The payer’s perspective: What is the burden of MS and how should the patient’s perspective be integrated in health technology assessment conducted for taking decisions on access to care and treatment?

Abstract

Background: In Europe, there exists considerable variability in access to care and treatment for multiple sclerosis (MS). Objectives: To improve this situation, we identified key issues payers should take into account when making decisions on access to care and treatment for MS. We also give an overview of the different dimensions determining total MS burden and discuss why it is key to integrate the patient's perspective in estimating this burden. Results: The total burden of MS relates to three dimensions: clinical, humanistic and economic. Although the clinical burden is extensively studied, crucial information is still missing about MS pathophysiology, how MS-related symptoms will develop during the disease course and which patients will progress more rapidly. With regard to the humanistic burden, information on patient-reported quality of life systematically collected in clinical trials for registration purposes is still scarce. Early engagement between pharmaceutical companies, the European Medicines Agency and health technology agencies to prospectively identify key evidence needs for the regulatory and reimbursement processes is required as a first step towards more equal access to care and treatment in MS in Europe. Patients' expectations regarding treatment outcomes should be better researched and integrated into decision-making and patients should be counselled in this process.

Conclusions: Calls to action for improving the life of MS patients and their families

“…9 Moreover, whereas IHD and NIDDM usually affect older people, MS is a disease of young people which peaks around the age of 30 years when they are at the top of their productive life. 9 Therefore, MS may have a greater negative impact on productivity loss and unemployment rate than IHD or NIDDM. Moreover, as MS progresses, patients become increasingly dependent of their family, who may also have to take a part-time job or stop work completely.…”

“…9 With the introduction of several new disease-modifying drugs (DMDs) over the last years, the urgent need for a large, pan-European cost-of-illness study/model adequately evaluating not only direct (drug) costs but also indirect costs related to MS management was identified at the first Colloquium. 5,9 It was discussed at the second Colloquium that this call has been taken up by the EMSP who is currently overseeing the execution of such a study in around 16 European countries involving thousands of patients. 9 Additional support/funding by the European Commission (EC) may still be required.…”

“…5,9 It was discussed at the second Colloquium that this call has been taken up by the EMSP who is currently overseeing the execution of such a study in around 16 European countries involving thousands of patients. 9 Additional support/funding by the European Commission (EC) may still be required. Moreover, when during HTA the long-term cost-effectiveness of MS treatment is assessed and payers subsequently decide on access to treatment, it should be factored in that treatments which reduce disability progression (as recently demonstrated for interferon-β (INF) and glatiramer acetate (GA)) will keep patients with MS and their caregivers longer at work.…”

“…Moreover, as the HTA process is largely driven by the (long-term) cost-effectiveness of new treatment and it will take some time before the results of the large, cross-European cost-of-illness study/model (which includes all recently introduced treatments) will be available, this can lead to different decisions on treatment access in the European countries. 9 The EC is funding since 2010 a more closely working together of the CHMP/EMA and the European Network for HTA (EUnetHTA). 9 Nevertheless, the marketing authorisation process and HTA process are not fully aligned yet and, influenced by national economy and political considerations, this can still result in delayed access and/or inequalities in access across Europe.…”

Adaptation and Validation of a Spanish version of the Treatment Burden Questionnaire in Patients with Multiple Sclerosis

“…In addition, cognitive impairment and fatigue are present in a significant number of subjects. All of these health issues make disease burden very high in MS (5)(6)(7). Most patients require multiple clinic visits and treatments -pharmacological, physical therapy, cognitive neurorehabilitation -to manage the disease, avoid disability and maintain their quality of life.…”