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.

Biopharma Excellence Runs for Life! 🏃‍♀️🏃‍♂️

As one of the global charity events, Biopharma Excellence participated in the Wings for Life Run, 2018 in Munich (Germany). Aside from supporting leading biopharmaceutical and life science companies in tackling the biggest health challenges of our times, our team is excited to have contributed by participating in a charity event that donates to spinal-cord research projects and clinical trials at renowned universities and institutes worldwide. 🏃‍♀️🏃‍♂️

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.

Join Us - Vacancies!

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

Seasonal influenza vaccination and the vision of next generation vaccines

by Dr. Michael Pfleiderer

Read publication as  PDF

Read publication as PDF

Holiday season is over and seasons quickly turn from summertime to wintertime. Shorter days and colder temperatures are accompanied by the yearly official reminders released by vaccination advisory committees not to forget the annual shot of influenza vaccine in order to update immune protection against drifted influenza virus strains.

Meanwhile, numerous vaccines are available including tri- and tetravalent vaccines, inactivated and live attenuated vaccines, unadjuvanted and adjuvanted vaccines. Moreover, age indications are very heterogeneous ranging from 6 months of age upwards to over 65 years only.

This puzzling situation is further complicated by the fact that efficacy and effectiveness of various seasonal influenza vaccines is quite heterogeneous in different age and risk categories turning choice of the right vaccine into a difficult exercise for both, doctors and vaccines. Furthermore, the short shelf life naturally inherent to seasonal influenza vaccines is problematic for regions with untypical and repeated epidemiological patterns not matching with those of the Northern or Southern hemispheres for which most of the globally available influenza vaccines are tailor made for.

For those regions, suitable vaccines are either not available or shelf life of available vaccines may have already expired when these vaccines are needed. To simplify these rather complex scenarios scientists and regulators call since a long time for next generation influenza vaccines providing broader protectivity across strains and seasons, circumnavigating the need for seasonal revaccination and being freed from stringent production cycles. WHO is coordinating these initiatives and has recently launched a Preferred Product Characteristics (PPC) document summarizing the requirements for better or next generation influenza vaccines. We have been deeply involved in these activities by contributing to the drafting and finalization process of the PPC document. Read more about the WHO PPC concept.

The European GMO legislation for gene therapy products in clinical trials and its pitfalls

Advanced Therapy Medicinal Products (ATMPs) such as Gene and Cell Therapy medicinal products are innovative biopharmaceuticals with the potential to bring high therapeutic value. Especially the new gene editing tools such as CRISPR-Cas9 are expected to shift the focus of medicine from treating symptoms to curing diseases. However, especially their clinical development faces specific challenges. For products containing genetically modified organisms (GMOs), an additional risk assessment step in the clinical trial authorisation (CTA) procedure is required. Although a European regulatory framework for this matter exists, it has been implemented differently into the Member State (MS) national legislation, leading to different documentation requirements and review and approval frameworks between the Member States.

In this article, Theodor Tiko and Annegret Vaggelas review the current status of the European GMO legislation for gene therapy products in clinical trials, including its pitfalls for clinical trial applicants.

Small Companies: Do you know about the advantages of obtaining an SME status?

Small biopharmaceutical companies are powerful engines of innovation, however, typically lack the resources, funding and expertise compared to big pharma companies. The European Medicines Agency (EMA) addresses the unique needs of micro, small and medium-sized enterprises (SMEs) through specific incentives – accessible through the SME status. For example, the most immediate and obvious incentive for early development stages is to save 90 % or more of the scientific advice fee which could go up to 80,000 EUR. SMEs inside and outside of the EU can obtain EMA’s SME status if certain criteria are fulfilled.

Annegret Vaggelas explains the requirements, the incentives, and the process to obtain the SME status. 

Hospital Exemption for ATMPs: Perspectives & Regulatory Trends Under Discussion

Advanced Therapy Medicinal Products (ATMPs) bear game changing potential for the treatment of severe conditions for which we have no appropriate therapies today. The product class of ATMPs comprises gene therapy medicinal products (GTMPs), somatic cell therapy medicinal products (sCTMPs), tissue-engineered products (TEPs) as well as combined ATMPs. ATMPs are complex products, and ATMP developing companies face diverse challenges during development as well as for commercialization.