America’s biopharmaceutical research compa nies are currently developing 36 medicines to help the nearly 1 million Americans suffering from Parkinson’s disease, a motor system disorder resulting from the loss of dopamine producing brain cells. All of the medicines are either in clinical trials or awaiting review by the U.S. Food and Drug Administration. Each year, approximately 60,000 Americans are diagnosed with Parkinson’s disease, and incidence increases with age. The combined cost to the U.S. economy in direct and indirect expenses is nearly $25 billion a year, accord ing to the Parkinson’s Disease Foundation. The medicines today in the research and development pipeline offer hope of reducing the human and economic costs of Parkinson’s disease. They include: *HQH WKHUDSLHV WKDW WDUJHW VSHFL¿F DUHDV in the brain. &HOO WKHUDS\ WKDW XVHV D SDWLHQW¶V RZQ cells to reverse effects of the disease. 1HZ GHOLYHU\ PHFKDQLVPV RI FXUUHQWO\ approved treatments, including a transdermal patch and an intranasal formulation. 0HGLFLQHV WR WUHDW D PRWRU IXQFWLRQ disorder associated with Parkinson’s disease treatment. Researching and developing new medicines remains a risky investment and lengthy process—costing, on average, $1.2 billion, including the cost of failures, and taking between 10–15 years to bring a new medicine to patients. But advances in our understand ing of diseases and how to treat them have allowed America’s biopharmaceutical research companies to conduct the cuttingedge research needed to reduce the destructive toll of Parkinson’s disease and to allow more patients to lead healthier, happier, more productive lives. More Than 30 Medicines Are Being Developed to Treat Parkinson’s Disease and Related Conditions Medicines in Development PARKINSON’ S DISEASE presented by america’s biopharmaceutical research companies 2011 REPORT Diagnosis 26 2 13 Related Conditions Parkinson’s Disease Medicines in Development for Parkinson’s Disease* An estimated 1 million Americans suffer from the disease, with 60,000 newly diagnosed each year * Some medicines are listed in more than one category.
Transcript
America’s biopharmaceutical research compa-nies are currently developing 36 medicines to help the nearly 1 million Americans suffering from Parkinson’s disease, a motor system disorder resulting from the loss of dopamine-producing brain cells. All of the medicines are either in clinical trials or awaiting review by the U.S. Food and Drug Administration.
Each year, approximately 60,000 Americans are diagnosed with Parkinson’s disease, and incidence increases with age. The combined cost to the U.S. economy in direct and indirect expenses is nearly $25 billion a year, accord-ing to the Parkinson’s Disease Foundation.
The medicines today in the research and development pipeline offer hope of reducing the human and economic costs of Parkinson’s disease. They include:
in the brain.
cells to reverse effects of the disease.
approved treatments, including a transdermal patch and an intranasal formulation.
disorder associated with Parkinson’s disease treatment.
Researching and developing new medicines remains a risky investment and lengthy process—costing, on average, $1.2 billion, including the cost of failures, and taking between 10–15 years to bring a new medicine to patients. But advances in our understand-ing of diseases and how to treat them have allowed America’s biopharmaceutical research companies to conduct the cutting-edge research needed to reduce the destructive toll of Parkinson’s disease and to allow more patients to lead healthier, happier, more productive lives.
More Than 30 Medicines Are Being Developed to Treat Parkinson’s Disease and Related Conditions
Medicines in Development
PARKINSON’S DISEASE
presented by america’s biopharmaceutical research companies
2011 R
EP
OR
T
Diag
nosis
26
2
13
Rela
ted
Cond
itions
Park
inso
n’s
Dise
ase
Medicines in Development
for Parkinson’s Disease*
An estimated 1 million Americans suffer from the disease, with 60,000 newly diagnosed each year
* Some medicines are listed in more than one category.
