'Cell Therapies and Tissue Engineering'
Biotechnology’s techniques have opened up the prospect of a range of new therapies which may address some of these conditions. Protein based drugs, such as recombinant insulin, are of value in many conditions despite the problems of products that cannot be taken orally. Additionally, most protein-based products and many prospective antibody products suffer, like small molecules, from the need for regular administration of small doses, frequently for many years in the case of some essentially prophylactic products.
We believe that biotechnology will produce many new types of therapy in the future, some of which will make a permanent improvement to a patient’s condition with only a single application or a short programme. They will achieve this by harnessing the exquisitely accurate and efficient mechanisms that nature has itself devised. We also believe that regulatory systems designed for the examination and approval of small molecule drugs, and adapted somewhat for biopharmaceuticals, are not yet attuned to the cell- and gene-based products that can now be envisaged, and which are beginning to enter clinical environments.
This position paper identifies some potential therapies based on Cell Therapies and Tissue Engineering and comments on the regulatory developments that may be needed to address them.
This is anything but the case in the context of the new types of therapies under discussion in this paper. Safety and efficacy will of course remain paramount but it is not completely clear what these terms mean in the context of a therapy based on living cells. We are still largely at the stage of "guidelines", "notes" and "points to consider". Moreover, standard practice, such as positive controls and double-blind trials for efficacy assessment, will often be impractical. And, some diseases for which orphan drugs will be used have insufficiently large populations for statistical significance to be assessed by standard methodologies.
In only a few countries has there been adequate preparation of the regulatory systems to the extent that it is clear about what questions have to be answered before a therapy can be approved, and which regulatory bodies should be approached. Indeed, in some countries there is no specific "competent authority" (one which has legal status and empowerment to make judgements on this type of product) and establishing one may be part of the political process. In the UK for example, the possibility of xenotransplantation identified the fact that the necessary capabilities lay outside the remit of any existing regulatory body; as a result UKXIRA was rapidly convened with some guidelines issued by the government. But, the new body was obliged to spend its first several months of existence trying to define the processes that it considered relevant to its task, with consequent delay to interested parties.
As a result, MRL is extremely well placed to assist companies with such novel therapies, even more so when they address regulatory and market issues, including reimbursement, in more than one jurisdiction. In this sense, Europe may seem unified but there are differences of procedure and philosophy between different countries that can surprise the unwary. MRL’s aim is to protect its clients from the adverse impacts of such variations and smooth the path to market entry.
MRL at BIO