Pramod Kumar T.M

In view of digital global agenda, the time has come for Good eCTD Practices. This article holds discussion about the measures to be taken before preparing and while submitting the dossier in eCTD format. The eCTD is an interface between industry and agency for transferring regulatory information while at the same time taking into consideration the facilitation of the creation, review, lifecycle management and archival of the electronic submission. eCTD is an electronic arrangement with extension of CTD, whose structure is specified by XML (Extensible Markup Language) eCTD DTD (Document Type Definition). While many organizations have delayed their adoption of eCTD format, the USFDA and several other global agencies are pressing forward towards its mandate. Though eCTD specification lists the criteria that will make an electronic submission technically valid, many companies are suffering from delays that occur not due to lack of data but due to technical and other issues of eCTD submission. The eCTD submission requirements definitely pose great challenges to the industry and regulatory agencies. There has to be an investment in information and communication technology as well as development of digital competitiveness among the regulatory professionals within the industry and regulators, alike. The regulatory professionals should know the standards, groundwork, expertise and technology required to submit an electronic submission globally. Due to its cost effectiveness, and because it guarantees a response from the recipient regulatory agency, reducing time to market, the eCTD has become the standard for numerous regulatory agencies around the world.

  The imminent patent expiration of many biopharmaceutical products will produce the possibility for generic versions of these therapeutic agents (i.e. biosimilars). However, there are a number of issues that will make approval of biosimilars much more complicated than the approval of generic equivalents of conventional pharmaceuticals. These issues center on the intrinsic complexity of biopharmaceutical agents, which are recombinant proteins in most cases, and the heterogeneity of proteins produced by different manufacturing processes. The increased occurrence of antibody (Ab)-mediated pure red cell aplasia (PRCA) associated with change in the formulation of one particular epoetin-α product highlights the potential for increased immunogenicity of recombinant proteins with different formulations, or those manufactured by different processes. The subsequent production of ‘biosimilars’ has aroused interest within the pharmaceutical industry as biosimilar manufacturers strive to obtain part of an already large and rapidly growing market. The potential opportunity for price reductions versus the originator biopharmaceuticals remains to be determined, as the advantage of a slightly cheaper price may be outweighed by the hypothetical increased risk of side effects from biosimilar molecules that are not exact copies of their originators. This review focuses on the issues surrounding biosimilars, including quality control, clinical efficacy and side effects.

An orphan drug is a pharmaceutical agent that is used to treat a rare medical condition (viz., Huntington’s disease, myoclonus disease, Tourette syndrome etc.). They receive little attention from pharmaceutical companies as the small patient population could not justify the huge investment required for drug development. In the last 20 years, orphan drug act has been adopted in several countries around the world (USA, Japan, Australia, and the EU) and has successfully promoted R&D investments to develop new pharmaceutical products for the treatment of rare diseases, but it faces future challenges like returns on the huge R & D costs, funding sources and initiatives for development of orphan drugs.