’m really proud that Certara has supported the approval of several orphan drugs for rare diseases, one of which was developed by a NJ-based company. In recognition of this work, we were recently designated as a finalist in the “Innovation Heroes” category of the NJBIZ 2015 Healthcare Heroes awards program. In this blog post, I’ll discuss how we’re using biosimulation technology to bring safer, more effective medications to the people of New Jersey and beyond.

The challenges of orphan drug development

There are about 6,800 rare diseases, affecting nearly 30 million Americans, according to the National Organization for Rare Disorders. A disease is considered rare if it affects fewer than 200,000 Americans. The National Institutes of Health reports that about 80 percent of rare diseases are genetic in origin and about half of them affect children. These diseases can be debilitating, disabling, and life-threatening. And for 95 percent of rare diseases, there is currently no FDA-approved treatment.

Requirements to get a new drug approved for a rare disease are the same as for a more common condition. However, the smaller patient populations present unique challenges for conducting clinical trials. Not only are there very few patients available to participate in a new study, but they may be geographically dispersed, and have difficulty getting to the clinical trial sites. In addition, there is often very little data available about the disease, how it progresses, the most appropriate drug dose, how long the therapy should be given, and what the likely outcomes might be.

Using biosimulation technology to support regulatory approvals for orphan drugs

Clearly, a different R&D approach was needed to help get safer, effective new therapies into the market to treat these often life-threatening diseases. Biosimulation technology has been used to support most regulatory approvals for rare disease drugs in the past few years. It can help to determine the appropriate dose for a drug entering clinical trials, identify possible interactions between drugs, recommend the pediatric dose for a drug when there is only adult data available, and, in some cases, remove the need for conducting an additional clinical trial.

Our scientists use empirical pharmacokinetic and pharmacodynamic data and computer models to determine the relationship between a drug dose, how it is absorbed and excreted, and how the body responds. They combine data from preclinical studies and first-in-human clinical trials with trial design information from the medical literature. They also use computer models to test how patients’ age, weight and disease state can alter drug exposure and response, and to run “what if” simulations for patient populations that are taking several medications for different conditions.

Modeling can also help to determine whether a treatment effect is just modifying symptoms or actually changing the underlying disease process, an important distinction with cancer. These data help clients to make more informed go/no-go decisions about new drug candidates. Our consultants often accompany clients to FDA meetings to review trial designs and discuss models.

Supporting orphan drug development in New Jersey

Certara played an important role in the recent drug approval for Cerdelga® (Eliglustat tartrate), Genzyme’s drug for treating Gaucher’s disease type 1. For Cerdelga, we performed PBPK modeling that was accepted by the FDA for the drug label in lieu of drug-drug interaction studies and for determining dosing. We also partnered with NJ-based NPS Pharmaceuticals, providing strategic pharmacometrics services, on two of its new drug approvals for rare diseases. Their most recent regulatory success was with Natpara, an adjunct to calcium and vitamin D used to control low blood calcium levels in patients with hypoparathyroidism. Natpara was approved by the FDA in January 2015. Shire acquired NPS Pharma in February 2015.

Our scientific team has also helped develop treatments for short-bowel syndrome, smallpox, anthrax, and atypical hemolytic uremic syndrome. Furthermore, no patient population is too small: our biosimulation technology helped to get a new drug approved for an ultra-rare disease based on data from only six patients!

All information presented derive from public source materials.

Learn more about how Certara’s rare disease solutions can help you!

I hope that you’ll read this case study on how modeling and simulation supported the approval of an orphan drug using evidence from a previous indication.

About the author

By: Dana Cohen

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