In addition to disease-specific barriers, therapeutic developments in rare diseases face many challenges, such as incomplete understanding of natural histories to inform trial designs, need for alternatives to randomized controlled clinical trials, requirement for more sensitive outcome measures to quantify diseases, limited access to resources – including funding – required to set up clinical trials, and difficulties of recruiting small patient samples to participate in trials.
Among the above barriers, patient recruitment and enrollment is probably the most challenging, as enrolling enough patients in order to generate statistically meaningful results from a trial can turn to be highly time, energy and money consuming. Such a challenge may be overcome by good trial planning and design from the outset. Involving patient groups at as an early stage as study design could be rewarding as this can help suit patient needs and ensure those that join a study do not withdraw prematurely.
Planning, design and delivery of clinical trials is rare diseases may also be very challenging in small, dispersed populations, from both logistical and statistical perspectives. Here are some insights that could help on conducting a clinical trial in a rare disease:
In the setting of increasing challenges for the Pharma industry, medical affairs teams have wide opportunities to take the lead to improve their companies’ positions for a quality drug development, while better addressing needs from patient, physicians, regulators and stakeholders.
Tips to make medical affairs on the top could be summarized to the following:
To do so effectively, medical value teams need to be an integral part of cross-functional product development and product management teams. By working with all pharma functions, they can help increase the company’s understanding of unmet medical needs, and attractiveness of treatment pathways, thus valuating clinical trial protocols options, speeding regulatory approvals and increasing launch success rates.
Never before have Pharma companies developed data and scientific insights. But information channels are changing, and many companies have difficulty communicating science effectively with the growing array of stakeholders that influence purchasing decisions.