Cell and gene therapies are changing the face of modern medicine – But how to find the right dose?

by Diane Seimetz

Finding the right dose for the first in human study can be a challenging undertaking for biopharmaceuticals. For cell and gene therapy products (commonly referred to as ATMPs), this is even more challenging. In this article, Diane Seimetz reminds us what Paracelsus’ toxicology principle has taught us, and she explains what the key questions for ATMPs are to guide a tailored dose-finding exercise.

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.

Four important considerations for the successful development of an in vitro or companion diagnostic test

by Linsey Reavie

In vitro diagnostic (IVD) and companion diagnostic (CDx) tests are crucial for the rapid diagnosis, response and treatment of diseases. IVD and CDx developers must satisfy a diverse set of evaluators to ensure commercial success, such as regulators, health care providers, physicians, and reimbursement organizations. While these sectors have recently seen significant growth, they have also been met with an increased demand from regulatory bodies to demonstrate the clinical utility for patient treatment. Linsey Reavie explains what to consider for a successful development of in vitro or companion diagnostic tests.

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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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.

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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.