Drug repurposing: how to protect your asset by an integrated regulatory strategy

by Michael Firgens

Drug repurposing is an alternative cost-effective approach to drug development which builds on already approved drugs and compounds. However, the actual commercial success of a repurposed drug depends on achieving effective protection against competitors such as generics and other products with the same active pharmaceutical ingredient. In this article, Michael Firgens explains the key strategies for the protection of a repurposed drug.

Cutting costs for cutting edge therapeutic products: ATMP process optimization

by Elena Meurer

The affordability of ATMPs for the health care system and patients lies among the important aspects that clearly require further improvement. The overall high price of cell-based therapeutics, as known today, is driven by specific factors involved in development, manufacturing and clinical logistics. In this article, Elena Meurer explains where to cut costs in the manufacturing process of ATMPs.

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.

Advanced Therapies in Wound Management: What Needs to be Considered for an Integrated Development Strategy

by Diane Seimetz

The great potential of advanced therapies in wound management was recently demonstrated by a case study where the entire human epidermis of a boy with junctional epidermolysis bullosa (JEB) was regenerated using genetically modified autologous keratinocytes. Despite this, over the past 11 years, only 12 advanced therapies received a positive opinion for authorization in the EU, which underscores the necessity for an integrated development and regulatory strategy to realize the potential of advanced therapies, in particular for wound management. In this article, Diane Seimetz provides insights on the challenges companies are facing and what needs to be considered at each stage, from R&D up to authorization. Moreover, Diane Seimetz and Anne Vaggelas have recently published an article on this important topic in the Journal of Wound Care.

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.

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.

Reach the Market Smarter and Faster: Breakthrough Therapies, Orphan Drug Designation, Fast Track ... and the Right Strategy

“Breakthrough” and “Fast Track” are frequently heard buzzwords in the pharmaceutical arena. Looking at development times of up to 17 years from early R&D to product approval with further two years for pricing and reimbursement, it appears that the biopharmaceutical industry is rather on a “Slow Track”. Despite significant advances in medical science, there are still numerous patients without appropriate treatment options.