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...
Actively seeking the involvement of pediatric patients and their parents throughout the life cycle of a clinical trial can help drive recruitment, retention, patient satisfaction, and, ultimately, the success of the study. Involving children in clinical research has long been a sensitive topic associated with several ethical dilemmas and operational challenges. There is now a...
Rare disease and pediatric studies present significant challenges, including country selection, site selection, and patient recruitment. A thoughtfully-designed, well-executed feasibility study can help sponsors obtain a realistic assessment of the best path forward for developing and conducting successful global clinical trials. Performing a thorough assessment of clinical trial feasibility is an important early step in...
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...
Breakthrough therapy designation in the U.S. and PRIority MEdicines (PRIME) in the EU are two programs intended to facilitate and expedite development and review of new drugs to address unmet medical needs. By successfully engaging with regulatory authorities early in the clinical development process, sponsors of eligible investigational drugs can help ensure that their development...
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...