As our understanding of the genetic and molecular basis of cancer advances, rare oncology research is accelerating at an unprecedented pace. In fact, in 2014, more than 40 percent of U.S. Food and Drug Administration’s orphan drug designations were for rare cancers. Moreover, the trend toward increasing international cooperation among big pharma, biotech, and academia...
Pain is a significant health problem that impacts quality of life and imparts high costs to society. Despite intense research effort and progress in our understanding of the mechanistic and molecular basis of pain, there have been few real breakthroughs in novel analgesic drug development over the past 50 years. One major obstacle to the...
Children are not little adults, and protocols for pediatric pain studies must take into account the unique needs of children to meet regulatory and ethical standards and protect this vulnerable population from untreated pain. Sponsors of pediatric analgesia clinical trials are tasked with designing studies that are both realistic to execute and sufficiently rigorous to...
The success rate of investigational compounds eventually approved for clinical use in cancer remains the lowest among all diseases. Of the more than 750 drugs currently under development for the treatment of cancer, it is predicted based on past performance that only a few will ultimately demonstrate sufficient efficacy and safety for regulatory approval and...
When applied to clinical development, Quality by Design is an approach that focuses effort on prospectively identifying and managing risk to improve clinical trial quality and outcomes. The application of Quality by Design principles is particularly important in rare diseases, where the limited, geographically-dispersed, and often vulnerable patient pool leaves little margin for error. By...
Sponsors of psychiatric clinical trials may face significant challenges in collecting robust, quality data to support the efficacy and safety of investigative compounds. Identifying and mitigating study design-, subject-, and site-related factors that may influence data quality as early as possible in program development can help to drive study success. Ensuring that psychiatric clinical trial...
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....
Although current limitations in neuropsychological testing may discourage development of treatments for central nervous system (CNS) disorders, some researchers see these unmet needs as an opportunity for innovation. Take a look at three strategies that are improving the validity and viability of neuropsychological testing. Rater Training Success or failure of a CNS trial can come down to...
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 it comes to clinical trials for neurodegenerative disease (NDD), it’s easy to get overwhelmed by complexity. Not only are the mechanisms that underlie NDDs like multiple sclerosis and Alzheimer’s still largely a mystery, their inherent variability is hard to account for. The best way to combat the problem? Break trial design down into more manageable pieces....