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 […]

How to Overcome Challenges in Combination Immunotherapy Clinical Trials

Immunotherapy has quickly become a major focus in the world of cancer research and treatment. In recent years, there has been a surge in the number of biotech companies conducting clinical trials for combination immunotherapies.   What are Combination Immunotherapies?  The combination immunotherapy approach involves the use of two or more different types of immunotherapies in […]

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 […]

Infectious Disease Research: Importance of Site Location in Clinical Trials

Site location plays an important role in clinical trials. The impact of location comes into play for most therapeutic areas. In the same way that allergy studies need to be planned with respect to the allergens studied, infectious disease clinical trials have to take place where those diseases are prevalent. Natural Selection Amongst Pathogens Pathogens tend to […]

Fostering Inclusion and Diversity in Clinical Trials

Inaccessibility, inadequate engagement, and lack of trust—these are just some of the issues that limit inclusion and lead to a lack of diversity in clinical trials. So, why is diversity in clinical trials important? Diversity is important because there are many examples of drugs that behave differently from one population to another. Failing to understand […]

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 […]

Improving Informed Consent in Clinical Trials

Does more information mean better knowledge? Recruitment and retainment of patients for clinical trials can be a major challenge, especially for complex areas such as oncology since the accumulating knowledge in these areas leads to a major increase in the types of therapies under development. The field is expanding, both in the approaches used, from […]

Consideration for Ongoing Clinical Trials: Ensuring Participant Safety and Adapting New Processes during COVID‑19 Pandemic

1.0 INTRODUCTION The COVID‑19 pandemic has added an unprecedented set of challenges to the conduct of clinical trials including quarantines, travel limitations, site closures, and interruptions in the supply chain of investigational products. In acknowledgement of these challenges, the Food and Drug Administration (FDA) recognizes that protocol modifications may be required and protocol deviations may […]

Let’s Talk About Patient Engagement

Why does everyone talk about “Patient Engagement” in clinical trials? As trials are becoming longer and more expensive and many of them fail to meet their recruitment goal, patient engagement plays a key role in the success of a clinical trial. But what is exactly patient engagement and why is it so important? Getting to […]