Amyotrophic Lateral Sclerosis: Strategies for Drug Developers

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Published Date: May 2008
Published By: Decision Resources
Page Count: 49
Order Code: R55-584
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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 benefi ts—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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