Delivering results in the context of rare diseases poses specific challenges to Medical Affairs organizations, from defining Medical strategy and setting up operations to executing tactics. The main challenges stem from the nature of rare diseases, including a limited number of patients, gaps in the scientific knowledge, variable medical practices, restricted access to a small number of specialists in a few or even a single center, complex and often delayed diagnosis, scarcity of data at launch, limited precedence in regulatory paths, limited number of rare disease clinical trialists, high cost of developing therapies, low level of disease state awareness and communication challenges, and the ever-growing need for a continuous dialogue with patients, payers, and policymakers, among other hurdles. This chapter provides a framework for Medical Affairs professionals who are new to rare diseases or would like to compare their practices.
This chapter discusses the role of Medical Affairs at Medical Technology (MedTech) companies, highlighting the sole clinical responsibility along with aspects of Medical Affairs structure, strategy, and processes that are unique to MedTech organizations. Importantly, this chapter spans the many subtypes of MedTech including device technologies and diagnostics (implantable, in-vitro diagnostics, wearables, etc.) and will illustrate the important role that MedTech Medical Affairs plays in customer solutions, accurate and efficient patient care, and the evolution of new healthcare settings, including the home.
This chapter provides an understanding of how Medical Affairs is structured within different types and sizes of biopharmaceutical and MedTech companies. The chapter will describe affiliate, regional, and global structures, including Medical Affairs reporting lines and the role of the Chief Medical Officer (CMO) in Medical Affairs. This chapter will also discuss functions that are usually included in Medical Affairs, such as Medical Strategy, Field Medical, Medical Excellence, and others and will also address functions which may or may not be included in the structure of Medical Affairs, such as HEOR, Digital, Patient Centricity, clinical trial operations, and others.
