Gaining access to Europe’s pharmaceutical markets has become a multi-stage operation. The licensing of a new drug is only a preliminary step in what can be a lengthy and complex procedure for some pharmaceutical companies, particularly those with innovative biopharmaceuticals. First a drug has to be approved for its quality, safety, and efficacy by one or more regulatory authorities. This approval can be either by the central London-based European Medicines Agency (EMA) for a product to be marketed across the region or by a national licensing authority for one to be sold only in specific European countries.
To be made available, however, in the public sector health services a medicine has to be approved for reimbursement. These reimbursement decisions are increasingly determined by separate investigations of medicines often called Health Technology Assessments (HTAs). While the regulatory agencies deal with benefit-risk aspects of medicines, HTA bodies gauge their effectiveness in relation to existing products, particularly on the basis of cost.
The role of these assessments in European national pharmaceutical markets has become so important that EMA has agreed to collaborate with EUnetHTA, a European Union assessment network, on involving HTA bodies in giving advice to manufacturers in the early stages of a drug’s development.
The move has been welcomed by small- and medium-sized enterprises (SMEs), especially those developing advanced medicines. “SMEs are generally interested in receiving joint EMA/HTA advice, in particular when it comes to advanced therapies,” says Paolo Morgese, chairman of the HTA Topic Group of EuropaBio, a biotechnology trade association. “This is not necessarily linked to the development of new manufacturing processes per se, but more to the need of identifying a common set of data requirements,” he added.
EUnetHTA has been stepping up its efforts to achieve more consistency in the methodology and content of HTAs among the European Union’s 28 member states. The assessments, carried out by a range of public agencies and advisory bodies comprising key opinion leaders (KOLs), mainly comprise appraisals of clinical trial data to ensure that a medicine gives added therapeutic value. But they are also starting to cover types of formulation and manufacturing processes particularly when gauging cost effectiveness. National bodies do not always carry out assessments but they are also conducted by regional clinical and funding organizations within countries. Their support can be necessary for a drug to be put on local formularies.
The Impact of HTAs on Biosimilars
As a result of cuts in healthcare costs due to post-recession austerity programs, favorable HTAs are becoming crucial to the successful market launch of pharmaceuticals in Europe. HTAs backing reimbursement can be vital for new innovative medicines but can also be important to biosimilars.