Over the past 30 years, there have been dramatic advances in many areas, including biotechnology, genomics, diagnostic imaging,
molecular diagnostics, organ and tissue replacement, surgical techniques, wound care and computer technology, which have led
to improved healthcare delivery and patient outcomes (1). Patients and physicians alike want access to these latest treatment
options, but many have little regard for the cost involved and assume that these new technologies should be funded automatically.
Governments have introduced cost-containment measures to slow healthcare spending, but remain wary of public opinion when
making decisions about which treatments should receive funding. Of course, no government wants to be accused of overtly placing
cost restrictions on healthcare, particularly as this is likely to lose them votes in an election. In Europe, during the early
1990s, the word 'rationing' began to be introduced in the field of healthcare, but politicians began to steer away from this
terminology because it was found that voters tended to believe that 'rationing' was somehow depriving them of their basic
rights (2).
To manage the demand for expensive treatments in a more controlled way, governments have become increasingly reliant on healthcare
technology assessment (HTA). HTA is a multidisciplinary field of policy analysis, and studies the medical, social, ethical
and economic implications of development, diffusion and use of health technology. In principle, HTA is supposed to assist
payers in making informed decisions about allocating resources (including expenditure on medicines) in the health system and
its supporters believe that it better links medical innovation to the actual needs of the healthcare system (3, 4).
Many countries, both in Europe and elsewhere in the world, have dedicated HTA review bodies. HTA uses a number of different
disciplines to assess the benefits and, in some cases, costs of a given medicine. A large number of stakeholders are involved,
and HTA bodies typically include epidemiologists, economists, physicians, pharmacists, healthcare managers and, increasingly,
patient group representatives (3, 4).
Pharmaceutical companies, however, remain unconvinced that HTA results in an appropriate decision being made. Critics have
argued that a poorly designed or managed HTA risks denying patients appropriate access to medical technologies, and can also
restrict clinical freedom and lead to a waste of important resources (4). HTA approaches that target high-priced drugs also
send distorted signals to medical innovators and remove the incentive to research new therapies (4). Companies developing
new drugs have to recoup their initial R&D investment and commercialisation costs, and have no alternative but to set high
prices for their products. According to the Tufts Center for the Study of Drug Development (CSDD), the cost of developing
a new drug averages at about €2.1 billion (5).
One example of the frustrations that the pharmaceutical industry has had with HTA is illustrated by its dealings with the
UK's HTA, the National Institute for Health and Clinical Excellence (NICE), which frequently recommends against the adoption
of new drugs. Only last month (August) for instance, NICE decided not to recommend payment for Novartis' multiple sclerosis
(MS) drug Gilenya (fingolimod). NICE's provisional guidance stated that there were uncertainties over the clinical effectiveness
of the drug and that the evidence available did not suggest that it would be a costeffective use of healthcare system resources
(6). As well as angering Novartis, the decision attracted complaints from MS patient organisations, with the UK's MS Society
being quoted in the media as stating that MS patients would be better off living almost anywhere else in Europe than in the
UK because of the poor access to treatments (6). For Novartis, this was another setback with NICE. In April 2011, the body
had refused to recommend, on appeal, its kidney cancer drug Afinitor (everolimus).
