In many rare and ultra-rare disease studies, sponsors do not have the luxury of choosing the countries or sites where the study will be conducted. Instead, they must use the sites where those patients are already being treated, regardless of geography. Additional protocol criteria – such as treatment-naïve patients or general prevalence of a disease...
Orphan drugs are medicinal products intended to treat diseases so rare that sponsors are reluctant to develop them under usual marketing conditions. Orphan drugs are a growing market, due in large part to tax incentives, longer periods of market exclusivity, and shorter, smaller clinical trials, as well as the premium pricing associated with many orphan...
Despite the inherently small market for orphan drugs, orphan drug development has recently undergone significant growth, with global sales of over $100 billion in 2015. In 2015, more than 20 new drugs were approved by the U.S. Food and Drug Administration (FDA) and 18 new drugs were approved by the European Medicines Agency (EMA) for...
Ensuring the integrity of your surrogate endpoints Rare disease studies present unique obstacles to drug developers, including small patient populations, slow disease progression, lack of prior clinical research, and uncertainty around validating biomarker measurements intended for use as surrogate endpoints. Our white paper describes a fit-for-purpose approach to validating bioanalytical methods in orphan drug trials....
While drug discovery has seen major advances over the last few years — especially in the domain of rare disease research — the number of new drug approvals has not kept pace with increasing development costs. However, the innovative use of biomarkers has the potential to change that trend. Biomarkers in Rare Disease Research Biomarkers are objectively measurable characteristics...
A variety of factors can make it difficult to conduct traditional full-scale clinical trials for new treatments of rare cancers. Consequently, because so little information is available, treatments for rare oncology patients are inadequate or nonexistent. Luckily for both patients and researchers, the FDA and the European Medicines Agency (EMA) have introduced regulations that expedite review and approval of certain investigative drugs. Expedited FDA...
A rare disease, also referred to as an orphan disease, by definition affects a small percentage of the population. Most are genetic and appear early in life, with one in three children with a rare disease dying before their fifth birthday. While the definition of “rare” varies from country to country, the US defines a rare disease as...
A rare cancer is often defined as one with an incidence of less than 15 per 100,000. The patient populations of these conditions are so small that traditional clinical trial design strategies can be unfeasible. But, with such a low incidence, just why is rare oncology research so important? Why Rare Oncology Needs Innovation Keep in mind...
When developing treatments for a relatively common illness or disease, patient recruitment is relatively straightforward. Screening and data collection can generally be done in a centralized location in a highly controlled environment. Now, imagine the concentration of people with the disease is not high enough in your local area, or even your country for that...
The year 2015 was a productive one for introduction of drugs that target rare diseases. U.S. regulators approved 21 new orphan drugs, a 40 percent increase from the previous year. European regulators approved a record 18 orphan compounds, a small increase over 2014. Any progress is a good thing, but these advances pale when weighed...