Fairfoundation.org2

HIV/AIDS
Researchers Are Testing 79 Medicines and Vaccines
For HIV and Opportunistic Infections
To better combat one of the world’s most devastating MEDICINES IN DEVELOPMENT FOR AIDS*
diseases, pharmaceutical and biotechnologyresearchers are testing 79 medicines for HIV/AIDS and related conditions, intensifying their efforts and working toward the development of vaccines. The medicines now in the pipeline will add to the 82 thathave already been approved since the virus that causes AIDS was first identified 20 years ago.
Vaccine research is considered crucial to control the AIDS pandemic. In what has been called “a silent revo-lution in AIDS vaccine research,” 16 vaccines are in AIDS has killed 28 million people and infected an *Some medicines are listed in more than one category.
estimated 42 million people worldwide, 70 percent ofwhom live in sub-Saharan Africa, where the pharmaceu- Examples of other AIDS medicines in the pipeline tical industry is making special efforts to counter the disease (see page 14). As many as 950,000 U.S. resi- • A protease inhibitor that, unlike other protease inhibitors, dents are estimated to be living with HIV infection, is not a peptide. The medicine, the first non-peptidic though the increased use of new medicines has helped to protease inhibitor with a unique chemical structure, substantially reduce the AIDS death rate in this country in has shown in clinical trials to reduce the levels of the recent years. By 2010, it’s estimated that 45 million new virus in patients who have become resistant to other people could be infected with the HIV virus—unless there’s a dramatic breakthrough. “A safe and effectiveHIV vaccine is critical to the control of HIV globally,” • An antisense gene therapy that uses two novel tech- says Dr. Anthony Fauci, Director of the National Institute nologies to boost immune responsiveness against HIV.
One involves the insertion of a new type of geneticmaterial into blood cells to inhibit the growth of the In addition to the 16 vaccines, the medicines in devel- virus. The second involves inserting new genes into opment for HIV/AIDS, which are either in human clinical target cells and integrating the gene into the chromo- trials or are at the Food and Drug Administration awaiting some of the cell. The cells containing the new genes approval, include 34 antivirals, 6 anti-infectives, 5 cancer are then transferred to the patient.
treatments, 8 immunomodulators, 3 antifungals, 2 genetherapies, and 8 other drugs.
While HIV/AIDS remains a formidable foe and world- wide scourge, pharmaceutical and biotechnology companies Of the medicines and vaccines in development, one are meeting the challenge by continuing their all-out efforts vaccine is designed to induce different types of immune to develop novel and more effective therapies and vaccines response, enhancing the overall immune response. The to contain the disease. We fully understand that patients first dose primes the body to induce cellular immunity.
all over the world are depending on them.
The booster dose, consisting of genetically engineeredHIV subunits, induces neutralizing antibodies. Anothervaccine candidate is made from a noninfectious copyof a protein from the surface of the AIDS virus. Thevaccine is designed to induce an immune response through the production of antibodies that will prevent the invading virus from attaching to a patient’s T-cells.
Medicines in Development for HIV/AIDS
A N T I C A N C E R S
Product Name
Indication
Development Status*
Avastin™
Rituxan®
Cambridge, MAGenentechSouth San Francisco, CA A N T I F U N G A L S
Product Name
Indication
Development Status
A N T I - I N F E C T I V E S
Product Name
Indication
Development Status
* For more information about a specific medicine in this report, please call the telephone number listed.
A N T I - I N F E C T I V E S
Product Name
Indication
Development Status
Ushercell
A N T I V I R A L S
Product Name
Indication
Development Status
Rsch. Triangle Park, NCVertex PharmaceuticalsCambridge, MA Alferon LDO®
Advanced Life SciencesWoodbridge, IL Doxovir™
A N T I V I R A L S
Product Name
Indication
Development Status
(Orphan Drug)
Kaletra®
lopinavir/ritonavir Abbott Park, IL Upsher-Smith LaboratoriesMaple Grove, MN Racivir®
Reverset™
A N T I V I R A L S
Product Name
Indication
Development Status
Valtrex™ XR
G E N E T H E R A P Y
Product Name
Indication
Development Status
I M M U N O M O D U L A T O R S
Product Name
Indication
Development Status
Alferon LDO®
Ampligen®
(Orphan Drug)
IMMUNITIN™
Remune®
V A C C I N E S
Product Name
Indication
Development Status
Simplirix
O T H E R S
Product Name
Indication
Development Status
Prosaptide™
Serostim™
origin) for injection
(Orphan Drug)
The content of this survey has been obtained through government and industry sources based on the latest informa-
tion. Survey current as of November 12, 2004. The information may not be comprehensive. For more specific
information about a particular product, contact the individual company directly. The entire series of “Medicines
in Development” is available on PhRMA’s web site.
