Premier Perspectives Blog

Insider Insights in Clinical Development

Over the last two decades, biologics have improved the management of patients with psoriasis and advanced melanoma, many of whom either did not respond to traditional treatments or experienced severe side effects from them. With their success in deadly forms of melanoma and more common chronic inflammatory conditions such as psoriasis, drug developers are now targeting rarerRead more

In 1976, pop culture icon John Travolta starred in the made-for-television movie The Boy in the Plastic Bubble about a young man with a compromised immune system. The premise of the film is that Travolta’s character has to decide between living in isolation behind a protective bubble or dying engaged with the physical world. HollywoodRead more

The FDA has released a new draft guidance for sponsors of drug and biological products for the treatment of rare diseases in early development and in the planning of and participation in formal pre-investigational new drug application (pre-IND) meetings. Read the full guidance here or click the image to download.

Clinical research certainly has evolved over the past three decades. There are more trials crossing a broader spectrum of indications, and advances in study design and execution have dramatically changed the way drugs and medical devices are evaluated and sold. But perhaps no change has been more significant than the growth in engagement on theRead more

This episode, we’re proud to share the first part of our conversation with Alan Thomas, patient advocate and founder of Ataxia & Me, ahead of International Ataxia Awareness Day on September 25. Alan talks about his personal journey with ataxia and how he first got started with patient awareness. Follow Alan and Ataxia & MeRead more

 Patient registries and natural history studies are vital tools for orphan drug researchers, who often struggle with limited understanding of diseases and sparse data due to small patient numbers. In the second installment of the Premier Voices podcast with Angi Robinson, Vice President, Clinical Development Services, Rare Disease & Pediatrics, and Juliet Moritz, ExecutiveRead more

It’s been 46 years since researchers Theodore Friedmann and Richard Roblin published a landmark paper in the journal Science that posed a provocative question: “Gene therapy for human genetic disease?” And while clinical trials for gene therapy drugs have become much more common in the past 20 years — more than 2,400 had been conductedRead more

Between navigating relatively uncharted territory and the practical limitations of most gene therapy delivery systems, ensuring successful clinical trials of these products is a serious challenge for even the most experienced researchers. Compounding this is the long-term nature of most gene therapy trials — it’s not unusual to have follow-up periods of a decade or more.Read more

Like any new medical product, gene therapies must undergo rigorous approval processes. However, there are a number of additional regulations for gene therapies that don’t apply to conventional pharmaceuticals. This is especially true within the European Union. (It’s important to note that while most gene therapy studies are based in the U.S. and Europe — whichRead more

Gene therapy is a hot topic in clinical research today — and for good reason! These technologies have the potential to treat — and in some cases even cure — a wide range of conditions, including rare genetic disorders that previously had no effective therapies. What Is Gene Therapy? Gene therapies are a diverse group ofRead more

From researchers to sponsors to patients to advocacy groups to clinicians, rare disease research is full of passionate, driven, and determined people. As the greatest asset to the field, all people involved must be utilized to their greatest potential for a study to be successful. That’s why significant attention must be paid to the operational challenges withinRead more

Rare disease and orphan drug research has a long history of serious challenges, thanks to both intrinsic factors—fewer patients means less available data; less available data means a less complete picture—and market forces—it’s more difficult to find investors when a smaller pool of patients may mean a more uncertain potential return on investment. Luckily, recent changesRead more

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