Perusing the Pipeline of Epilepsy Research
To address this issue, key decision-makers within the epilepsy community convened in February at the Epilepsy Pipeline Conference organized by the Epilepsy Foundation of America. This biennial conference surveys the current state of therapeutic innovation and product development in epilepsy and encourages strategic partnerships among stakeholders. Attendees included researchers and clinicians, as well as leaders from pharma, biotech, and nonprofit organizations.
A number of venture philanthropists, as well as a representative of the Milken Institute’s Center for Strategic Philanthropy (CSP) participated in the event. CSP works with a number of philanthropists who are interested in strategically deploying philanthropic capital to amplify medical solutions that can drive a field forward and change the lives of patients. With a robust portfolio of projects related to neurological disorders, including epilepsy, CSP was invited by the Epilepsy Foundation of America to inform our work in this area, with the specific aims of understanding the nuances of the clinical landscape of epilepsy research and identifying key gaps where philanthropy can have a high impact on the field.
Comparing key takeaways from the 2014 and 2016 conferences, it is evident that drug development in the epilepsy arena is slowly shifting away from a one-size- fits-all approach, given that epilepsy is now more commonly viewed as a collection of separate disorders referred to as “the epilepsies.” Depending on the cause of the seizures, the severity and type of seizure, and whether accompanying cognitive and behavioral impairments are observed, different treatment plans may be necessary. Researchers and industry are beginning to search for disease-modifying therapies that address the underlying causes of the different epilepsies separately rather than focusing on the outward symptom of seizures.
This shift is highlighted in the drugs currently in clinical trials, which are being tested for:
- specific epilepsy syndromes,
- specific etiologies that can cause epilepsy, or
- specific gaps in epilepsy management.
As one speaker highlighted, it is much more difficult now for companies to make a blockbuster epilepsy drug; however, they can make “mini-busters,” which would still significantly change the epilepsy treatment landscape. This paradigm shift has been, in part, prompted by industry realizing that a specific epilepsy syndrome or etiology may fall under a rare disease designation and therefore can be granted “orphan status” by the FDA.
Orphan designations are alluring to drug development because they provide a company with tax credits, fast track the company’s FDA application, and, if approved, allow the company to have seven years of market exclusivity. Just within the last six months, 10 new companies representing small and big pharma companies have entered the epilepsy space.
This is an exciting time for epilepsy research. However, there were also words of caution. As one attendee noted during a panel discussion, not all epilepsies can fall under the rare designation, and we cannot leave those patients behind. Moreover, not all epilepsy syndromes have a clear etiology, and research needs to better understand the difference between having a seizure and having that specific type of epilepsy. It will be interesting to see how the clinical pipeline landscape continues to change by 2018, when the next epilepsy pipeline conference is scheduled.