PhRMA Internet address: http://www.phrma.org
Provided as a Public Service by PhRMA. Founded in 1958 as the Pharmaceutical Manufacturers Association.
Copyright 2004 by the Pharmaceutical Research and Manufacturers of America. Permission to reprint is awarded
if proper credit is given.
A P P R O V E D M E D I C I N E S F O R H I V I N F E C T I O N / A I D S
Product Name
Indication
Agenerase®
Rsch. Triangle Park, NCVertex PharmaceuticalsCambridge, MA Combivir®
Crixivan®
Emtriva®
HIV infection in combination with other antiretroviral Epivir®
HIV infection, HIV infection (once-daily dosing) Rsch. Triangle Park, NCBioChem PharmaLaval, Quebec Epzicom™
HIV infection in combination with other antiretroviral Fortovase®
treatment of HIV infection in adults in combination with Fuzeon®
in combination with other antiretroviral agents for Gamimune®-N
in combination with other antiviral agents for treatment of Invirase®
treatment of HIV infection in combination with other Kaletra®
treatment of HIV infection in adults and children A P P R O V E D M E D I C I N E S F O R H I V I N F E C T I O N / A I D S
Product Name
Indication
Lexiva™
treatment of HIV infection in combination with other Vertex PharmaceuticalsCambridge, MA Norvir®
Rescriptor®
Retrovir®
HIV positive (asymptomatic [CD4<500] and symptomatic [ARC, AIDS]), pediatric and adult, prevention of maternal/fetal Reyataz™
treatment of HIV-1 infection in combination with other Sustiva®
Sustiva®
Trizivir®
Truvada™
HIV infection in combination with other antiretroviral HIV infection, pediatric HIV infection, once-daily dosing VIDEX® EC
Viracept®
Viramune®
for use in combination with other antiretroviral agents for the A P P R O V E D M E D I C I N E S F O R H I V I N F E C T I O N / A I D S
Product Name
Indication
Viread®
HIV infection in combination with other antiretroviral HIV infection, pediatric HIV infection, first-line in Zerit® XR
treatment of HIV infection in combination with other Ziagen®
treatment of HIV infection in combination with other A P P R O V E D M E D I C I N E S F O R A I D S - R E L AT E D C O N D I T I O N S
Product Name
Indication
ABELCET®
treatment of severe systemic fungal infections in patients refractory to or intolerant of amphotericin B therapy Abreva™
topical treatment for recurrent oral-facial herpes simplex GlaxoSmithKlinePhiladelphia, PARsch. Triangle Park, NC Alferon N
genital warts (condyloma acuminata) Injection®
AmBisome®
primary treatment for fever of unknown origin in neutropenic patients, visceral leishmaniasis, secondary treatment for certain systemic fungal infections, cryptococcal meningitis, Amphotec®
aspergillosis, opportunistic systemic fungal infections Bactrim™
Bexxar®
Philadelphia, PARsch. Triangle Park, NC Biaxin™
Mycobacterium avium complex (MAC) prophylaxis Cancidas®
treatment of invasive aspergillosis in patients who caspofungin acetate Whitehouse Station, NJ are refractory to or intolerant of other therapies A P P R O V E D M E D I C I N E S F O R A I D S - R E L AT E D C O N D I T I O N S
Product Name
Indication
Cytovene®
CMV retinitis treatment of immunocompromised Cytovene®
CMV retinitis prophylaxis in AIDS patients Daraprim®
Philadelphia, PARsch. Triangle Park, NC DaunoXome®
DepoCyt™
Diflucan®
cryptococcal meningitis, candidiasis, pediatric use for candidiasis fungal infection prophylaxis and treatment Famvir®
recurrent herpes simplex, including infections in Foscavir®
CMV retinitis in AIDS patients, acyclovir-resistant herpes simplex virus (HSV) in immunocompromised patients Intron® A
