Precision medicine offers a lot of promise for targeting specific genetic markers in cancers and helping to ensure drugs used to treat them are effective. The hard part is when running clinical trials for patients with specific markers, finding those patients can be difficult, especially when the markers are rare.
Finding better ways to identify and reach patients and help the patients find the trials in turn will be the topic of a panel discussion at next month’s MedCity CONVERGE conference, taking place July 11-12 in Philadelphia. One of the panelists, Sameek Roychowdhury, talked about some of the efforts to accomplish that. Roychowdhury is an assistant professor of medicine at Ohio State University in Columbus and a medical oncologist at the university’s Arthur G. James Cancer Hospital.
The panel’s title is “Clinical Trials 2.0,” and it’ll discuss how patient networks and new technology can be used to boost trial recruitment and manage trials more efficiently. For Roychowdhury, the issue is that trials are becoming more selective as clinicians gain a better understanding of who is likely to benefit from investigational drugs, but it’s also becoming harder to find those patients and for the patients to find the right trials.
——————————————————————————————————-
Attend MedCity CONVERGE to hear from healthcare innovators like Sameek Roychowdhury. Use promo code MCN50 to save $50. Register now.
——————————————————————————————————-
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Case in point: Loxo Oncology is nearing a Food and Drug Administration approval decision for its drug larotrectinib, for solid tumors that exhibit NTRK fusions, regardless of where in the body they occur. The drug is highly effective, but only a tiny minority of cancer patients overall harbor the fusions. One remedy for the conundrum of finding such rare patients is the Oncology Research Information Exchange Network, or ORIEN, comprising academic cancer centers across North America. With ORIEN – of which Roychowdhury’s center was a founder – patients consent to have their tumors genetically profiled, thereby creating a network of data that can be mined and allowing researchers to identify who has a target while also facilitating the discovery of new targets. So if a study is looking at patients with NTRK fusions, Roychowdhury said, ORIEN can mine RNA data from thousands of patients and find those who harbor the fusions, as well as finding study protocols.
This differs from TAPUR, the American Society of Clinical Oncology trial, which uses existing drugs that are already on the market and enables them to be repurposed for different cancer indications based on patient genetics. The disadvantage to the TAPUR approach is that it may cause patients to miss out on novel therapies, though the advantage is that a patient’s gene and target may be so rare that there are no clinical trials nearby, whereas the study is open at numerous sites rather than just academic cancer centers, Roychowdhury said.
For its part, the pharmaceutical industry is starting to appreciate networks more and invest in them because while it is very costly to have to screen a large number of different patients, it saves on costs and also enables finding patients a lot faster if they can be screened across a network of cancer centers, he added. The networks can also help to bring preclinical drugs into clinical testing by helping with target validation and discovery.
Meanwhile, contract research organizations – companies to which drugmakers will usually outsource clinical trial functions like data monitoring and site monitoring – will not likely change their clinical trial management practices, Roychowdhury said.
Photo: MedCity News
