Sifting Through Rarity: Advanced Patient Screening for Rare Disease Trials

By Maria-Cruz Morillo (Global Therapeutic Operations Lead, Rare Disease) Because rare diseases affect a small population of people who are dispersed across the world, it makes country and site selection, patient identification, clinical trial recruitment and long-term retention particularly complex. Furthermore, many rare diseases are difficult to diagnose — in some cases they have been […]

Rare Central Nervous System Disease Trials

Precision medicine offers the promise to treat a variety of conditions, especially rare diseases, with targeted approaches. But developing more personalized medicines can introduce new complexities in the clinical trial process, which can hold back drug developers on the road to commercialization. Find out the factors that make rare neurological diseases a prime target for precision […]

When it comes to small populations, biotechs need to think big

Originally published by The challenges biotechnology companies face in developing new medicines are increasingly dynamic and complex—particularly in rare disease clinical research, where patient populations for some conditions may be less than 25 people worldwide. Now, more than ever, companies need to have the right expertise in place to remain competitive and meet the […]

Rare Disease Research – Why We Do What We Do

written by Richard Vesely, MD, VP, Regulatory Strategy Consulting Systemic juvenile idiopathic arthritis – can we do better? When I was a young pediatrician choosing my career path, I started flirting with the idea of pediatric rheumatology, and at the time, an older colleague told me: “Definitely choose pediatric rheumatology! It has one great advantage; Whilst […]

Roadmap To Marketing Authorisation in Europe: Taking All the Right Turns

Approval of your Marketing Authorisation Application (MAA) is the final hurdle to pharmaceutical product launch in Europe. Adequate and timely navigation of this roadmap is essential to achieve a successful review by the EMA. Navigation of the drug development pathways towards MAA approval starts much earlier than you may know till date and is essential […]

FDA Orphan Drug Designation for Rare Diseases

An estimated 30 million people in the United States are afflicted with rare (orphan) diseases or conditions; however, the availability of novel treatments for these diseases and conditions has been historically low compared to conditions with a higher prevalence. This is mostly due to the financial risks of recouping the costs of drug development. In […]

Rare Disease Research in Central and Eastern Europe

There are more than 7,000 rare diseases in existence right now and together they impact millions of people around the world. However, individually, a rare disease can be defined as impacting fewer than 5 in 10,000. When indications are that sparse, developing treatments for rare diseases is challenging. Choosing the right location can make a difference. While the […]

Understanding the Importance of Location in Rare Disease Studies

Rare disease isn’t rare. As a whole, more than 350 million people in the world have at least one rare disease – more than the population of the United States. Exactly what qualifies as a rare disease is another story. The statistical definition of “rare” varies by country. Officials in the European Union require that no […]

Next Generation Strategies for Orphan Drug Development

Original Air Date:  Monday, October 19, 2020 Time:  10:00 AM EDT/4:00 PM CET The past decade has witnessed a remarkable surge in rare disease research leading to an impressive pipeline of promising investigational products. Novel approaches, including genomics, data mining and computational repurposing of drugs will continue to fuel discovery and consequent clinical development of medicines. In […]

The Value of Orphan Drug Designation in your Drug Development Program

Original Air Date:  Tuesday, July 28, 2020 Time:  11:00 AM EST/4:00 PM London As a drug developer you may wonder if you have a portfolio product or development candidate that qualifies as an orphan drug and thus provides options unique to orphan drug development. There are approximately 7000 known rare diseases and more expected to be […]