The pharmaceutical industry, characterized by its ever-changing landscape, is witnessing a profound surge in orphan drug development. And while drugs aimed at treating or curing rare diseases might seem niche, their impact is surprisingly vast. What are some of the key orphan drug challenges?
In MedCity News, Within3 Vice President of Product Commercialization and Training Natalie DiMambro writes about the opportunities within the orphan drug market – and how, even with several of the top-selling orphan drugs also among the world’s best-selling pharmaceuticals overall, the journey of developing an orphan drug isn’t without its challenges.
Clinical trials for these drugs come with a unique set of complexities. Given the rarity of the conditions they target, there’s an inherently limited patient pool, further compounding challenges in trial design and recruitment, especially without comprehensive data or consensus on the diagnosis of certain rare conditions.
Drug developers also need policies and technological infrastructure to execute their goal of being patient-focused. At a minimum, this means a compliant way to engage patients and truly hear their voices; at its most effective, patient engagement also meets patients where they are – whenever and wherever that may be.
Now, pharma companies are leveraging technology, especially network analytics and artificial intelligence, to bridge these gaps. By identifying and effectively engaging key stakeholders, from pioneering researchers to patient advocates, they’re reshaping how these trials are conducted.
Read the full article in MedCity News.