Newsletter | September 9, 2021

09.09.21 -- Principles And Pathways In Clinical Trial Development

A Primer on Cancer Immunotherapy Part 1: Goals and Major Approaches

Immunotherapy aims to manipulate the balance and bend the immune system curve to eliminate cancer while avoiding autoimmunity. This article provides an introduction to cancer immunotherapy, exploring its immunological basis and the fundamental principles guiding the development of new treatments.

Site Selection, Manufacturing, and Long-Term Follow-Up in a Transition Phase 1/2 Gene Therapy Trial

Designing and conducting a gene therapy trial is a complex undertaking. Understanding, planning for, and overcoming the myriad challenges of operationalizing these studies will help bring safe, breakthrough treatments to patients with unmet medical needs.

Tumor-Agnostic Treatments: A Game Changer for Rare Oncology Trials

As our understanding of the molecular characteristics of tumors has improved, there has been a tremendous leap forward — not only in targeted therapies but also in the development of tumor- or tissue-agnostic treatments. Now, with precision or personalized medicine, treatments can be targeted to a subgroup of patients rather than relying on a one-drug-fits-all model.

505(b)(1) Versus 505(b)(2): They Are Not the Same

The 505(b)(2) pathway can yield significant benefits in drug development cost and time. But what are the differences in 505(b)(1) versus 505(b)(2)? They are not the same. There are other pathways to approval for biologics (BLA) and medical devices (510k and PMA), but this back-to-basics article compares the innovative small molecule pathways for approval, 505(b)(1) and 505(b)(2).

Pre-IND Meetings: How to Achieve Success for 505(b)(2)

One of the greatest mistakes that the sponsor of a 505(b)(2) can make is to have an unsuccessful Pre-IND meeting. Common errors occur at the Pre-IND meeting because sponsors and CROs that are more familiar with traditional 505(b)(1) drug development programs fail to appreciate the different goals and the impact of a Pre-IND meeting on a 505(b)(2) program. Learn what tools can make a Pre-IND meeting successful.