|
Autism: A Developmental Disorder
That Is Massively on the Rise
Unmet Needs
• No single parameter, or combination of parameters, has
been unambiguously corroborated as a cause of human autistic
disorders.
• No medications have been proven to be efficacious in the
treatment of the core social or communication impairment seen
in autism. Some of its other symptoms or frequent comorbidities
(aggression, hyperactivity, and seizures) can be managed with
currently available drugs.
Market Outlook
If passed into law, the “Combating Autism Act of 2005” could
massively expand the number of autism-related diagnoses within
a few years, creating skyrocketing demand for prescription medications.
This act would amend the Public Health Service Act to require
that all children receive screening for autism before their second
birthday and that children at risk for autism receive services
as early as possible, with referral for intervention to schools
and other agencies. The bill would authorize significant federal
funds annually over the next 5 years and would establish the
Interagency Autism Coordinating Committee to coordinate all efforts
within the Department of Health and Human Services concerning
autism. Notably, 4 years after the Children’s Health Act
was passed in 2000, the Centers for Disease Control revised its
estimate of the prevalence of autism from 1 in 500 to 1 in 166
children in the United States.
Table of Contents
Symptoms, Diagnosis,
and Epidemiology
Etiology
Inconclusive Results of Genetic Investigations
Current Drug Treatments and Trials Involving Drugs Approved
for Other Indications
Anticonvulsants
Selective Serotonin Reuptake Inhibitors
Atypical Antipsychotics
Cholinesterase Inhibitors
Antioxidants and Antiexcitatory Drugs
Potential New Drugs for Autism and Autistic Spectrum
Disorders
Specific Challenges for Drug Development in Autism
Secretin: A Failed Hope
CureMark’s Digestive Enzymes
Oralgam: An Oral Immunoglobulin with Presumed Intestinal Action
RG-2077: Another Potential Immunotherapy
SRX-251
Other Approaches Described in the Patent Literature
The Autism Drug Therapy Market to 2015
A Target Population with a Largely Unpredictable Development
Sales Development To Be Driven by New Drugs
References
Index
Figures and Tables
Figure 1. Key Symptoms of Autism
Figure 2. Incidence of U.S. Autism Cases, 1992 to 2003
Table 1. Drugs Approved for Other Indications That Are Used to Manage or Are
in Clinical Trials for Autism
Table 2. Potential New Drugs for Use in Autism
back to top
Compulsions, Phobias, Panic Attacks: Disruptive Intrusions of
Normal Life
Unmet Needs
• Anxiety disorders affect approximately 19 million American
adults; overall, they represent the single largest cost to the
healthcare system.
• Obsessive-compulsive disorder (OCD), anxiety and panic disorder
(APD), and social anxiety (SAD) can severely interfere with the
daily routines, vocational aspirations, and general quality of
life of those who are affected by them. Nevertheless, they are
believed to be underreported to a significant extent.
• Pharmacologic mechanisms need to be identified that specifically
and reliably address ritualistic behavior in OCD or the breakthrough
panic attacks in APD.
Market Outlook
In the 2005 to 2015 time frame, large increases in the absolute
patient numbers for these disorders are not expected. What
could change is the extent to which these disorders are diagnosed
and treated with drugs other than SSRIs, which will all pass
into the low-priced generic drug realm within our forecast
period. Furthermore, the adolescent segment for these three
anxiety disorders could offer a significant growth opportunity
for the pharmaceutical industry.
Table of Contents
Obsessive-Compulsive Disorder
OCD Etiology
Panic Disorder and Phobias
Current Drug Treatments for OCD, APD, and Social Phobia
Selective Serotonin Reuptake Inhibitors (SSRIs): The Mainstay of Therapy
Other Drug Therapies and Developments Based on Available
Drugs
Antidepressant Drug Augmentation for OCD
Few Acceptable Alternatives to SSRIs in Panic Disorder
Anticonvulsants Used Off-Label for Social Phobia
Drug Candidates in Development
At the Beginning, the Model Systems
Few Promising New Drug Candidates in Development
Market Scenarios for OCD, APD, and Social Phobia
The SSRI Era Has Exhausted Its Potential
New Mechanisms Are Needed to Compete with Psychotherapy
A Market Waiting for One More Boost
References
Index
Figures and Tables
Figure 1. Disturbed Neurotransmitter Pathways in OCD
Figure 2. Projected Combined Market for OCD, APD, and SAD, to 2015
Table 1. Symptoms of OCDs, Panic Attacks, and Phobias
Table 2. SSRIs Currently Used in Treatment of OCD, APD, and SAD
Table 3. Drug Candidates in Development for OCD and Panic Disorders
back to top
Chronic Fatigue Syndrome and Fibromyalgia:
Enigmatic Etiological Models
Unmet Needs
• The National Institute of Arthritis and Musculoskeletal
and Skin Diseases estimates the number of adults in the United
States with fibromyalgia syndrome (FMS) to be 3.7 million, but
other estimates go to 8 million and beyond. The fraction of patients
actually receiving an FMS diagnosis is not known but could exceed
2 million. The Chronic Fatigue and Immune Dysfunction Syndrome
Association of America currently estimates the number of diagnosed
chronic fatigue syndrome (CFS) cases in the United States at 800,000.
