ACCESS AND EQUITY

PROMOTE UNIVERSAL HEALTH COVERAGE. LEARN MORE ABOUT ORPHAN DRUG POLICIES, ESSENTIAL MEDICINES AND INNOVATIVE ACCESS MODELS.

INTEGRATED CARE AND UNIVERSAL HEALTH COVERAGE

COURSES, FACTSHEETS AND ARTICLES ON INTEGRATED CARE AND UNIVERSAL HEALTH COVERAGE FOR RARE DISEASES.

SOURCE	TITLE	DESCRIPTION
Rare Diseases International (RDI)	Common Needs of the Rare Disease Population Identified Globally	RDI report on the common needs of Persons Living with a Rare Disease. Follows consultation with rare disease patients organisations worldwide.
Rare Diseases International (RDI)	RDI Fact - Universal Health Coverage for Rare Diseases (English, French, Spanish, Arabic)	Factsheet to support advocacy for Persons Living with a Rare Disease through the framework of Universal Coverage.
World Health Organization (WHO)	E-Learning Course on Health Financing Policy for Universal Health Coverage (English, French)	E-learning course comprises six modules which cover the core functions of health financial policy as conceptualised by WHO.
The World Bank	E-Learning Course on Advocacy for Universal Health Coverage	Course on universal health coverage advocacy and the essence of the UHC2030 Advocacy Guide.
Health Policy (Henrike Hannemann-Weber, Maura Kessel, Carsten Schultz)	Research performance of centers of expertise for rare diseases—The influence of network integration, internal resource access and operational experience	Explores the relationship between a Centers of Expertise's internal resource availability, operational experience and functional network integration and its research performance.
Rare Disease and Orphan Drugs Journal (Bolz-Johnson)	Enhancing the value of clinical networks for rare diseases	Explores of the core themes of effective clinical networks around the world.
Prepared by Iqvia	Innovative Funding Models for Treatment of Rare Diseases: A global study of new financing solutions for rare diseases	A global study of new financing models for rare diseases.

DIAGNOSIS

ARTICLES, REVIEWS AND INFOGRAPHICS ON ACCESS TO DIAGNOSIS FOR PERSONS LIVING WITH A RARE DISEASE.

SOURCE	TITLE	DESCRIPTION
World Health Organization	WHO Science Council Workshop Series: Accelerating access to genomic technologies for global health	WHO hosted workshop series on genomic technologies for global health
European Joint Programme on Rare Diseases (EJP RD)	Diagnosing Rare Diseases: from the Clinic to Research and back	E-Learning course. Discover the role of research, clinical investigation and data sharing in diagnosing rare diseases.
National Institutes of Health and Undiagnosed Diseases Network International (UDNI)	Undiagnosed Diseases Network International (UDNI): White paper for global actions to meet patient needs	White paper for global actions to meet the needs of undiagnosed persons worldwide.
Global Commission to End the Diagnostic Odyssey for Children with a Rare Disease	Progress Update - Rare Disease Day 2021	Progress report on the Global Commission pilot projects to advance timely, accurate diagnosis for children living with a rare disease.
National Institutes of Health (Baynam et al.)	Barriers and Considerations for Diagnosing Rare Diseases in Indigenous Populations	Analysis of barriers to accessing genetic services, participating in genomic research, and other considerations impacting the diagnosis of indigenous populations.
American Society for Human Genetics (Boycott et al)	International Cooperation to Enable the Diagnosis of All Rare Genetic Diseases	A review of the current and future bottlenecks to gene discovery and strategies to enable progress.

INFOGRAPHICS

ACCESS AND ORPHAN DRUG POLICIES

ARTICLES, REVIEWS AND WEBINARS ON REGIONAL ORPHAN DRUG POLICIES AND INNOVATIVE ACCESS MODELS.

SOURCE	TITLE	DESCRIPTION
Health Policy Analysis (Chan et al.)	Access and Unmet Needs of Orphan Drugs in 194 Countries and 6 Areas: A Global Policy Review With Content Analysis	An overview of global policies that optimize development, licensing, pricing, and reimbursement of orphan drugs.
PLOS One (Gamme et al.)	Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries	Review of existing regulations and policies utilised by countries to enable patient access to orphan drugs.
Frontier in Public Health (Czech et al.)	A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries	A literature review with five focus areas: rare disease definition, newborn screening, registries, national plans, access to/reimbursement of orphan medicinal products.
Frontier in Medicine (Stevens and Huys)	Innovative Approaches to Increase Access to Medicines in Developing Countries	An overview of innovative approaches to address health challenges in developing countries with a focus on business models for healthcare delivery.

ESSENTIAL MEDICINES FOR RARE DISEASES

WEBINAR AND ARTICLES ON THE WHO MODEL LISTS OF ESSENTIAL MEDICINES AND OTHER ESSENTIAL LISTS FOR RARE DISEASES.

SOURCE	TITLE	DESCRIPTION
Rare Diseases International (RDI)	Working with WHO to improve access to rare disease medicinal products	Webinar on the WHO Model Lists of Essential Medicines. Includes a case study from the International Patient Organisation for Primary Immunodeficiencies (IPOPI).
World Health Organization (WHO)	WHO Model Lists of Essential Medicines	Platform for the WHO Model Lists of Essential Medicines.
International Rare Diseases Research Consortium (IRDiRC)	Essential list of medicinal products for rare diseases: Recommendations from the IRDiRC Rare Disease Treatment Access Working Group	List of essential medicinal products for rare diseases developed by IRDiRC. Intended to initiate discussion and collaboration to enhance access.
Bulletin of the World Health Organization (Stolk et al.)	"Rare Essential": Drugs for rare diseases as essential medicines	Commentary advocating for a complimentary WHO Orphan Medicines Model List.