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市場調査レポート
希少疾病：筋萎縮性側索硬化症（ALS）
ORPHAN DISEASE SERIES - Amyotrophic Lateral Sclerosis: Strategies for Drug Developers
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Abstract
Introduction
Only one drug is approved for the treatment of ALS, and its only benefi t 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 suffi cient effi cacy and safety data for emerging ALS
treatments, and those requirements pose fi nancial 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 fi nancial, marketing, and drug-approval benefi ts-signals
substantial opportunity for a company that develops an effi cacious 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 effi cacy 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 fi nancial 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.
- Benefi ts 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, profi les 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 benefi ts
available and the potential for partnership with charitable organizations.
Table of Contents
- Executive Summary
- Strategic Considerations
- Stakeholder Implications
- Introduction
- Overview of ALS
- The Nervous System
- Familial vs. Sporadic ALS
- Symptoms, Diagnosis, and Disease Progression
- Epidemiology
- Current Treatments
- Sanofi -Aventis' s Rilutek
- Symptomatic Treatments
- Anti-Drooling Medications
- Anti-Anxiety Drugs and Antidepressants
- Muscle and Neuropathic Pain Medication
- Insomnia Medication
- Emerging Therapies
- Antiglutamatergic Agents
- Roche' s Rocephin
- Teva' s Copaxone
- AMPA Receptor Antagonists
- Potential of Antiglutamatergic Agents in ALS
- Induction of Heat Shock Proteins
- CytRx' s Arimoclomol
- Potential of Heat Shock Proteins in ALS
- Antioxidants
- Co-enzyme Q-10
- Aeolus Pharmaceuticals' AEOL-10150
- Potential of Antioxidants in ALS
- Neurotrophic Factors
- Insulin-Like Growth Factor-1
- Potential of Neurotrophic Factors in ALS
- Lithium
- Emerging Trends in ALS Treatment
- Multidisciplinary Clinics
- Noninvasive Positive Pressure Ventilation
- Stem Cell Therapy
- Outlook for ALS: Considerations for Drug Developers
- Appendix A. Incentive Programs for the Research, Development, and
Marketing of Orphan Drugs
- FDA
- Expanded Market Exclusivity
- Tax Credits
- Support During the Drug Approval Process
- Grants
- EMEA
- Private Organization Incentives
- Appendix B. Epidemiology Methods
- Appendix C. Bibliography
- Appendix D. Thought Leaders Interviewed for This Report-Amyotrophic
Laterial Sclerosis
Tables
- ES1. Thought Leaders' Opinions of ALS Issues
- 1. Proposed Theories for the Cause of Sporadic ALS
- 2. The World Federation of Neurology Research Committee on Motor Neuron
Diseases Guidelines for ALS Diagnosis
- 3. Commonly Used Drugs to Treat ALS Patients
- 4. Current Clinical Trials of Emerging Therapies to Treat ALS Patients
- B1. Number of Diagnosed Prevalent Cases of Amyotrophic Lateral Sclerosis
in the United States, 2007-2017
- B2. Key Sources for Epidemiology Estimates, Amyotrophic Lateral Sclerosis
Figures
- 1. The Nervous System
- 2. Diagram of a Neuron
- 3. Age and Gender Distribution of Diagnosed ALS Prevalent Cases in the
United States, 2007
- 4. Glutamate Excitotoxicity
- 5. Number of Emerging Therapies, By Mechanism of Action, for Amyotrophic
Lateral Sclerosis
- 6. Multidisciplinary Approach to ALS Treatment
- 7. Worldwide Sales and Sales Growth of Rilutek, 1995-2007
- A1. Number of Drugs Receiving FDA Orphan-Drug Designation and Approval,
1983-2007
- A2. Growth in the Number of Orphan Drugs Available, 1983-2007
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