Linked to EAHP Statements
Section 2 - Selection, Procurement and Distribution: Statements 2.1, 2.5.

ACPE UAN: 0475-0000-21-013-L04-P. A knowledge-based activity. 

Abstract

There are a number of medical indications in clinical practice with unmet medical need regarding treatment with effective and safe drugs (e.g., in oncology and rare diseases). The pharmaceutical industry is in a position, either alone or together with academic research, to address this unmet medical need to the benefit of all patients. However, we see that the pharmaceutical industry focuses far too much on a few lucrative indications, coupled with extremely high prices making affordability sustainability and access to new drugs a significant challenge.

The pharmaceutical industry complains low returns on investments and an unpredictable and often infeasible regulatory approval pathway. This, in turn, is cited by many companies as a reason to leave indications that don't bring the requested return on investment. At the same time, we see companies nowadays focussing on biologicals, biosimilars, and generics - primarily in the field of cancer drugs - for their developmental pipeline.

What options are there to overcome this situation and are those pipelines appropriate to address indications where we find an unmet medical need? What are the ethical aspects of drug research and development? Is return-on-investment adequate or can we implement also other mechanisms to motivate pharmaceutical industry to bring really innovative drugs in the market which cover an unmet medical need instead of mainly being targeted on the expectations of shareholders?

This seminar will present approaches to the problems above and discuss different viewpoints from two different stakeholders in the health care system: an oncologist and a medical ethicist who will describe the current unsatisfying situation regarding research and development of new drugs as well as their call for proposals from politicians, pharmaceutical industry as well as regulatory agencies.

Learning objectives
After the session, participants should be able to:

• discuss the different measures to overcome the problem of unmet medical needs due to empty (research) pipelines; 
• list possible measures to motivate drug research and development of new drugs for relevant indications; 
• list ethical aspects of drug development costs and prices for new drugs; 
• discuss the existing problems of drug therapy in indications where an unmet medical need regarding drug therapy already exists; 
• list possible activities of health care professionals to solve these problems; 
• discuss the role of regulatory science.

Educational need addressed

Hospital pharmacists are not only confronted with drug shortages but also with the situation that many drugs are used off-label. More than that some drugs for some indications are not available in the market anymore. Hospital pharmacists, therefore, should learn about incentives from the regulatory and scientific perspective to do research and development in those indications where an unmet medical need exists.

Keywords: Unmet medical need, research and development of new drugs, return on investment, regulatory science, high-priced drugs and their added therapeutic benefit, pharmacoeconomics.