The EveryLife Foundation, from its founding, has been focused on improving the development process for rare diseases through scientifically sound and practical solutions. Based on the experiences of Foundation President, Emil Kakkis, M.D., Ph.D, the Foundation has embraced a special niche role in addressing the complex scientific and regulatory issues. We believe that modest and intelligent changes to the system can dramatically help improve the way drugs are developed, and help more patients get treated. We are honored to have 181 patient organizations endorsing our CureTheProcess campaign.
One of our key goals has been improving access to the Accelerated Approval Process, which has been difficult to utilize for many rare and ultra-rare diseases. The Accelerated Approval Process allows for a surrogate endpoint or biomarker (such as a measurement from a blood or urine test that shows the effects of the drug) to be used in clinical trials to determine whether the drug is working instead of a clinical endpoint, such as death, which takes much more time to determine and significantly increases the cost to develop a treatment. Currently, the FDA requires prior clinical data to qualify what the biomarker means, in order to use it as a surrogate endpoint. For well studied diseases that have clinical data such as Cancer and HIV, Accelerated Approval has been extremely successful in getting treatments to patients.
However, the FDA should not require prior clinical data to qualify the use of a surrogate endpoint or biomarker in clinical studies for the rarest of disorders where such data is never available. For more common orphans, the issue is less problematic, as there is often more data on biomarkers or surrogates, and clinical endpoint-based studies are far more readily achievable. This change would make it easier to use existing good science in the laboratory to help move a treatment forward as long as the that science is sound and well done.
In the case of ultra-rare disorders, the number of orphan designations, the degree of development pressure, and the number of drugs approved have all lagged behind the more prevalent orphan diseases (see published analyses of Heemstra et al and our own analyses from BioMedical Insights). We have also shown that the cost of development is so high under the current regulatory requirements that the number of drugs being developed is still relatively few with only two approvals a year, on average. Greater access to the Accelerated Approval pathway is needed to help make the development of therapies for extremely rare diseases plausible.
Our proposal in the ULTRA Act is that for the rarest of diseases in the US, the FDA should be empowered to use all of the science available to decide if a surrogate endpoint is qualified for use in a clinical study and to accept surrogate endpoints without requiring prior historical clinical data when it does not and cannot exist because no study has ever been conducted in that disease. If this straightforward, commonsense approach is adopted, more effective drugs would attract investment and become available to treat individuals with ultra-rare diseases, which are now virtually ignored and underrepresented in drug approvals.
The bill does not lower the standard for drug approval but just makes it clear that FDA can focus its review on existing scientific information and not feel compelled to require clinical data, which is not available for the rarest of diseases. This change, small in focus, is the right step forward to opening the door to treatment of the forgotten and devastating disorders that are too rare for investors to care about, and too difficult (with respect to biology) for typical drug development. Those in the larger orphan disease community must see the plight of the ultra-rare disease community and come to their aid.
No one should be satisfied with the status quo or just an assumption of flexibility. We should take advantage of this opportunity, which only comes every five years with the reauthorization of the Prescription Drug User Fee Act (PDUFA), to make a simple but important change that will help bring life saving treatments for the rarest disorders.
Bill Language
Rep Stearns letter to FDA regarding ultra rare diseases
FDA Week: Patient Advocates Seek To Accelerate Approval of Ultra Orphan Drugs
For more information on the Bill go to CureTheProcess.org/ULTRA_Act
The ULTRA ACT is supported by more than 90 patient organizations, click to read the sign on letters to see the complete lists: House Sign on Support letter 11.29; Contact patientadvocate@kakkis.org today if you would like to add your organization to the letter of support.