Medicines in Development Parkinson’s Disease 20112
Medicines in Development for Parkinson’s Disease
PARKINSON’S DISEASE
Product Name Sponsor Indication Development Status*
nitisinone Biotie TherapiesSouth San Francisco, CA
Parkinson’s disease Phase II(650) 244-4850
Fort Lee, NJParkinson’s disease(Fast Track)
Phase II(866) 604-8665
OS-320 Osmotica PharmaceuticalWilmington, NC
Parkinson’s disease Phase II completed(910) 509-0114
Parkinson’s disease gene therapy San Diego, CA
Oxford, United Kingdom
Parkinson’s disease Phase I/II(858) 677-6500
Parkinson’s diseasestem cell therapy San Diego, CA
Parkinson’s disease in clinical trials(858) 658-0910
preladenantWhitehouse Station, NJ
early-stage Parkinson’s disease (Fast Track)(see also related conditions)--------------------------------------------------late-stage and mid-stage Parkinson’s disease(Fast Track)
Rockland, MAearly-stage Parkinson’s disease (see also related conditions)--------------------------------------------------late-stage and mid-stage Parkinson’s disease
levodopa-induced dyskinesia(see also Parkinson’s disease)
Phase I(800) 672-6372
Rockland, MAcognitive impairment associated with Parkinson’s disease (see also Parkinson’s disease)--------------------------------------------------levodopa-induced dyskinesia
Medicines in Development Parkinson’s Disease 20116
Glossary
application submitted—An application for marketing has been submitted by the company to the Food and Drug Administration (FDA).
dyskinesia—An impairment in the ability
spasmodic or repetitive motions or lack of coordination. Levodopa is a drug that effec-tively eliminates the major motor symptoms of PD and helps a person move again;; however, levodopa sometimes creates dyskinesia by causing too much movement.
Fast Track—A process designed to facilitate the development and expedite the review
unmet medical need. The status is assigned by the U.S. Food and Drug Administration. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a
determining factors include whether the drug will have an impact on such factors as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe condition to a more serious
as providing a therapy where none exists or providing a therapy which may be potentially superior to existing therapy. Once a drug receives Fast Track designation, early and frequent communication between the FDA and a drug company is encouraged throughout the entire drug development and review process.
The frequency of communication assures that questions and issues are resolved quickly, often leading to earlier drug approval and access by patients.
neurogenic orthostatic hypotention—A drop in blood pressure when changing position from lying to sitting or from sitting to standing, which
common symptom of Parkinson’s disease and can make patients pass out and fall.
Parkinson’s disease (PD)—PD belongs to a group of conditions called motor system disorders, which are the result of the loss of dopamine-producing brain cells. The four pri-mary symptoms of PD are tremor, or trembling in hands, arms, legs, jaw, and face;; rigidity, or stiffness of the limbs and trunk;; bradykinesia, or slowness of movement;; and postural insta-bility, or impaired balance and coordination. PD is both chronic, meaning it persists over a long period of time, and progressive, meaning its symptoms grow worse over time. As these symptoms become more pronounced, patients
-ing other simple tasks. Early symptoms of PD are subtle and occur gradually. In some people, the disease progresses more quickly than in others. As the disease progresses, the tremor, which affects the majority of PD patients, may begin to interfere with daily activities. Other symptoms may include depression and other
chewing, and speaking;; urinary problems or constipation;; skin problems;; and sleep disrup-tions. Although some people become se-verely disabled, others experience only minor
symptoms will affect an individual patient, and the intensity of the symptoms also varies from person to person.
Phase 0—First-in-human trials conducted in accordance with FDA’s 2006 guidance on
studies designed to speed up development of promising drugs by establishing very early on whether the agent behaves in human subjects as was anticipated from preclinical studies.
Phase I—Safety testing and pharmacological
Phase II—Effectiveness and safety testing in humans.
Phase III—Extensive clinical trials to demon-
sialorrhea—Drooling or excessive salivation, which is a common problem in neurologically impaired children (e.g., those with mental retardation or cerebral palsy) and in adults who have Parkinson’s disease or have had a stroke. It is commonly most caused by poor oral and facial muscle control.
The content of this report has been obtained through public, government and industry sources, and the Adis “R&D Insight” database based on the latest information. Report current as of October 24, 2011.about a particular product, contact the individual company directly or go to www.clinicaltrials.gov. The entire series of is available on PhRMA’s web site.
