NICE is the health technology assessment [HTA] agency in England, the one upon which others in many countries are modelled. More generally, HTA is increasingly being used by governments for more than simple economic evaluation of medicines and devices, but is being used as a form of rationing of access to technologies (medicines or devices are considered technologies in this sense), and for budgetary control. The more mundane scientific assessment of technologies alone, required for licensure, is gradually being replaced across Europe with a patchwork of regulatory HTA systems. Their operation has a direct bearing on what technologies enter health system use in specific countries and therefore what benefits patients and clinicians derive from the use of those technologies.
So much is not new.
With release of the Commission’s findings in its Pharmaceutical Sector Inquiry Report, 9 July 2009, there is the opportunity to reflect further on HTA methods across Europe. The report notes that one reason given by industry why fewer novel medicines were reaching the market was uncertainty about financial rewards (a factor in whether technology assessment would lead to regulatory and reimbursement approval). The report notes that with prescription medicines the patient is not the ultimate consumer, it being the doctor or pharmacist; to some extent this is true, but these clinicians are making decisions based on what products are on the market at any one time, and it is regulatory systems and the structure of formularies that determine this. In more extreme cases, decisions are embedded in prescribing guidelines and substitution protocols, which a child could follow.
The report also misunderstands how HTA works, and this may reflect the fact that member states pursue HTA for various reasons. Using the rather oblique term “added value” they see HTA as essentially linked to the “budgetary situation and health priorities of each member state”. This is, of course, precisely the problem. HTA IS an economic assessment, based on value add, but it is NOT defined purely in terms of affordability. It provides information to decision-makers about what the economic and social costs are for different thresholds of health outcome — a polite way of pricing rationing. In other words it informs decision-making, it does not determine it. It is quite possible to arrive at a Europe-wide HTA finding on anything you like, and leave it up to member states to quantify in their own terms where this sits within their own national system of affordability and a desired health benefits.
I doubt the pharmaceutical industry would think of advocating for a European Health Technology Assessment Agency, using consistent scientific and economic standards applicable to all countries, thereby simplifying their own regulatory hurdles (HTA is referred to as a 4th hurdle), because they dislike the 4th hurdle in the first place. But HTA is not going to go away, but misuse and abuse of HTA methods will likely continue.
My view is that the industry should advocate for such a system.
The argument is straight-forward. A “Euro-NICE” would bring order to the HTA process, and establish EU-wide standards for the application of HTA methodologies and enhance regulatory certainty which industry should like. It would have the additional benefit of creating a level-playing field amongst the different health systems, which would be a general benefit to patients.
It is important to understand that the pharmaceutical industry and individual member state health systems are locked in a very tight embrace, helped along by a country’s regulatory system and medicines pricing mechanisms. Movement by either has an impact on the other — reimbursement policies signal industry behaviours, availability of medicines determines what clinicians get to use and the medicines budget, regulatory ‘style’ links to investment in research, research investment acts to attract the big brains. It is a complex system, not a simple set of linear relationships (an impression one gets from the Commission Inquiry, unfortunately). And complex systems can cause perverse behaviours, which are invariably embedded in the rules themselves, and particularly important rules are HTA practices, not codified with in the formal medicines regulatory system, but which condition different member state markets.
While there is action on European HTA, through EUnetHTA, it is necessary to distinguish between doing HTA itself, and how HTA is used in decision-making within national policy systems, and regulation. A Euro-NICE would provide consistency in the former, to ensure transparency with the latter.