Despite the increasing range of guidance documents available, and the best efforts of those involved in producing them, product development sponsors are often faced with challenges that require a “case-by-case” and a “scientific-based” approach.

Stuart Mudge...

Regulation (EC) No. 141/2000 1 of the European Parliament and of the Council on Orphan Medicinal Products was drafted on December 16, 1999 and formally adopted by the European Commission on April 27, 2000. This regulation established a Community procedure for...

Technical advancements in molecular and cellular biology have the potential to provide an amazing array of human tissue engineering products (hTEP), and in doing so may revolutionize medical practice. The range of potential applications and the marketing potential...

With the exponential growth in biotechnology product development, it follows that there is a corresponding increase in biotech clinical trial experience. However, despite this increase in experience, biotech clinical development continues to be a challenging...

Cell therapy consists of the prevention or treatment of human diseases by the administration of viable cells which have been selected, cultured, often multiplied, and possibly pharmacologically treated or altered outside the body (ex-vivo). This article will present...

Innovative therapies pose special problems for drug developers and regulators alike. By definition, such therapies tend to leap forward into unregulated or incompletely regulated territory. Orphan drugs often fall into the category of “innovative therapies” and...

Biotechnology has the potential to provide solutions to a number of the world's problems but must develop globally harmonized procedures and regulations, while still acknowledging and embracing regional issues and differences. Emmanuelle Voisin reports from the...

The European Member States have decided that they cannot accept that certain individuals be denied the benefits of medical progress because these individuals suffer from a disease that affects only a small fraction of the population. It is, therefore, their role to...

Rare diseases are referred to as “orphan diseases”, and the products intended to treat them as “orphan medicinal products”. In addition to rare diseases, orphan diseases include those for which developing new therapeutic tools is not profitable. Following the...

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