Treatment Is Worth a Lot - But Just How Much

Abstract

More than at any other time in history, science offers enormous potential to transform the prevention, diagnosis and treatment of many diseases. However, patients are urgently awaiting new therapies - and too often not getting them. And researchers and companies also face significant development obstacles as only one in ten drugs entering clinical trials ever makes it over the finish line. This article looks at the issues involved in bringing innovation into healthcare systems from a political and policy level. It takes a close look at Health Technology Assessment (HTA) across Member States, how it is working and, more to the point, how it isn't, and how it often serves to stymie the introduction of new medicines by undermining the European Medicines Agency (EMA). The article argues that the EU could strengthen HTA cooperation via a common framework, and promote higher quality and fairer assessments; while joint work could reduce duplication of efforts, and promote better synergies as well as better use of Member States' resources. The author also takes an in-depth look into criticism of private-sector pricing of innovative medicines in the light of discussions about what constitutes “value” in the context of new treatments - a discussion that shows no signs of abating - while advocating enhanced contact and the need for compromise between all stakeholders in the great value debate.

Defining ‘therapeutic value’ of medicines: a scoping review

“…We identified 48 articles that matched this category, focusing on the following aspects: quality of life (31 studies),3 4 11–39 burden of disease (including severity of the disease, unmet medical need, 39 studies)3 11 13 15–17 19–22 24–32 34–37 39–54 and convenience (eg, administration route, 16 studies)3 11–13 15 22 24 28 29 31 35 36 49 52 53 55 (table 2).…”

“…Forty-nine studies in our study cohort focused on this perspective when assessing value of medicines. We identified the following clusters: epidemiological endpoints (eg, size of population, 13 studies)14 15 24 26 35 37 40 42 45 46 50 57 58 and clinical endpoints (eg, safety, efficacy or evidence, 45 studies)3 4 12 13 15–25 27–33 35–37 39–42 44–55 57–60 (table 3).…”

“…Multiple studies referred to epidemiological endpoints (for example, incidence or prevalence), as an important aspect for the evaluation of value of medicine (10 studies) 15 24 26 35 37 42 45 46 50 57. Some studies argued that the rarity of a disease increases the value of the medicines targeting those diseases,25 26 35 while others stated that this approach is unethical 24 58.…”

“…Many studies considered outcomes of clinical trials, such as safety or effectiveness as core determinants for the evaluation of value of medicines,3 4 12–20 22–33 35 37 39–42 44–55 58–60 even more so in more recent studies 16 22 24 32. Trial outcomes are influenced by the understanding of the disease,37 the stage of the disease (the benefit of medicines targeting the treatment of advanced-stage diseases may be more modest compared with those applied in a curative setting),3 16 31 38 the duration of the study (some medicines result in a short tumour response and in case of short study duration, the long-term effects may be unclear),16 the size of the targeted population evaluated,26 37 47 50 the study design,24 26 the subjects of the study and the potential resulting heterogeneity in medicine response 11 16 22 27 32 60.…”

Strengthening the Interface of Evidence-Based Decision Making Across European Regulators and Health Technology Assessment Bodies

“…This can contribute to streamlining of decision making and reduction of time-to-market delays. [15][16][17] Thus, the potential of productspecific regulator-HTABs collaboration to enhance access to medicines seems clear. Nevertheless, before understanding the impact of such collaborative activities and potential areas for improvement, evidence requirements and use of evidence of both regulators and HTABs need to be clarified.…”