Registrat-MAPI

New legislation in the US, Europe and other countries has made demonstration of drug safety a key priority for drug approval and continued marketing. To thrive in this new era, the pharmaceutical industry needs to incorporate new strategies and tactics.

Pharmaceutical companies will need to change their drug development efforts to plan for higher thresholds of approval related to the relative benefits and risks of pharmaceuticals. This involves the development of a more comprehensive process of assessing potential risks in relation to likely benefits, alternative therapies and the disease being treated to drive the development of the product profile.

This process also makes it easier to anticipate and develop post-marketing surveillance and risk minimisation activities that may be needed in the post-marketing period. Both of these activities are increasingly required by regulatory authorities throughout the world to ensure that the benefit to risk profile remains positive for patients using the medication.

These changes to both the drug development and commercialization approach will help companies to improve management of product risks and ensure commercial success.

Advancing Rare Disease Research

NIH, FDA, biopharmaceutical companies, and advocacy groups are actively involved in advancing research efforts to develop treatments for up to 30 million people in the U.S. with one of approximately 6,000 rare diseases. I am most fortunate to be involved with an NIH working group which has been assembled to design and implement a standardized registry for patients with rare diseases.

A workshop, Advancing Rare Disease Research: The Intersection of Patient Registries, Biospecimen Repositories and Clinical Data, was held January 11–12, 2010, in Bethesda, MD. The objective of the workshop was “to discuss the development of an infrastructure for an internet-based platform with common data elements utilizing a federated rare disease registry able to incorporate existing rare disease registries; patient organizations with no registry looking to establish one; and patients with no affiliation with a support group looking to belong to a registry.” The workshop agenda, presentations, publications, and information about ongoing registry development can be viewed at: http://rarediseases.info.nih.gov/PATIENT_REGISTRIES_WORKSHOP/

Food and Drug Administration Amendments Act of 2007

With the passage of the Food and Drug Administration Amendments Act of 2007 (FDAAA), Volume 9A Pharmacovigilance Guidance, and increasing focus on evidence based medicine, risk management, and comparative effectiveness research (CER) it is an exciting and challenging time to be involved in clinical research and the healthcare industry. During the past several years I have been directly involved in pioneering the concept and establishment of national/international patient and healthcare provider (HCP) driven disease registry programs supported and accessed by multiple stakeholders (patients, HCPs, Sponsors, payers, and governmental and regulatory agencies) to more effectively and efficiently meet the evolving regulatory, industry, and clinical practice demands in our healthcare environment. As a thought leader, and through my involvement with REGISTRAT-MAPI, I feel an obligation to leverage my years of experience working with numerous companies and multidisciplinary teams to be a leader in industry and innovation.