Lost in translation? – How you can smoothly progress to clinical development and beyond

by Diane Seimetz

For a smooth transition to clinical development stage, careful and intertwined upfront planning across important drug development disciplines is essential. Based on our experience, even mature research teams and start-up companies often underestimate the challenge to progress their product candidates as well as their teams from the R-stage (Research) to the D-stage (Development). In this article, Diane Seimetz tackles the issue and provides solutions.

What ATMP developers should know when looking for a CMO

by Elena Meurer

As ATMP start-ups mature, they will face the following challenging decision: whether to develop, test, and manufacture its products in-house or to outsource the activities to a contract manufacturer (CMO). ATMP developers who do not have their own GMP facility or plans to set-up their own plant, will need to look for a CMO. In this article, Elena Meurer provides a comprehensive and structured guide how to select a CMO for the manufacture of ATMPs.

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.

Regulatory Science as an Important Principle of Good Consultancy Practices (“GCP”)

by Michael Pfleiderer

Regulatory science is the foundation of regulatory decision-making and is used to assess the quality, safety, and efficacy of human medicinal products throughout their life-span. The domains, covered by regulatory science, are considered to include both basic and applied biomedical sciences, clinical trial methodology and epidemiology, and social sciences. In the report by Michael Pfleiderer, you can read about the importance of regulatory sciences to contribute to the development of new tools, standards, and approaches to assess the safety, efficacy, quality, and performance of regulated products.

The Devil is in the Details – Trouble Shooting for Biopharmaceutical Products

by Karl Heller

Harmonious flow of the industrial processes greatly depend on systematically complying, updating and maintaining the SOPs. However, sometimes the worst happens and the product of the industrial manufacturing is not in compliance with the quality standards, which underscores the deviance from the SOPs. Karl Heller will tell you how such cases are handled and how trouble shootig is conducted in the processes of industrial scale.

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.

Regulating Bacteriophages: The First Steps into the Post-Antibiotic Era

by Luka Kotrikadze

It was in the year of 1915 when Frederic William Twort published the paper in The Lancet describing the discovery of an infectious agent that causes the lysis of the Staphylococcus hyicus bacteria. This served as the first ever published description of bacteriophages, therapeutic potential of which was only later assumed by Fe´lix d’Herelle. With the advancements into the research soon foregone with the discovery of the antibiotics, nowadays, with the increasing attention towards the antimicrobial resistances, the bacteriophage therapy is slowly stepping into the light of its come back. You can read about the current regulatory and product associated issues of bacteriophages, and the industry standpoint in the article by Luka Kotrikadze.

Biopharma Excellence at BIO International Convention 2018

by Gabriele Dallmann

You typically react with respect – big respect - when you talk about participation in the annual BIO International Convention, THE event for all biotechies. BIO celebrates this year 25 Years on BIO Innovation, and we decided to be there this year! Read about Gabriele Dallmann’s plans and expectations from BIO 2018 in Boston.

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We are expanding our team and are looking for unique biopharmaceutical experts for our office in Munich. 

Biopharma Excellence is a biopharmaceutical consulting company that supports national and global life science companies from top 10 pharma to emerging start-ups. The Biopharma Excellence team consists of experienced experts with a proven track record at agency, industry and service provider. We provide targeted solutions for the development and approval of biopharmaceutical products and support our clients to excel. This is our commitment to excellence.


Three important factors to consider for successful clinical development

by Diane Seimetz

Clinical studies are not only a pre-requisite to bring new therapies to patients, they are substantial value drivers within the biopharmaceutical industry. By progressing to the review of the marketing authorization application, the value of a drug increases to approx. 1,300 million. Yet, an analysis performed by our Biopharma Excellence team shows that consistently about 20% of marketing authorization applications fail. To achieve the goal of selecting the right patients while at the same time managing uncertainty within the drug development process, adaptive clinical trial designs and biomarkers are important development instruments.

In this article, Diane Seimetz shares insights on three important factors for successful clinical development.

Orphan Drugs Around the World – Current Situation and Future Perspective of New Markets

by Marietta Seuß

Orphan drugs (ODs) which are used for the treatment of rare diseases are often not developed or marketed because their extremely limited use makes them unprofitable. In 1983, the US took a first step to meet people’s needs: the Orphan Drug Act (ODA) was established. Based on this law, the developing company of an orphan drug can rely on numerous beneficial incentives when bringing an orphan drug to the market.

