DSN was invited to attend the “Leveraging Real-World Treatment Experience from Expanded Access Protocols” event at the FDA Headquarters in Silver Spring, Maryland recently. At this event, the team learned about numerous advancements in Expanded Access (EA) that will begin to “revolutionize how we see Expanded Access programs moving forward”.
1. We’ve all heard that perception is reality. How does this affect EA programs?
Scott Gottlieb, 23rd Commissioner at the FDA, noted that the goal was ensuring that the right drug gets to the right patient at the right time. The FDA noted that a need to ‘disrupt’ the system was evident. This conference served as a vehicle to do so. On the contrary, pharmaceutical companies believe that EA programs can be a threat to post marketing approval. The FDA, however; conveyed that EA programs do not impede development progress. These programs often provide valuable information sooner including refining of dosage, which has historically been done in the post marketing setting. An article from Therapeutic Innovation and Regulatory Science stated that out of 10,000 expanded access INDs submitted between 2005 and 2014, only two cases were identified in which an adverse event on an expanded access IND led to a clinical hold being placed on the commercial IND. What does this mean for you? DSN will work with you to ensure that we are processing and tracking this valuable clinical data so that you can make decisions early on regarding formulations, contraindications, and get the right treatment to the right patients sooner.
2. Planning is everything - how can DSN help?
Several patient advocates and the FDA noted that planning for EA programs as early as possible is key! Typically, waiting until there is a life-threatening situation and then trying to navigate the EA process can be chaotic and ineffective. It is a burdensome process for everyone involved, none more so than the patient with a life-threatening condition. Being prepared early is not only more efficient, it can be lifesaving. With a 99% authorization rate for EA programs by the FDA, chances are your company will be faced with addressing this scenario. Most organizations don’t have time for this activity in the midst oftheir ongoing workload. DSN can help. DSN will facilitate mitigating this burden early on, for you and patients. DSN partners with WEP Clinical who manages EA programs and will assist in the implementation and management of EA programs.
3. Decreasing the burden.
Decreasing the burden on pharmaceutical companies, healthcare providers, and patients was a running theme during the conference. A primary example of this was the EA application process. The FDA has made changes to this process by updating the application form, thus reducing the time of completion from more than seven pages, down to two pages.
Ultimately, the goal of this program for the patient, physicians, and drug companies involved, is to have access to a safe and effective drug. With the role of EA and support from the FDA, the teams at DSN and WEP Clinical can ensure your company reaches that goal.