As industry interest in rare diseases increases, so does competition within an already challenging clinical research environment where patient pools are inherently limited and often geographically dispersed. To differentiate their trials and inspire interest among patients and other key stakeholders,...
Premier Research
For researchers who are developing or working with analytical procedures, the new ICH Q14 Analytical Procedure Development and revised ICH Q2(R2) Validation of Analytical Procedures drafts represent foundational guidance documents. Together, they describe suggested development and validation activities for the...
For researchers who are developing or working with analytical procedures, the new ICH Q14 Analytical Procedure Development and revised ICH Q2(R2) Validation of Analytical Procedures drafts represent foundational guidance documents. Together, they describe suggested development and validation activities for the...
Every challenge is a chance to grow In Poland, I studied engineering in college. I really enjoyed the science, and my professors encouraged me to get a PhD. I started out in organic chemistry but found I was more interested...
In 2020, the European Joint Programme for Rare Diseases developed a guide on patient partnerships in rare disease research projects. This guide still rings true today, and we summarize in this blog the key points for sponsors of rare disease...
It comes as no surprise that putting patients first is imperative for achieving success in rare disease development programs. However, to what extent can sponsors integrate patient input into their study design while still securing the high-quality data they need? ...
On November 1, 2022, the U.S. Food and Drug Administration (FDA) adopted an addendum to the guidance titled “S1B(R1) Testing for Carcinogenicity of Pharmaceuticals,” which had previously been finalized by the International Council for Harmonization (ICH). The guidance document and...
A job that combines teamwork and independence I loved science in high school; I couldn’t imagine going into any other field. And I was always interested in cancer. I did my PhD in Australia and had begun my career as...
The Pediatric Research Equity Act (PREA) was signed into law in 2003 to increase pediatric use information in product labeling and close knowledge gaps regarding the safety, efficacy, and appropriate dosing of drugs to treat children. PREA requires new drug...
Oncology & Hematology
The Rise of Radiotherapeutics: Brief History and Best Practices for Clinical Trials
Radiation therapy was first used to treat cancer more than a century ago, and nearly half of all cancer patients still receive it at some point during their treatment.1 Historically, most radiation therapy was given by delivering ionizing beams of...