Equitable Access to High-Cost Pharmaceuticals

Zaheer-Ud-Din Babar

• Publisher: Academic Press
• Year: 2018
• Print ISBN: 9780128119457
• eBook ISBN: 9780128119624
• Topic: Medicine
• Subtopic: Pharmaceutical, Biotechnology & Healthcare Industry

Equitable Access to High-Cost Pharmaceuticals seeks to aid the development and implementation of equitable public health policies by pharmaco-economics professionals, health economists, and policymakers. With detailed country-by country analysis of policy and regulation, the Work compares and contrasts national healthcare systems to support researchers and practitioners identify optimal healthcare policy solutions. The Work incorporates chapters on global regulatory changes, health technology assessment guidelines, and competitive effectiveness research recommendations from international bodies such as the OECD or the EU. Novel policies such as horizon scanning, managed-entry agreement and post-launch monitoring are considered in detail. The Work also thoroughly reviews novel pharmaceuticals with particular research interest, including cancer drugs, orphan medicines, Hep C, and personalized medicines. - Evaluates impact and efficacy of current access policies and pricing regulation of high-cost drugs - Incorporates existing guidelines and recommendations by international organizations - Compares and contrasts how different countries fund and police high-cost drug access - Explores novel and emergent policies, including managed entry agreement, analysis of real world data and differential pricing - Reviews novel pharmaceuticals of current research interest

Chapter 1

Access to High Cost Medicines: An Overview

Syed Shahzad Hasan^*

Christine Y. Lu^**

Zaheer-Ud-Din Babar^*†
* University of Huddersfield, Queensgate, Huddersfield, United Kingdom
^** Harvard Medical School and Harvard Pilgrim Health Care Institute, Boston, MA, USA
^† University of Auckland, Auckland, New Zealand

Abstract

High-cost medicines (HCMs) have been largely defined based on monetary value because of their potential to increase pharmaceutical spending for either the individual patient or healthcare systems. One of the biggest challenges encountered by many healthcare systems around the globe is to provide equitable access to HCMs while maintaining the sustainability of systems that are already constrained in resources. The global medicine spending is expected to increase with growing use of HCMs for an increasing number of diseases. Many newly developed medicines that offer therapeutic advantages for a range of diseases are reported to be efficacious, but are expensive for healthcare systems and/or patients compared to traditional medicines. Country’s wealth and/or affordability of the healthcare system has a direct relationship with patients’ access to HCMs. The developed markets are better resourced than developing countries and therefore are more likely to meet the demands for access to and use of HCMs. Nevertheless, all countries are facing the challenges of rationing and prioritising access to HCMs. Disproportional access to HCMs, particularly in nations where patient out-of-pocket costs largely support their treatment, might be particularly concerning.

Keywords

high cost medicines

overview

biologics

targeted therapies

access to medicines

Abbreviations

CDF Cancer Drugs Fund

DMARDs Disease modifying anti-rheumatic drugs

HCMs High-cost medicines

HTA Health technology assessment

NICE National Institute of Clinical Excellence

MEAs Managed entry agreements

MOA Mechanisms of action

QALY Quality-adjusted life years

SMART-D State Medicaid Alternative Reimbursement and Purchasing Test for High-Cost Drugs

UK United Kingdom

US United States

VBP Value-based pricing

WHO World Health Organisation

1.1. Defining HCMs

Date back to 1990s, in a time where only little attention was paid to high-cost medicines (HCMs), several groups of researchers have commenced to conceptualise ‘high-cost’ or ‘high-priced’ medicines. Although some referred to certain medicines as being ‘high-cost’ without pre-specified criteria, others provided the price point at which a medicine can be defined as being ‘high-cost’. For instance, Powe etal. (1994) considered cyclosporine and erythropoietin as HCMs. The cost of treatment with these medicines in 1994 was US $4000–US $6000 per patient per year in the United States (Powe etal.,1994). In 1995, Poirier etal. defined foscarnet, monoclonal antibodies for septic shock treatment, and granulocyte colony-stimulating factors, as HCMs with no reference being made to the cost of these medicines (Poirier and Gianetti,1995). In that same year, Crump etal. (1995) defined ‘expensive treatments’ as those costing more than £2000 per patient per year.

The more contemporary definitions of ‘high-cost’ or ‘high-priced’ medicines almost exclusively provided the cut-off price point beyond which a medicine would be classified as ‘high-cost’ or ‘high-priced’. This is illustrated in a 2015 report by the World Health Organisation (WHO) Regional Office for Europe which defines a medicine as high-priced if the therapy exceeds €10,000 per patient per year to be reimbursed by a public payer (WHO,2015). This definition is limited to European countries which use Euro as their currency. On the other hand, in British Columbia, Canada, the minister may designate a medicine as a HCM if the minister determines that the maximum daily amount the minister may pay for the medicine when averaged over a 1-year period is equal to or greater than Can $40 (for the typical prescribed dose or the typical number of doses per year), as stipulated in the Drug Price Regulation of Pharmaceutical Services Act of British Columbia (British Columbia,2017). While in New Zealand, McCormack etal. (2013) acknowledged that there is no precise definition for New Zealand and considered HCMs to be more than NZ $20,000 per patient per year in their report to Minister of Health of New Zealand.

Definitions could also vary within the same country according to who defines it and how they are defined. This is exemplified in both the United States and Australia. In the United States, while the Office of Inspector General of the Department of Health & Human Services considered HCMs as those with an average price of more than US $1000 per month, the researchers at State Medicaid Alternative Reimbursement and Purchasing Test for High-Cost Drugs (SMART-...

Table of contents

• Cover
• Title page
• Table of Contents
• Copyright
• Dedication
• Contributors
• Foreword
• Chapter 1: Access to High Cost Medicines: An Overview
• Chapter 2: Health Technology Assessment
• Chapter 3: High-Cost Medicines in England
• Chapter 4: Managed Entry Agreements and High Cost Medicines (European Perspective)
• Chapter 5: Setting a Public Health Agenda to Support Access to High-Cost Medicines
• Chapter 6: Human Rights and Intellectual Property for Universal Access to New Essential Medicines
• Chapter 7: Access to High Price Medicines in Portugal
• Chapter 8: Improving Access to High-Cost Medicines in Low Income Countries in Africa: Creating a Functioning Pharmaceutical System in Uganda
• Chapter 9: Equitable Access to Biosimilars: An Overview
• Chapter 10: Access to High-Cost Medicines in Europe
• Chapter 11: Access to High-Cost Medicines in Australia
• Chapter 12: Access to High-Cost Medicines in New Zealand
• Chapter 13: Access to High-Cost Medicines in Wales
• Index