Marinol® Capsules Solvay Pharmaceuticals
treatment of anorexia associated with weight loss in AIDS patients and for the treatment of refractory nausea and vomiting associated with cancer chemotherapy Megace®
treatment of anorexia and cachexia associated with AIDS Mepron®
treatment of mild to moderate PCP in individuals intolerant Mycobutin®
MAC prophylaxis in patients with advanced HIV infection NebuPent®
Neutrexin™
treatment of moderate-to-severe PCP in immunocompromised patients, including patients with AIDS, who are intolerant of or are refractory to TMP/SMX or for whom TMP/SMX is A P P R O V E D M E D I C I N E S F O R A I D S - R E L AT E D C O N D I T I O N S
Product Name
Indication
Nizoral® 2%
Nizoral®
systemic fungal infections (blastomycosis, candidiasis, chronic mucocutaneous candidiasis, chromomycosis, coccidioidomycosis, histoplasmosis, oral thrush, paracoccidioidomycosis) Onxol™
Oxandrin®
treatment of involuntary weight loss due to severe trauma, chronic infection, extensive surgery or unknown pathophysiology Panretin® Capsules Ligand Pharmaceuticals
Panretin® Gel
PASER™
Extended Release
Granules
para-aminosalicylic
acid 4-aminosalicylic
acid (PAS)
Pentam® 300
Priftin®
PROCRIT®
anemia in Retrovir®-treated HIV-infected patients
Prosorba® Column Cypress Bioscience
Roferon®-A
Kaposi’s sarcoma in patients 18 years of age or older Septra®
Serostim®
treatment of AIDS-associated cachexia (AIDS wasting) Sporanox®
histoplasmosis, blastomycosis, second-line aspergillosis Capsules
A P P R O V E D M E D I C I N E S F O R A I D S - R E L AT E D C O N D I T I O N S
Product Name
Indication
Sporanox®
Injection
Sporanox®
Oral Solution
Trovan™
Valtrex™
episodic treatment of recurrent genital herpes and herpes zoster in immunocompetent adults, suppression of genital herpes simplex virus (HSV), suppression of HSV in immunocompromised patients, prevention of HSV transmission, suppression of recurrent genital herpes in HIV-infected individuals, reduction of risk of transmission of genital herpes in otherwise healthy, heterosexual adults Vistide®
Vitravene™
WinRho SD™
immune thrombocytopenic purpura (ITP) secondary to Zithromax®
Mycobacterium avium intracellulare (MAI) Zovirax®
herpes zoster/simplex, treatment of initial episodes and management of recurrent episodes of genital herpes, I M P R O V I N G H E A L T H I N T H E D E V E L O P I N G W O R L D
In collaboration with local non-governmental and West Africa was set up by one company. The organizations and international health and relief program includes community outreach, education groups, pharmaceutical companies are working to improve public health in the developing world. From 1998 through 2003, the pharmaceutical industry • A pharmaceutical company-funded program known provided more than $4.1 billion in financial assistance as Step Forward .for the world’s children works to and medical products to developing nations. These improve the lives of AIDS orphans and vulnerable programs represent long-term commitments to form children in Tanzania, Burkina Faso, India, and global partnerships to help patients in these countries.
In addition to the medicines in development to • One company has joined with the Bill & Melinda treat HIV infection and related conditions, pharma- Gates Foundation to improve HIV/AIDS education, ceutical companies are helping the developing world care and treatment in Botswana. The company has combat HIV infection and AIDS in several ways.
donated more than $50 million worth of antiretro-viral medicines.
