FDA’s Expedited Programs for Serious Conditions, as well as the Orphan Drug Designation, remain exceptional by design. However, the time has come to consider ways of transforming the exception into the rule: providing the benefits of expedited programs beyond products for serious conditions with unmet medical needs. The success of FDA’s expedited programs demonstrates that accelerated programs can work. The question is why there should not be an expedited program that is open to developers of virtually all drugs.
Addressing indications in which patients have serious unmet medical needs should remain a top priority. However, it is unclear why this should preclude expedited development of treatments for the quite substantial unmet medical needs of vast populations with chronic diseases. The current situation of patients with type 2 diabetes mellitus illustrates.
The National Diabetes Statistics Report, 2014 reports that 29.1 million people in the United States have diabetes. There are at least eight classes of drugs for treatment of type 2 diabetes and a standard of care that may start drug therapy with metformin, α-glucosidase inhibitors, orlistat or thiazolidinediones. But does the availability of such medications for type 2 diabetes mean this patient population has no serious unmet medical needs? Consider these datapoints on diabetes:
- Emergency room visits for hypoglycemia in 2011: 282,000
- Emergency room visits from hyperglycemic crisis 2011: 175,000
- Diabetics 40 or older with advanced diabetic retinopathy: 655,000
- Diabetics having chronic dialysis or kidney transplant: 228,924
- Non-traumatic amputations of the lower limbs of diabetics: about 73,000
- Deaths from hyperglycemic crisis 2010: 2,361
- Cardiovascular disease death rate, diabetics vs. nondiabetics: 1.7x
Note that in several instances, the incidence or prevalence of serious complications of diabetes exceeds the 200,000 upper limit on patient populations for diseases eligible for the Orphan Drug designation.
A new white paper proposes replacing phase 3 trials with an expedited pathway centered on something new, a phase 2c trial. This expedited pathway would be accessible to developers of new drugs even in indications where treatments exist that to some degree meet the needs of many patients.
Regardless of reaction to the proposed phase 2c pathway, it is essential to find ways to expedite development of new drugs for diseases in which serious complications alone reach epidemic proportions.