A publication of PhRMA’s Communications & Public Affairs Department. (202) 835-3460
Medicines in Development Parkinson’s Disease 2011 7
Selected Facts about Parkinson’s Disease in the United States
The number of people in the United States with Parkinson’s disease is estimated to be as many as 1 million, more than all people diagnosed with multiple sclerosis, muscular dystrophy and amyotrophic lateral sclerosis combined. Approximately 60,000 Americans are newly diagnosed each year.1
As the American population ages, those numbers are expected to grow. Worldwide, the number of people with Parkinson’s disease is expected to double in the next 25 years.2
Parkinson’s disease affects both men and women. The average age of onset of Parkinson’s disease is 61, but it may begin as early as age 40 or even before.2
People as young as 18 have been diag-nosed with Parkinson’s disease.3
Forty percent of people affected by Par-kinson’s disease are under the age of 60, placing them squarely in the workforce. Ex-perts say that about one-third of employed individuals will lose their jobs within a year of a Parkinson’s diagnosis, making lost
productivity a major factor in the societal impact of the disease.2
Parkinson’s disease reduces life expec-tancy by an average of three to nine years and is now the 14th leading cause of death in the United States.2
Red haired people have double the risk of developing Parkinson’s Disease. The pigment that colors hair red is made from L-dopa, just as is dopamine, the substance
disease.4
Sources:
1. Parkinson’s Disease Foundation (www.pdf.org)
(www.parkinsoninfo.org)
(www.parkinsonsaction.org)
4. Viartis (www.viartis.net)
Overview
Economic Impact
According to the Parkinson’s Action
Parkinson’s disease cost between $1,000 and $6,000 each year per patient.2
Annual medical care, including doctors’ visits, physical therapies, and treatment for co-occurring illnesses (such as depres-sion), is estimated at $2,000 to $7,000 for
people in early stages of the disease, and it is probably much higher for advanced stages.2
Surgical treatments for Parkinson’s dis-ease can cost $25,000 or more.2
As Parkinson’s disease progresses, insti-tutional care at an assisted-living facility
or nursing home may be required, and the costs can exceed $100,000 per person annually.2
In the United States, the combined direct and indirect cost of Parkinson’s disease is estimated to be $25 billion per year. This includes treatment costs, Social Security payments, and lost productivity.1
Clinical Trials
Discovery/ Preclinical Testing Phase I Phase II Phase III FDA Phase IV
Years 6.5 1.5 2 3.5 1.5
Additionalpost-
marketingtestingrequiredby FDA
Test Population
Laboratory and animal studies
20 to 100 healthy volunteers
100 to 500patient
volunteers
1,000 to 5,000patient
volunteersReviewprocess/approval
Purpose
Assess safety, biological activity and formulations
Determine safety
and dosage
Evaluate effective-
ness, look forside effects
Confirm effectiveness, monitor adverse reactions from long-term use
Success Rate
5,000compounds evaluated
5enter trials
1approved
The U.S. system of new drug approvals is
perhaps the most rigorous in the world.
It takes 10-15 years, on average, for an experimental drug to travel from lab to U.S.
compounds that enter preclinical testing make
is approved for sale.On average, it costs a company $1.2 billion, including the cost of failures, to get one new medicine from the laboratory to U.S. patients,
for the Study of Drug Development.
the laboratory, medicines are developed as follows:Preclinical Testing. A pharmaceutical com-pany conducts laboratory and animal studies to show biological activity of the compound against the targeted disease, and the com-pound is evaluated for safety.Investigational New Drug Application (IND). After completing preclinical testing, a company
Administration (FDA) to begin to test the drug
experiments;; how, where and by whom the new studies will be conducted;; the chemical structure of the compound;; how it is thought to work in the body;; any toxic effects found in the animal studies;; and how the compound is manufactured. All clinical trials must be reviewed and approved by the Institutional Review Board (IRB) where the trials will be conducted. Progress reports on clinical trials must be submitted at least annually to FDA and the IRB.Clinical Trials, Phase I. These tests usually involve about 20 to 100 healthy volunteers. The
safe dosage range. The studies also determine how a drug is absorbed, distributed, metabo-
its action.Clinical Trials, Phase II. In this phase, controlled trials of approximately 100 to 500 volunteer patients (people with the disease) assess a drug’s effectiveness and determine
Clinical Trials, Phase III. This phase usually involves 1,000 to 5,000 patients in clinics and
hospitals. Physicians monitor patients closely to
New Drug Application (NDA)/Biologic
License Application (BLA). Following the completion of all three phases of clinical trials,
demonstrate both safety and effectiveness.
information that the company has gathered. Applications typically run 100,000 pages or more. The average review time for the 26 new medicines approved by the FDA in 2010 was 14.1 months.Approval.
BLA, the new medicine becomes available for physicians to prescribe. A company must continue to submit periodic reports to FDA, including any cases of adverse reactions and appropriate quality-control records. For some medicines, FDA requires additional trials (Phase IV) to evaluate long-term effects. Discovering and developing safe and effective
process. Biopharmaceutical companies invested an estimated $67.4 billion in research and development in 2010.
The Drug Discovery, Development and Approval Process
