The Orphan Drug Act (ODA) has driven an increase in research and drug approvals for rare diseases over the last five years. In fact, 17 of the 39 drug approvals granted by FDA in 2013 were for orphan drugs exclusively. Despite this focused commitment to bringing new treatments to market that support rare diseases, clinical trials on orphan drugs remain uniquely challenging, in an already-challenging environment.
There are a host of logistical challenges that come with orphan drug research – which add significant complexity to a study protocol with a small patient population and a difficult-to-define disease progression.
These challenges include:
- A disease that is not well-understood
- A small, geographically dispersed patient population
- Patients have waited years for diagnosis or access to a clinical trial
- Patients may have expended personal resources to take control of their disease
- Patients may be distrustful of the medical system
- Little opportunity to make and learn from mistakes
- Limited number of seasoned investigators with expertise in the disease
This is why orphan disease studies cost an average of 25 times more per patient than non-orphan trials.
Greenphire has become the partner of choice for many sponsors and CROs developing orphan drugs, with a recent addition of Anthera Pharmaceuticals to its client roster.
Our ability to recognize the value and importance of both patient AND site centricity has established key best practices to removing the logistical challenges of orphan drug research. These include:
- Provide a single, convenient resource for patients and study coordinators to access help with logistical arrangements
- Place no financial burden on the patients whatsoever
- Ensure no cash outlay by clinical sites and investigators
- Enable remote data collection when possible
- Provide instant, accurate reimbursement for other misc. costs that may arise during the study
- Limit administrative tasks associated with financial management and reimbursements
- Establish and execute clear, defined logistical policies and maintain adherence to these throughout the course of the trial, while simultaneously allowing sponsors and sites to respond flexibly to the challenging needs of patients and caregivers
- Allow sponsors to maintain blinded patient identities
- Support automatic communication with patients via text, email alerts
- Manage costs without impacting the patient experience
- Automate and streamline payments to investigators to ensure quick cycle times and accuracy