HIV/AIDS afflicts an estimated 39 million adults and three million children worldwide, and more than • With a $5 million grant, one company has funded 70 percent of the afflicted people live in sub-Saharan the Enhancing Care initiative program for people Africa. According to the World Health Organization, living with HIV/AIDS in developing countries.
today AIDS is the leading cause of death in Africa and • One company actively supports SHARE, a multina- the fourth worldwide. Pharmaceutical companies are tional program that teaches doctors, health care working in a variety of ways to fight HIV/AIDS in workers, resource planners and public health experts Africa and other parts of the developing world.
about prevention and management of HIV.
• One company provides rapid HIV tests and anti- • Through the Accelerating Access Initiative, several retroviral drugs in Africa and the 49 least developed companies are selling AIDS medicines in develop- countries (LDC); 20 million tests were pledged to ing countries at deeply discounted prices. Other programs for the prevention of mother to child companies are selling medicines at or below cost.
• One company is giving a medicine to treat deadly • A company pledged $20 million in a partnership opportunistic fungal infections free to patients in with Tanzania to build the country’s AIDS response South Africa and other developing nations.
• One company is supplying a protease inhibitor free • Through Positive Action, one company enhances of charge to prevent mother-to-child transmission of the capacity of community organizations in 27 nations to deliver HIV education, prevention, careand support, and eliminate stigma/discrimination.
• One company has established the Academic Alliance for AIDS Care and Prevention in Africa and will • One company supports training in China, Thailand fund a clinic that will be operated by the alliance and other countries on touch/massage therapy for in partnership with Makerere University in Uganda.
orphaned/abandoned HIV-infected babies andchildren.
• Through the Elizabeth Glaser Pediatric AIDS Foundation, one company donated $1 million in • One company has extended indefinitely a program grants to hospitals and universities in Africa to that offers training and free medicine in Africa and reduce mother-to-child transmission of the virus.
Haiti to combat opportunistic infections associatedwith AIDS.
• Secure the Future, a program to address the needs of women and children affected by AIDS in South G L O S S A R Y
application submitted—Application for
produce the sores. Herpes varicella
aspergillosis—Infection caused by
zoster virus (HVZ), also called
primitive or are poorly differentiated.
shingles, consists of very painful
MAC/MAI—MAC refers to Myco-
bacterium avium complex. Myco- bacterium avium intracellulare (MAI) candidiasis—A fungal (Candida)
most internal organs, resulting in widely disseminated disease in AIDS patients.
histoplasmosis—A disease caused
neuropathic pain—Caused by disease,
CMV (cytomegalovirus)—This is
HIV positive/infection/disease—
(the virus that causes AIDS). HIV-l
NRTI—Nucleoside reverse transcrip-
can cause CMV retinitis, an inflam-
NNRTI—Non-nucleoside reverse
human papillomavirus (HPV)—More
cryptococcal meningitis—A fungal
NtRTI—Nucleotide analogue reverse
types of HPV cause genital warts.
PCP (Pneumocystis carinii pneumo-
nia)—A type of lung infection rarely
genital herpes—See herpes simplex
genital warts (condyloma acuminata)
STDs in the world.
immune thrombocytopenia
purpura—A condition in which there
Phase I—Safety testing and pharma-
caused by a human papillomavirus.
Phase II—Effectiveness testing and
identification of side effects in humans.
hepatitis—Inflammation of the liver
Phase III—Extensive clinical trials in
IM—Intramuscular.
infection, e.g., hepatitis B, but also by
certain drugs, chemicals or poisons.
immunocompromised—A condition
PI—Protease inhibitor.
prophylaxis—Treatment intended to
ing). Hepatitis B virus (HBV) can be
IV—Intravenous.
TMP/SMX—Refers to trimethoprim-
Kaposi’s sarcoma—A rare malignant
sulfamethoxazole, an approved
herpes simplex virus—Three strains
AIDS patients: Herpes simplex virus I
toxoplasmosis—A disease due to infec-
(HSV I), which causes cold sores or
lymphoma—Cancers in which the cells
encephalitis (inflammation of the brain).
wasting syndromes—Any number of
conditions, such as anorexia and
cachexia, resulting in a loss of body
Herpes simplex II (HSV II) causes
abnormal cell. All others are non-
painful sores of the anus or genitals.