• Both conditions lack a consistent and testable model of
disease etiology.
Market Outlook
In contrast to the industry’s ongoing efforts to achieve
approval extensions for drugs that are already on major markets,
the few candidate drugs that currently are in clinical trials
address not only FMS but also CFS. New interdisciplinary approaches
that involve addressing the stress hormone cascades and the immune
system might open new approaches that are based on the interplay
between neurohormonal shifts and pain processing.
Table of Contents
Chronic Fatigue Syndrome and Fibromyalgia
Syndrome: Diagnostic Criteria
Chronic Fatigue Syndrome
Fibromyalgia Syndrome
The Enigma of Etiology
Epidemiology and Economic Implications of Chronic Fatigue Syndrome and Fibromyalgia
Syndrome
Incidence and Prevalence
Economic Sequels
Currently Available Drugs under Development for Fibromyalgia
Syndrome as Additional Indication
Milnacipran (Ixel; Pierre Fabre Medicament/Cypress Biosciences/Forest Laboratories)
Duloxetine (Eli Lilly’s Cymbalta)
Paroxetine (GlaxoSmithKline’s Paxil CR)
Pregabalin (Pfizer’s Lyrica)
Sodium Oxybate (Gamma-Hydroxybutyrate; Orphan Medical’s Xyrem)
Tropisetron (Novartis’ Navoban)
Pharmacotherapeutic Concepts for the Future
Ampligen (Hemispherx Biopharma, Inc.)
Eplivanserin (sanofi-aventis)
AD-337 (Arakis Ltd.)
Androgen Hormone Therapy (Bentley Pharmaceuticals, Inc.)
Scenarios for the Chronic Fatigue Syndrome and Fibromyalgia
Drug Market to 2015
References
Index
Figures and Tables
Figure 1. Location of the “Tender Points” in Fibromyalgia
Figure 2. Projected Growth in Drug Sales for FMS and CFS, to 2015
Table 1. Signs and Symptoms Associated with FMS
Table 2. Marketed Drugs under Study for Label Extension to FMS
Table 3. Pharmacotherapies under Investigation for FMS and CFS
back to top
Eating Disorders: Beyond the Obesity
Debate
Unmet Needs
• The three classic eating disorders—anorexia nervosa,
binge eating disorder, and bulimia nervosa—are considered
to be chronic conditions interrupted only by intermittent periods
of short-lived remission.
• Currently, no medications are approved by the FDA for the
treatment of eating disorders except for fluoxetine, which, at
a dose of 60 mg per day, is approved for bulimia nervosa. Drugs
that can be used to treat eating disorders come in two major categories:
psychotropics, which attempt to address impulse control and obsessions,
and anabolics, which are primarily used for weight gain in anorexia
nervosa.
Market Outlook
Epidemiologic studies indicate that binging disorders have a
prevalence of 1% to 4% in the general population but that they
occur significantly more often in at-risk populations, probably
30% in patients with bipolar II disorder and at least 10% in
persons who are significantly overweight or clinically obese.
Taking these factors into consideration, the target population
could increase to approximately 8.8 million people in the United
States who have (diagnosed or undiagnosed) binge eating disorder,
bulimia nervosa, or related conditions of periodic compulsive
food overconsumption. The theoretical target population could
also expand significantly if the current definition of binging-related
eating disorders is broadened considerably as the notion gains
ground that becoming obese (i.e., having a BMI much over 30)
requires a treatable psychopathology to be present, at least
to some degree. The broadening definition of bipolar disorder
during the past decade provides a precedent.
