Why Pancreatic Cancer?
Pancreatic cancer is typically diagnosed in the late stage of the disease, making it the fourth leading cause of cancer-related death in the United States. It is also one of the few cancers with an increasing incidence, particularly in the younger population. By 2030, it is expected to become the second leading cause of cancer-related death in the U.S. Patients with pancreatic cancer encounter monthly medical costs 15 times higher than those without, with costs highest in the later stages of the disease.
Since a pancreatic cancer diagnosis is typically made late in the disease process, the cancer is likely advanced or has spread to other parts of the body. Thus, fewer than 20% of patients are eligible for potentially curative surgical treatment. Given its typical late diagnosis, individuals diagnosed with pancreatic cancer have a very poor prognosis, with a relative 5-year survival rate of 11% overall. Even patients diagnosed with local disease (10%) have a 5-year survival rate of just 34.3%.
If diagnosed late and surgery is not an option, there is no broadly effective FDA-approved therapeutic intervention, specifically for pancreatic cancer. The lack of an effective therapy highlights a growing concern and demonstrates that the opportunity and timing for KAT 3-BP development and marketing for pancreatic cancer could not be more important than it is right now.
What is Significant About an Orphan Designation?
Unique Benefits of Orphan Designation Status
Why is it a good strategic decision to focus PrimoCure Pharma’s efforts on pancreatic cancer and eventually, other cancers including those with orphan cancer designations? An estimated 30 million people in the United States are afflicted with rare (orphan) diseases or conditions; however, the availability of novel treatments for these diseases and conditions has been historically low compared to conditions with a higher prevalence. In 1983, Congress passed the Orphan Drug Act to incentivize development of orphan drugs to meet unmet needs.
Orphan Drug Act
The Orphan Drug Act of 1983 was designed to promote the development of drugs, biologics, devices, or medical foods that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. By definition, a disease or condition is classified as “rare” if it affects fewer than 200,000 people total in the United States, or if the cost of developing a drug and making it available in the United States for such diseases or conditions will exceed any potential profits from its sale.
Incentives of Orphan Drug Designation
One of the greatest challenges for companies developing drugs for rare diseases is that due to the small target population size, sponsors are unlikely to recoup the cost of research, development, and approval from the orphan drug product. Critically, the broad therapeutic potential for KAT to treat many orphan cancer types, our ultimate "target population" is not limited to one specific cancer type. However, in response to these usual challenges, the FDA has created multiple incentives to make orphan drug development more financially possible for companies to pursue. Some of the incentives include:
7-year marketing exclusivity to sponsors of approved orphan products
Up to (50%) federal tax credit for qualified expenses incurred in conducting clinical research within the United States
Tax credits may be applied to prior year or applied over as many as 20 years to future taxes
Waiver of Prescription Drug User Fee Act (PDUFA) fees for orphan drugs
A value of approximately $2.9 million in 2021
Ability to qualify to compete for research grants from the Office of Orphan Products Development (OOPD) to support clinical studies for orphan drugs
Eligibility to receive regulatory assistance and guidance from the FDA in the design of an overall drug development plan.