Tag: Rare Disease

Consulting

BIO International: Discover the Latest in Innovative Rare Disease Trial Design

DURHAM, N.C., June 5, 2019 — Premier Research is sponsoring the Orphan & Rare Disease session track at this year’s BIO International Convention, June 3-6 in Philadelphia. Our own Angi Robinson, vice president for clinical development services in rare diseases and pediatrics, will participate in the panel discussion Rare Finds: Innovative Clinical Trial Design for...

Medical and Regulatory Affairs

The FDA’s New Guidance on Natural History Studies in Rare Diseases: What You Need to Know

In March 2019, the FDA released draft guidance on the design and implementation of natural history studies to support the development of safe and effective treatments for rare diseases. The document, Rare Diseases: Natural History Studies for Drug Development,[1] addresses one of the major challenges sponsors encounter when developing therapies for rare disease: the lack...

Patient and Stakeholder Engagement

Patient Engagement Expert to Address Orphan Drug Congress April 11

DURHAM, N.C., March 25, 2019 — Premier Research’s head of patient engagement will discuss a patient-centered look at drug development at the World Orphan Drug Congress (WODC) USA in Oxon Hill, Maryland. Juliet Moritz, Premier Research’s newly appointed vice president of patient and stakeholder engagement (PASE), will present From the End to the Beginning: A Patient-Centric...

Consulting

Regulatory Oversight on Gene Therapy in the U.S. and EU

Decades of painstaking research have recently begun to yield gene therapy products that are delivering meaningful benefits to human health. The approvals of voretigene neparvovec-rzyl (Luxturna) for inherited vision loss, tisagenlecleucel (Kymriah) for lymphoblastic leukemia, and axicabtagene ciloleucel (Yescarta) for lymphoma have ushered in a new era of therapeutics. With the rapid evolution of the...

Patient and Stakeholder Engagement

Premier Research Champions Patient Advocacy Community

DURHAM, N.C., February 28, 2019 — As part of its commitment to supporting the patient advocacy community and as a kickoff to Rare Disease Day on February 28 and March as Rare Disease Month at the company, Premier Research is announcing both a new scholarship for rare disease patient advocates in partnership with Professional Patient...

From Psoriasis to Behçet’s: The Evolution of Biologics in Dermatology

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 rarer...

Consulting

Ashanthi DeSilva’s Story: A Look Back at the First Gene Therapy Trial

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. Hollywood...

Medical and Regulatory Affairs

Draft Guidance: Early Drug Development and the Role of Pre-IND Meetings

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.

Patient and Stakeholder Engagement

Premier Voices #8: Engaging Patients in Clinical Trials w/ Alan Thomas & Juliet Moritz

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 the...

Patient and Stakeholder Engagement

Premier Voices #7: Rare Disease Advocacy & International Ataxia Day w/ Alan Thomas (Ataxia & Me)

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 & Me...