Abstract
Pancreatic cancer has a dismal prognosis and only a few treatment options are available. In the European Union, pancreatic cancer classifies as a rare disease, allowing drug developers to apply for orphan medicinal product (OMP) designation. The aim of this study was to provide more detail on OMPs for pancreatic cancer. All applications for OMP designation submitted to the EMA between 2000 and 2019 were identified. For each medicinal product that received an OMP designation, the mode of drug action, use of protocol assistance, and current life cycle status was determined. Fifty-two medicinal products received an OMP designation. At the time of submission, eighteen OMPs were at the non-clinical and 34 OMPs were at the clinical stage of development. At least fourteen kinds of mode of action were explored in the condition. For eighteen out of 52 OMPs protocol assistance was sought. At the time of data analysis, one OMP received marketing authorisation and 24 OMPs were ongoing in development. Many medicinal products for pancreatic cancer received an OMP designation and the majority of these products was already in the clinical stage of development. Nonetheless, the success rate of OMPs for pancreatic cancer that reach the market is low, and increasing this rate is something to aspire. Fortunately, development is still ongoing for a part of the OMPs, and a few developers are planning to submit a marketing authorisation application in the near future. This however does not guarantee success, as pancreatic cancer remains a difficult disease to treat. Developers are advised to make optimal use of incentives such as protocol assistance, establishing (early) dialogue between regulators and drug developers and to agree on important topics such as clinical trial design.
Highlights
Pancreatic cancer has a poor prognosis and is currently the seventh leading cause of cancer-related deaths worldwide [1]
All applications for orphan medicinal product (OMP) designation for medicinal products for the treatment of pancreatic cancer submitted to the Committee for Orphan Medicinal Products (COMP) between 17 April 2000 and 31 December 2019 were included in this study
Between 2000 and 2019, a total of 80 applications for OMP designation for pancreatic cancer were evaluated by the COMP
Summary
Pancreatic cancer has a poor prognosis and is currently the seventh leading cause of cancer-related deaths worldwide [1]. The most common type of pancreatic cancer is pancreatic ductal adenocarcinoma [2], and many patients are diagnosed when the cancer is already in the advanced stage of the disease [3]. A few treatment options exist for patients with pancreatic cancer. Palliative treatment can be considered for patients with advanced or metastatic disease. Dependent on the performance status (PS) of the patient, FOLFIRINOX (PS 0 or 1), albuminbound paclitaxel in combination with gemcitabine (PS 0 or 1) or gemcitabine monotherapy (PS 2 and/or bilirubin higher than 1.5 x upper limit normal) can be considered as a first-line treatment option, according to clinical practice guidelines [6]. Overall survival is generally poor, as reflected by the median OS being less than 1 year in patients with advanced pancreatic cancer.
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