 Spectrum --
May 2008
 Introduction:
Only one drug is approved for the treatment of ALS, and its
only benefit is a modest increase in survival time. Commercial development in
this disease has been minimal, with companies being dissuaded by the limited
understanding of the mechanisms of the disease and by the small patient
population. In addition, thought leaders interviewed by Decision Resources
caution that recent failures of promising agents have re-emphasized the need
for large trials to provide sufficient efficacy and safety data for emerging
ALS treatments, and those requirements pose financial and operational
challenges for companies choosing to enter this market. Nonetheless, the high
unmet need in the treatment of ALS, plus the disease’s orphan status—which
provides numerous financial, marketing, and drug-approval benefits—signals
substantial opportunity for a company that develops an efficacious agent to
treat this disease.
Get the Answers You Need to Shape Your Strategy:
 ALS is a debilitating and fatal disease with limited treatment
options. What are the current treatment options for ALS? What considerations
drive neurologists’ prescribing choices? What do thought leaders identify as
the primary goals of ALS treatment?
A number of drugs, with a variety of mechanisms of action, are
being investigated for efficacy in ALS. What new agents are in development?
What drugs that are approved for other indications are being studied for use in
ALS? What are thought leaders’ opinions of the potential of these agents in the
treatment of ALS?
The antibiotic minocycline, which showed promise in
small-scale trials, was shown in a large clinical trial to be detrimental to
ALS patients. What does the failure of minocycline mean for other emerging
agents? What data do neurologists require before they will prescribe an
emerging agent? What level of clinical data is necessary for an emerging agent,
and what should companies expect when designing a clinical trial in ALS
patients?
The Orphan Drug Act of 1983 paved the way for the development
and approval of numerous orphan drugs. How does the U.S. government
encourage the pharmaceutical industry to investigate treatments that qualify
for orphan status? What other organizations are willing to provide financial
incentives to drug companies?
Scope:
Thought-leader opinions: Neurological experts give
their opinions on the status of ALS treatment and explain their expectations
for the potential of emerging treatments for the disease.
Overview of ALS: The nervous system; familial vs.
sporadic ALS; symptoms and disease progression.
Epidemiology: Prevalent cases over a ten-year forecast
period; age and gender trends.
Current therapies: Rilutek; anti-drooling medications;
anti-anxiety drugs and antidepressants; pain medication; insomnia medications.
Emerging therapies: antiglutamatergic agents; induction
of heat shock proteins; antioxidants; neurotrophic factors.
Emerging trends in ALS treatments: multidisciplinary
clinics; noninvasive positive pressure ventilation; stem cell therapy.
Considerations for drug developers: the success of
Rilutek; advantages of developing drugs for ALS; challenges in designing and
running clinical trials.
Benefits of orphan-drug status: Financial and marketing
incentives associated with orphan-drug status.
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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