Drug Development

Genotype vs Phenotype - The impact of new insights from human genetics on the future of drug development

by Maria Schacker

The genetics of disease inheritance is becoming more and more important for the pharmaceutical industry. Until the early 2000s, the only way to study genetic diseases was through family studies and linkage analysis in families with high occurrences of disease. However, these studies are extremely tedious as they can only be done on a small scale and they are by and large only applicable to diseases that follow mendelian inheritance, not multifactorial diseases. In this article, Maria Schacker provides an overview on how the insights from human genetics can be used for the development of new drugs. 

Drug Repurposing – A Smart Development Strategy to Make Drug Development Cheaper, Faster, Safer and More Successful

by Michael Firgens

Drug discovery and the translation of research findings into new therapies require significant investments and resources by biopharmaceutical companies. Analyses across all therapeutic areas indicate that the development of a new medicine, from target identification through marketing authorization, takes over 12 years and often much longer. Michael Firgens will tell you how the repurposing of known drugs, serves as a more attractive, alternate development strategy.

Expediting the Development of Advanced Therapies such as CAR-T cells by making use of FDA’s RMAT designation

by Annegret Vaggelas 

In 2017, the first CAR-T cell therapies (Kymriah, Yescarta) have been approved by FDA. End of June 2018, EMA followed by recommending marketing authorizations for both of them in the EU. These first-in-class medicines hold enormous potential to cure some of the deadliest and largely untreatable cancers and are expected to shift the focus of medicine from treating symptoms or delaying progression to curing diseases. Further CAR-T cell therapies are under development and are expected to reach the market not too far from now. Read the article below to learn how RMAT works and which products can be considered for designation.

PRIME and Adaptive Pathways: A True Acceleration of Drug Development and Approval?

by Dr. Diane Seimetz

Read full article as  PDF

Read full article as PDF

Drug development times have increased enormously over the past decades with excessive investments of $1 billion for new drugs. Therefore, the EMA has launched the following two initiatives to improve timely access for patients to new and promising medicines: PRIME (PRIority MEdicines) and Adaptive Pathways. As PRIME and Adaptive Pathways are still young and up to now no PRIME designated products were approved, it is time to take a first look on their relative contribution to accelerate drug development and approval.


Diane Seimetz discusses the following questions related to PRIME and Adaptive Pathways:

·       What is Adaptive Pathways and how can it be used

·       What is PRIME and how can it be used

·       Can Adaptive Pathways and PRIME be used together

·       What other tools are available to facilitate drug development and approval

·       What is the value of the two concepts and further recommendations

Scientific Advice in the EU and US: Practical Experience and Rules of the Game on Both Sides of the Atlantic

by Dr. Gabriele Dallmann

Read complete article as  PDF

Read complete article as PDF

Drug development is a long-term, costly and high-risk commitment, becoming increasingly complex and global. What brings at the end the product successfully and fast to approval and to market at the price level envisaged by the company?

It is a holistic and well-integrated drug development and regulatory strategy, which had been put in place from very early on. An essential element is the interaction with agencies, i.e., a series of events planned globally and for a long-term period, at which the development program is exposed to agencies properly selected for the specific purpose in question to get their feedback and to enable the company to proceed with their planned next steps of development.

In her publication, Gabriele Dallmann provides practical hands-on advice on the scientific advice procedures in the EU and US and on how to use scientific advice in a value creating regulatory strategy.