Masterstudiengang "Drug Regulatory Affairs"
Master-Thesis
Scientific and regulatory concepts for the development of molecular targeted anti-cancer drugs ***
Dr. Jan Richter (Abschlußjahr: 2004)
Safety and efficacy are key requirements for approval of any new therapeutic agent. This has led to the traditional paradigm for drug development where typically thousands of patients, hundreds of millions of dollars, and many years of clinical trials are required. Unless this traditional approach is changed, it will be challenging to find enough patients, money, or time to evaluate all of the promising new anti-cancer agents in development. New clinical trial designs and endpoints are urgently needed to permit more efficient evaluation of putative cancer treatments so that the most promising agents can move forward quickly, while disappointing agents are rapidly identified and discarded.
Based on these evolving insights, it has been proposed to amend and revise the current CPMP Note for Guidance on Evaluation of Anti-cancer Medicinal Products in Man (CPMP/EWP/205/95, July 2003) to better comply with current methodological thinking regarding the development of non-cytotoxic, targeted anti-cancer compounds. In this context, the CHMP actually discusses approval criteria for non-cytotoxic therapies with government agencies, academic centers, co-operative groups and professional societies.
Likewise, the FDA is currently engaged in an internal systematic review of disease related endpoints and plans subsequent discussions in a variety of public venues. In addition, a collaborative FDA/NCI/ASCO/AACR effort is also sponsoring a series of public workshops to review clinical endpoints in major cancer settings, which will then be discussed at FDA Oncologic Drugs Advisory Committee meetings. Surrogate endpoint biomarker expert panels complement these efforts by providing a forum targeted toward discussion and review of advances in biomarker development and implementation.
This thesis discusses scientific and regulatory concepts of the changing paradigm in mechanism-driven oncology drug development. It also elaborates the regulatory environment and requirements for combination trials of marketed and investigational agents to obtain more effective anti-tumor activity. It concludes with a discussion of recommendations, new initiatives and collaborations that have evolved from recent trends to expedite drug development with the goal to improve public health and to provide rapid access to affordable, innovative and safe medicines.
Pages: 61