 Spectrum --
June 2008
 Introduction:
The cystic fibrosis (CF) market is poised for significant
change. The next generation of CF treatments all target the central mechanism
responsible for organ damage in CF. These agents, as one thought leader states,
"represent the closest we have come to a cure for CF" and will almost certainly
bring about a change in the treatment algorithm of the disease. As a result,
new market leaders will emerge, and current first-line therapies will find
themselves relegated to second- or third-line options.
Get the Answers You Need to Shape Your Strategy:
 Physicians use a variety of medications and nonpharmacological
treatments to address the needs of CF patients. What are the strengths and
weaknesses of these treatments? Which work best in specific subpopulations? Do thought
leaders believe these treatments will still have a role in the treatment
algorithm over the next five to ten years?
Several drugs with trusted and novel mechanisms of action are
in development for CF. What new agents are in development? What are thought
leaders’ opinions of the potential of these agents in the treatment of CF? What
impact will these agents have on treatment algorithms and the CF market?
Emerging agents target the central mechanism responsible for
organ damage in CF and have the potential to be disease modifying rather than
just treating symptoms. What unmet needs remain in drug development? Will
the formulations of these emerging therapies improve patient compliance? What
more is required to effectively treat the pediatric subpopulation?
Scope:
Overview of CF: disease development, symptoms,
complications, and methods for diagnosis.
Epidemiology: disease definition; incidence by race;
growth in the number of prevalent cases.
Current therapies: mucolytics, antibiotics, pancreatic
enzyme replacement therapy, as well as thought leaders’ opinions on where these
agents fit into the treatment algorithm.
Emerging therapies: strengths and weaknesses of agents
in development; novel mechanisms of action; an analysis of market position for
these agents.
CF market: details on the 2007 market and prescribing
trends.
Outlook: the impact of novel agents on the CF market.
Orphan Disease Series: As competition increases in
larger disease states, companies are turning to orphan diseases that offer
substantial pricing advantages and lower regulatory hurdles because of high
unmet need. The Orphan Disease Series provides critical market
information, including disease prevalence, profiles of marketed and emerging
agents, and insight into the prescribing choices of disease specialists for
diseases with smaller patient populations. These reports will help you to
maximize the potential of your agents in these challenging markets, including
the regulatory benefits available and the potential for partnership with
charitable organizations.
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