Marietta Seuß summarizes the situation of orphan drugs in the US and other countries and explains the incentives for developing orphan drugs such as the Rare Pediatric Priority Review Voucher in the US.

From IMPD to IND – same but different

by Rajeswara Rao Pannem

The aim of an investigational new drug application (IND) is to obtain approval from FDA to perform clinical trials of an investigational medicinal product (IMP) in humans in the US. The IND follows the CTD structure developed by ICH and requires very detailed product and development data such as information of manufacture, data from nonclinical studies, previous clinical studies if any related to the IMP (Figure 1). It is also required to provide comprehensive source documentation including study reports. It is mandatory to submit an IND in the eCTD format starting from May 2018.

Ensuring regulatory and scientific compliance with the requirements of the Common Technical Document (CTD) of data generated by manufacturers or manufacturing consortia

by Michael Pfleiderer

Manufacturers of medicinal products operating within and outside the European Union (EU) or the European Economic Area (EEA) who aim at placing their products on the EU/EEA market often find it difficult to correctly interpret data requirements for a valid CTD. Uncertainties on the amount and quality of scientific data to be generated and on the way how to optimally present these data within the individual sections of the CTD very often create a major hurdle that cannot be overcome easily. Most notably, uncertainties and discrepant opinions on these issues can significantly complicate partnering processes between two or more enterprises that are dependent on each other for successful drug development and licensure.

A guide to scientific due diligence: How to set up an efficient process

by Diane Seimetz

When being asked “What is a scientific due diligence?”, what would you respond? Would you have a straightforward and simple definition at hand? If not, you may want to consider our definition: Due diligence is making sure you get what you expect. This sounds simple, yet the process can be a daunting prospect. In particular for those companies where the due diligence has to run in parallel to the day-to-day drug development business. In this article, Diane Seimetz explains how to set up an efficient due diligence process.

How should we regulate genome editing for pharmaceutical applications?

by Dr. Diane Seimetz

Genome editing technologies are not only having a game-changing effect on research. They also have the potential to transform health care by curing diseases for which there are currently no or no satisfactory therapies available.

In particular with the emergence of novel gene editing technologies such as CRISPR/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats) in the pharmaceutical arena the question arises how to best regulate these technologies.

On 18 October 2017 the European Medicines Agency (EMA) organized an expert meeting on genome editing technologies used in medicinal product development. Diane Seimetz contributed to the meeting as invited expert and has summarized the discussion and critical aspects in this article.

The value of human challenge trials in vaccine development

by Dr. Michael Pfleiderer

Clinical trials in vaccine development are often hugely dimensioned sometimes comprising several ten thousands of individuals for phase III safety and efficacy studies. These sample sizes are required in order to demonstrate efficacy of vaccines for which no correlate of protection exists or for which very rare severe adverse effects need to be investigated pre-licensure. Besides the ongoing debate whether regulatory practice should ask for even more extended studies or whether there should be an upper limit of study sizes in order to ensure that clinical studies continue to be feasible, controllable and affordable there is an increasing number of vaccines that cannot be investigated for efficacy for several reasons.

These belong to the growing group of vaccines against newly emerging or regularly re-emerging pathogens such as Chikungunyavirus, Ebolavirus, MERS and SARS Coronavirus, West Nil Flavivirus, Zikavirus as well as against numerous microbial pathogens and parasites. Diseases caused by many of these pathogens do not occur regularly but appear and disappear in unpredictable epidemiological patterns making it virtually impossible to design a field efficacy trial since the region of emergence or re-emergence is unknown.

Co-development Drug and Companion Diagnostic: Lessons Learnt from the Development Program of Pembrolizumab (Keytruda)

by Michael Firgens

Companion diagnostics are medical devices which provide information that is essential for the safe and effective use of a corresponding drug facilitating personalized therapies in which treatment can be customized for an individual patient. It is reasonable to combine drug and companion diagnostic development, however, co-development programs must overcome regulatory hurdles and additional complexity until marketing authorization approval.

The development program of Pembrolizumab is an example for the successful co-development of a new biological drug accompanied by a companion diagnostic. Michael Firgens summarizes several lessons from the co-development of Pembrolizumab and its companion diagnostic that can be considered for future co-development programs of medicinal products.

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