In recent years, there has been growing recognition of the importance of rare diseases as a global health priority, leading to increased research efforts, policy initiatives, and collaborative partnerships aimed at accelerating progress in this field.
Key drivers of innovation in the rare disease space include advances in genomics, molecular diagnostics, precision medicine, and therapeutic development, which hold promise for personalized approaches to diagnosis and treatment. Furthermore, the rare disease community has mobilized to advocate for greater awareness, resources, and support for individuals living with rare diseases and their families. Through grassroots activism, patient advocacy organizations, and social media campaigns, stakeholders in the rare disease space strive to amplify the voices of affected individuals, promote equitable access to care, and drive policy reforms that address the unique needs of this population.
This report consolidates the analyst’s latest thinking and forecasts around how the healthcare, macroeconomic, technology, and regulatory trends will impact the rare disease space, as well as providing insights into the leading players and future disruptors across the value chain, and providing insights into key drugs and markets from the analyst’s Pharma Intelligence Center. Additionally, this report is designed to provide strategic planners, competitive intelligence professionals and key stakeholders in the pharmaceutical industry a clear view of the opportunities and risks over the foreseeable future for rare disease.
Key drivers of innovation in the rare disease space include advances in genomics, molecular diagnostics, precision medicine, and therapeutic development, which hold promise for personalized approaches to diagnosis and treatment. Furthermore, the rare disease community has mobilized to advocate for greater awareness, resources, and support for individuals living with rare diseases and their families. Through grassroots activism, patient advocacy organizations, and social media campaigns, stakeholders in the rare disease space strive to amplify the voices of affected individuals, promote equitable access to care, and drive policy reforms that address the unique needs of this population.
This report consolidates the analyst’s latest thinking and forecasts around how the healthcare, macroeconomic, technology, and regulatory trends will impact the rare disease space, as well as providing insights into the leading players and future disruptors across the value chain, and providing insights into key drugs and markets from the analyst’s Pharma Intelligence Center. Additionally, this report is designed to provide strategic planners, competitive intelligence professionals and key stakeholders in the pharmaceutical industry a clear view of the opportunities and risks over the foreseeable future for rare disease.
Scope
- A focused chapter on the key healthcare, technology, macroeconomic, and regulatory trends impacting rare disease, and a review of the ways in which these trends will accelerate or hinder the rare disease market.
Reasons to Buy
- Understand the key trends accelerating or hindering the rare disease space.
- See market forecasts for different therapies within rare disease up to 2027.
- Understand recent and influential developments in rare disease.
- Review of leaders and disruptors across the rare disease value chain.
Table of Contents
- Players
- Thematic Briefing
- Trends
- Industry Analysis
- Value Chain
- Companies
- Drug Development Scorecard
- Abbreviations
- Further Reading
- About the Authors
- Thematic Research Methodology
Table 1: Healthcare trends impacting rare disease
Table 2: Technology trends impacting rare disease
Table 3: Macroeconomic trends impacting rare disease
Table 4: Regulatory trends impacting rare disease
Table 5: Recent strategic partnerships in rare disease
Table 6: Recent funding deals in rare disease
Table 7: Recent M&As in rare disease
Table 8: Examples of public companies in rare diseases
Table 9: Examples of private companies in rare diseases
Table 10: Abbreviations
Table 11: the analyst reports
List of Figures
Figure 1: Examples of players in rare disease and where they sit in the value chain
Figure 2: Global patient-based forecast for rare disease drugs
Figure 3:Global patient-based forecast for rare disease by highest generating drugs and indications
Figure 4: Social media discussions related to rare diseases fluctuating since 2020
Figure 5: Word cloud with trends related to rare disease social media discussions
Figure 6: Examples of popular posts related to rare disease
Figure 7: The rare disease value chain
Figure 8: Examples of drug discovery and development providers in rare disease
Figure 9: Examples of clinical trial providers in rare disease
Figure 10: Examples of manufacturing providers in rare disease
Figure 11: Examples of patient access providers in rare disease
Figure 12: Who does what in the drug development space?
Figure 13: The thematic screen ranks companies based on overall leadership in the 10 themes that matter most to their industry, generating a leading indicator of future performance.
Figure 14: Our valuation screen ranks our universe of companies within a sector based on selected valuation metrics.
Figure 15: Our risk screen ranks companies within a particular sector based on overall investment risk
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- Vertex
- Roche
- Pfizer
- AstraZeneca
- Novartis
- BMS
- GSK
- IQVIA
- Lonza Group
- Catalent
- Thermo Fisher
- Sanofi,BeiGene
- Regeneron
- CSL
- Argenx
- Abzena
- Curebase
- Raremark
