LONDON, Nov. 26, 2015 /PRNewswire/ --
INTRODUCTION
The service oriented and dynamic nature of the healthcare industry
has led to the discovery of a plethora of treatment options for a
wide range of ailments. However, there are certain diseases, termed
as rare, that continue to remain untreated and neglected. Rare /
orphan diseases, as the name suggests, are those diseases which are
relatively infrequent in occurrence. Many orphan diseases are
life-threatening and progressive in nature. Absence of potential
treatment regimes catering to these diseases is a big unmet need in
the market.
The prevalence and adversity of these ailments has gained
significant attention in the global healthcare industry.
Stakeholders have realized the necessity and importance of urgently
addressing the widening concerns and the need for discovering
treatment options for several rare diseases. There is a tremendous
scope offered by the innovation driven research being executed by
various organizations in an effort to bridge gaps in the current
market landscape.
The enactment of the Orphan Drugs Act in the US and EU emerged as
ground breaking and provided the necessary support to guide
research focused on rare diseases worldwide. The orphan designated
drugs are granted important incentives which include market
exclusivity and fees reductions. This regulatory assistance has
provided a strong impetus to the pharmaceutical companies and led
to their active participation in the market. Collaborative
agreements and partnerships, amongst the pharmaceutical firms and
other active organizations (such as EURORDIS, NORD and Global
Genes), are also playing a key role in addressing the needs of this
market.
The wide gap between the demand and supply of drugs is likely to
provide a major boost to the market in the coming decade. Several
drugs for various rare diseases are currently under development
and, once approved, are likely to be readily accepted by the
untreated patient population.
SCOPE OF THE REPORT
The 'Rare Diseases: The Pharmaceutical Landscape and Opportunity
Areas' report is a comprehensive study of the market of drugs being
developed by the top pharmaceutical companies for rare diseases.
The focus of this study is primarily to uncover several initiatives
currently underway to harness the potential presented in different
therapeutic areas. One of the basic building blocks for analysing
the market was to prepare an extensive pipeline of drugs for rare
diseases. This effort encompassed reviewing several databases,
company websites and other information available in the public
domain.
We confined the focus of our study to 50 large pharmaceutical
companies which invest a proportion of their R&D spend on drugs
for rare diseases. These pharmaceutical companies were profiled to
get an in-depth view of the work being done by them in this domain.
The profiles include a general overview of the firms, financial
performance over the last five to six years, and the current
marketed and clinical pipeline products specific to rare
diseases.
With a robust pipeline in place, we then analysed where the
industry's current vision lies and highlighted a number of efforts
which have been taken by pharmaceutical companies, healthcare
organizations and other industry stakeholders. Specifically, our
market potential analysis outlines key patient demographics and
underscores potential areas anticipated to emerge as major revenue
generators for the pharmaceutical companies. In addition to the
above, we have also provided our own independent perspective on the
key drivers behind the growth of this market, and the associated
challenges.
RESEARCH METHODOLOGY
Most of the data presented in this report has been gathered via
secondary and primary research. For most of our projects, we
conduct interviews with experts in the area (academia, industry,
medical practice and other associations) to solicit their opinions
on emerging trends in the market. This is primarily useful for us
to draw out our own opinion on how the market will evolve across
different regions and technology segments. Where possible, the
available data has been checked for accuracy from multiple sources
of information.
The secondary sources of information include
- Annual reports
- Investor presentations
- SEC filings
- Industry databases
- News releases from company websites
- Government policy documents
- Industry analysts' views
CHAPTER OUTLINES
Chapter 2 provides a general introduction to rare diseases. We have
highlighted the indispensable role played by several regulatory
organizations in accelerating this market. The chapter also
elaborates upon the various drivers responsible for the growth in
this market.
Chapter 3 provides a comprehensive market landscape of drugs in
development for rare diseases. This chapter includes an elaborate
analysis of pipeline drugs which are currently in different stages
of development. The detailed analysis includes information on the
targeted indications, types of molecules and the current phases of
development. The chapter also provides the historic ladder for the
orphan drug designations conferred to several drugs in the US and
the European markets.
Chapter 4 offers an insightful discussion of six most popular
indications targeted by the drugs under development. This analysis
revolves around current landscape, prevailing competition,
anticipated shift in the market trend and the key focus for the
future.
Chapter 5 provides detailed company profiles of the top
pharmaceutical companies focusing in the field of rare diseases.
Each company profile includes information such as financial
performance, geographical presence, marketed / pipeline drugs,
recent collaborations specific to rare diseases and the initiatives
taken by the firm towards the treatment of rare diseases.
Chapter 6 summarises the overall report. In this chapter, we have
provided a recap of the key takeaways and our overall opinion on
where the market is headed in the mid-long term.
Chapter 7 provides the list of companies covered in this
report.
EXAMPLE HIGHLIGHTS
1. With over 7000 rare diseases reported in the world, lack of
awareness, dispersed target population and inefficient reporting of
these diseases limits the pace of development in this industry.
However, the industry-academia coalition has helped in filling some
of these gaps. An active participation by pharma firms through
various initiatives, such as the Rare Disease Day, has helped to
address the existing lacunae in the demand supply gap for these
drugs.
2. The market is characterized by the presence of several pipeline
drugs under development for the treatment of rare diseases. Of
these, 30% drugs are in phase III of clinical development whereas
38% of drugs are under development in phase II clinical trials.
Phase I drugs account for 20% to the total market.
3. Companies such as Roche, BMS, Sanofi, GSK, Boehringer Ingelheim,
Celgene, Novartis and Pfizer are some of the key players in the
market. However, the market remains opportunistic for newer players
to step in and benefit from the vast potential in different
therapeutic areas.
4. With a share of more than 50%, oncology remains the key focus
area of pharmaceutical firms. Blood associated diseases and neural
disorders are some other well-known areas being targeted.
5. On an average, 13-14 drugs are being developed by each
pharmaceutical company covered in our analysis. However, there is a
stark variation amongst the companies. Some of the bigger pharma
companies, such as Roche and BMS, have a much wider portfolio.
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