Hodgkin’s lymphomas, which vary in
S E L E C T E D F A C T S A B O U T H I V / A I D S
U.S. AIDS Cases
U.S. AIDS Deaths
through December 20021
through December 20021
HIV/AIDS Worldwide 2
• At the end of 2003, an estimated 37.8 million people worldwide—35.7 million adults and 2.1 million children younger than age 15—were living with HIV/AIDS. About two-thirds (25 million) of these people live in sub-
Saharan Africa; another 20 percent (7.4 million) live in Asia and the Pacific. Women account for nearly half of all
people worldwide living with HIV/AIDS.
• An estimated 4.8 million new HIV infections occurred worldwide during 2003—about 14,000 infections each day.
More than 95 percent of these new infections were in developing countries. Also that year, some 6,000 youngpeople ages 15 to 24 became infected with HIV every day.
• In 2003 alone, HIV/AIDS-associated illnesses caused the deaths of about 2.9 million people worldwide, including
an estimated 490,000 children younger than age 15.
HIV/AIDS in the United States
• The Centers for Disease Control and Prevention (CDC) estimate that 850,000 to 950,000 U.S. residents are living with HIV infection, one-fourth of whom are unaware of their infection.2 • Approximately 40,000 new HIV infections occur each year in the United States: about 70 percent among males and 30 percent among females. Half of these newly infected people are younger than age 25.2 • Fewer than half of all HIV-infected American adults receive regular medical care. Only one-third of all HIV-infected Americans have private insurance, while fully one-fifth is uninsured. Public insurance—Medicaid and Medicare—covers the remaining half.3 • HIV transmission from mother to child during pregnancy, labor, and delivery or by breast-feeding has accounted for 91 percent of all AIDS cases reported among U.S. children. During the early 1990s, before perinatal preventivetreatments were available, an estimated 1,000 to 2,000 U.S. infants were born with HIV infection annually.
Between 1992 and 2002, perinatally acquired AIDS cases declined 90 percent in the United States, from 912 casesto 90 cases.1 • Diagnoses of HIV/AIDS increased 3.2 percent from 2001 (25,643) to 2002 (26,464). The age group 25-34 repre- sented 28 percent of all new diagnoses of HIV/AIDS. Although the rate of diagnoses remained stable among AfricanAmericans, in 2002 they accounted for 54 percent of all new diagnoses. That year, 71 percent of all new HIV/AIDSdiagnoses occurred in men.1 • In 2001, 39 percent of all HIV infections progressed to AIDS within 12 months after the HIV diagnosis.1 • AIDS incidence increased throughout the 1980s, declined from the mid-1990s through 2001, and increased 2 percent in 2002 (compared with 2001). The age group 35-44 represented 41 percent of all new diagnoses of AIDS in 2002.
The number of AIDS diagnoses increased 7 percent among women and decreased 5 percent among men from 1998through 2002. That year, men accounted for 74 percent of all new AIDS diagnoses among U.S. adults.1 • AIDS prevalence continued to increase over the period 1998-2002. At the end of 2002, an estimated 384,906 people in the United States were known to be living with AIDS. Of those, 42 percent were in the age group 35-44;50 percent were black, 38 percent white, 10 percent Hispanic; and 73 percent were men.1 S E L E C T E D F A C T S A B O U T H I V / A I D S
HIV/AIDS in the United States (continued)
• The estimated number of deaths among people with AIDS declined 14 percent from 1998 (19,005) to 2002 Opportunistic Infections
• Esophageal candidiasis has been reported to be the most common opportunistic infection in developed countries,
affecting up to 20 percent of AIDS patients.4 • Worldwide, cryptococcosis is the second or third most common opportunistic infection in HIV/AIDS.2
• Combination antiviral therapy has reduced the rate of cytomegalovirus (CMV) in people with HIV by 75 percent,
yet about 5 percent of people with HIV still develop CMV.5 • The rates of opportunistic fungal infections in AIDS can range from 60 percent to 90 percent for mucosal candidia-
sis to 6 percent to 30 percent for invasive mycoses (such as cryptococcosis, histoplasmosis and coccidioimycosis).2
• Hepatitis B is the most common serious liver infection in the world and is 100 times more infectious than the AIDS