Table of Contents
Introduction
Anorexia Nervosa
Binge Eating Disorder
Bulimia Nervosa
Assessment Tools for Eating Disorders
Epidemiology and Psychiatric Comorbidity
Etiology of the Eating Disorders
Current Pharmacotherapeutic Approaches
Selective Serotonin Reuptake Inhibitors for Anorexia and Bulimia
Several Drug Classes Seem Effective in Binge Eating Disorder
Atypical Antipsychotics
New Pharmacologic Approaches to Eating Disorders
Ghrelin, the Major Orexigenic “Brain-Gut Peptide”
Neuropeptide Y
Melanin and Melanocortin-Related Agents
Histamine H3 Receptor Ligands
Serotonin Receptor Ligands
Other Mechanisms under Investigation for Eating Disorders
Scenarios for the Developing Eating Disorder Pharmacotherapy
Market
References
Index
Figures and Tables
Figure 1. Synergizing Risk Factors for the Development of Eating Disorders
Figure 2. Schematic Representation of Neuropeptide Circuitries Involved in
Appetite Control and Eating Disorders
Figure 3. Projected Market Size in U.S. Millions of Dollars, to 2015
Table 1. Most Frequently Used Assessment Tools for Eating Disorders
Table 2. Medications Currently Used in Treatment of Eating Disorders
Table 3. Experimental Approaches to the Pharmacotherapy of Eating Disorders
Table 4. Estimated Number of U.S. Individuals Meeting Criteria for Eating Disorders
and Corresponding Market Size, 2005 to 2015
back to top
Restless Legs Syndrome: Much More
Than a Nuisance
Unmet Need
• The restless legs syndrome (RLS) market has only begun to
be created in the past few years, and physicians are still, for
the most part, undereducated about this condition. As a result,
RLS is a heavily underdiagnosed disorder and symptoms are often
misdiagnosed as anxiety disorder.
• Virtually all published population-based studies report
that fewer than 20% of patients with moderate or severe RLS have
sought medical help for their specific symptoms. Probably only
a quarter of these were ever investigated for, let alone appropriately
diagnosed with, RLS.
Market Outlook
The most important factor for development of the pharmacotherapy
market is the extent to which the disorder is being perceived
as a medical problem by those afflicted with it and the degree
to which primary care physicians are aware of RLS and willing
to diagnose it as an entity that is distinct from common sleep
problems or anxiety. Should awareness and acceptance of RLS
increase, the diagnosed treatment-eligible RLS population in
the United States could increase 10-fold during the coming
decade. Because RLS responds well to dopamine agonists (ropinirole
is already approved for RLS), the expansion of the diagnosed
population will almost immediately translate into prescriptions
of dopamine agonists.
Table of Contents
Symptoms and Sequels
Prevalence and Assessment of Severity
Causes
Current and Potential Future Pharmacotherapies
Ropinirole
Pramipexole
Transdermal Rotigotine
Transdermal Liseride
Cabaser (Cabergoline)
Radafaxine
SEP-0226330
Nondopaminergic Candidate Drugs
Novel Opioids: A Potential Field of Discovery
Projections for the RLS Market to 2015
The Treatment Population and Its Projected Development
Population Aging as a Driver
Renal Disease Not a Significant Modifier
RLS Awareness among Patients and Physicians: The Largest Market Driver
Estimation of the Sales Volume Potential
A Scenario for the RLS Drug Market in 2015
References
Index
Figures and Tables
Figure 1. Possible Pathophysiologic Basis of Restless Legs Syndrome (RLS) and
Periodic Limb Movements (PLM)
Figure 2. Recommended Treatment Algorithm for Intermittent, Daily, and Refractory
RLS
Figure 3. RLS-Related Drug Sales (Estimates)
Table 1. Summary of Recently Published Cross-Sectional, Population-Based Restless
Legs Syndrome/Periodic Limb Movement Disorder Epidemiologic Studies
Table 2. Drugs in Development for RLS Indications
Table 3. Estimated Number of U.S. Individuals with Moderate or Severe RLS as
Opposed to the Diagnosed Population, 2005 to 2015
Table 4. Estimated Number of Individuals in the United Kingdom, Germany, France,
Spain, and Italy with Moderate or Severe RLS as Opposed to the Diagnosed Population,
2005 to 2015
back to top
|