virus. The CDC estimates that 1.25 million Americans are chronically infected with hepatitis B, and 100,000 peoplewill become newly infected this year.6 Of those new cases, about half will be the result of sexual intercourse. In1995, about one-third of people with acute hepatitis B virus infections had a history of another sexually transmitteddisease.1 Up to 6,000 people die of hepatitis B-related liver complications each year.6 • Hepatitis C virus (HCV) is the most common chronic bloodborne infection in the United States. Some 4 million
people have been infected with HCV. Up to 85 percent of the people infected with HCV each year will developchronic infection.7 • In 1999, the CDC added HCV infection to the list of opportunistic infections associated with HIV. Up to one-fourth
of HIV-positive individuals in this country are co-infected with HCV. Since 2000, HCV-related end-stage liverdisease has been recognized as a leading cause of death among people with HIV.8 One study found that morethan 40 percent of deaths in HIV-positive individuals were associated with liver disease either as a primary orassociated cause of death.9 • Transmission of HCV to offspring among mothers with HCV infection alone is estimated to be about 2 percent, but
rates two to three times as high have been reported among those with both HCV and HIV infection.10 • Genital herpes affects an estimated 1 out of 4 (or 45 million) Americans, with about 500,000 new cases developing
• Health experts estimate that there are more cases of genital human papillomavirus (HPV) infection than of any other
sexually transmitted disease (STD) in the United States. Approximately 5.5 million new cases of sexually transmittedHPV infections are reported every year, and as many as 24 million Americans are already infected.2 • About two-thirds of people who have sexual contact with a partner who has genital warts (caused by low-risk types
of HPV) usually develop warts themselves within three months of contact.2
• In the past, about 1 in 4 homosexual or bisexual males with AIDS developed Kaposi’s sarcoma (KS). Today,
because of more effective HIV treatments, the rate of KS among that population has dropped to about one-seventhof what it was at its peak.11 • AIDS-related lymphoma is sometimes called non-Hodgkin’s lymphoma (NHL). About 10 percent of people with
HIV may eventually develop NHL. The rate of lymphoma in people with HIV is over 80 times higher than for the
general population. Some new studies have shown a decrease of about 40 percent in NHL rates in HIV patients due
to combination antiviral therapy, but lymphoma still accounts for the deaths of about 20 percent of people with HIV.5
S E L E C T E D F A C T S A B O U T H I V / A I D S
Opportunistic Infections (continued)
• Pneumocystis carinii pneumonia (PCP) is the most common opportunistic infection in people with HIV. Without
treatment, more than 85 percent of people with HIV would eventually develop PCP. Between 1991 and 1997, therewas a 36 percent drop in the number of PCP cases in the United States, and since people started taking combina-tion antiviral therapy, the number of cases has dropped even further. PCP was the first AIDS-defining diagnosis foronly 32 percent of cases in 1993 compared to 63 percent in 1987. PCP was the cause of death for 14 percent ofpeople with AIDS in 1993 compared to 32 percent in 1987.5 • The United States has the highest rates of sexually transmitted diseases (STDs, including HIV/AIDS, gonorrhea,
genital herpes, human papillomavirus and hepatitis B) in the industrialized world—50 to 100 times higher. An esti-
mated 15.3 million new cases of STDs are reported each year in this country. Health problems caused by STDs
tend to be more severe for women than for men.1
• The average lifetime cost per case of genital herpes is $417 for women and $511 for men. The total direct medical
costs for the disease in 2000 were $292.7 million.12 • The average lifetime cost per case of hepatitis B is $779. In 2000, the total direct medical costs for the disease were
• The 48-week course of treatment for hepatitis C virus infection costs an estimated $40,000.8
• Researchers estimate that the lifetime treatment cost for a person with HIV now averages about $155,000. Estimates
are that 40,000 people are infected yearly, resulting in an annualized cost of more than $6 billion. The cumulativecost of lifetime treatment increases by more than $6 billion yearly if the number of infections stays steady, as it hasover the last decade. In the last five years alone, an estimated 200,000 people have been infected with HIV.
Treating them over the rest of their lives will cost the nation $31 billion.1 • In the United States, preventing mother-to-child HIV transmission costs about $33,000 per infection averted. The
total lifetime treatment cost for perinatally infected infants is estimated to be between $51.8 million and $68.5million, assuming 280-370 perinatal infections per year and a lifetime cost of $185,000 per infant.1 • The average lifetime cost per case of human papillomavirus (HPV) is $1,228 for women and $27 for men. In 2000,
the total direct medical costs for HPV were $2.9 billion.12 • In 1994, the direct costs (such as expenditures for medical and non-medical services and materials) and indirect costs (mainly lost wages) of the major STDs (including HIV/AIDS, herpes simplex, human papillomavirus and
hepatitis B) and their complications were estimated to total nearly $17 billion annually.1
Sources:
1. U.S. Centers for Disease Control and Prevention (www.cdc.gov)2. National Institute of Allergy and Infectious Diseases (www.niaid.nih.gov)3. Rand Health (www.rand.org)4. HIV InSite, a project of the University of California/San Francisco (www.hivinsite.ucsf.edu)5. The Body: An AIDS and HIV Information Resource (www.thebody.com)6. Hepatitis B Foundation (www.hepb.org)7. American Liver Foundation (www.liverfoundation.org)8. aidsinfonyc.org, Treatment Action Group (www.aidsinfonyc.org)9. Reuters Health Information (www.hivandhepatitis.com) 10. MedReviews, LLC: “HCV and HIV: A Tale of Two Viruses,” Kenneth E. Sherman, M.D., Ph.D., Division of Digestive Diseases, University of Cincinnati College of Medicine, Cincinnati, OH (www.medreviews.com) 11. American Cancer Society (www.cancer.org)12. Focus on the Family (www.family.org) T H E D R U G D I S C O V E R Y , D E V E L O P M E N T A N D A P P R O V A L P R O C E S S
It takes 10-15 years on average for an experimental drug to travel from the lab to U.S. patients.
Only five in 5,000 compounds that enter preclinical testing make it to human testing. One of these five tested in people is approved.
Clinical Trials
Discovery/
Preclinical Testing
Population
T H E D R U G D E V E L O P M E N T A N D A P P R O V A L P R O C E S S
The U.S. system of new drug approvals is perhaps Clinical Trials, Phase I. These tests involve about
20 to 100 normal, healthy volunteers. The tests study a It takes 10 to 15 years on average for an experimental drug’s safety profile, including the safe dosage range.
drug to travel from lab to U.S. patients, according to The studies also determine how a drug is absorbed, the Tufts Center for the Study of Drug Development, distributed, metabolized, and excreted as well as the based on drugs approved from 1994 through 1998.
Only five in 5,000 compounds that enter preclinical Clinical Trials, Phase II. In this phase, controlled
testing make it to human testing. And only one of those trials of approximately 100 to 500 volunteer patients (people with the disease) assess a drug’s effectiveness. On average, it costs a company $802 million to Clinical Trials, Phase III. This phase usually involves
get one new medicine from the laboratory to U.S.
1,000 to 5,000 patients in clinics and hospitals.
patients, according to a November 2001 report by the Physicians monitor patients closely to confirm efficacy Tufts Center for the Study of Drug Development.
Once a new compound has been identified in the New Drug Application (NDA). Following the
laboratory, medicines are developed as follows: completion of all three phases of clinical trials, a Preclinical Testing. A pharmaceutical company con-
company analyzes all of the data and files an NDA ducts laboratory and animal studies to show biological with FDA if the data successfully demonstrate both activity of the compound against the targeted disease, safety and effectiveness. The NDA contains all of and the compound is evaluated for safety. the scientific information that the company has gathered. NDAs typically run 100,000 pages or Investigational New Drug Application (IND). After
more. The average NDA review time for 21 new completing preclinical testing, a company files an IND molecular entities (NMEs) approved by the FDA in with the U.S. Food and Drug Administration (FDA) to begin to test the drug in people. The IND becomeseffective if FDA does not disapprove it within 30 days.
Approval. Once FDA approves an NDA, the new
The IND shows results of previous experiments; how, medicine becomes available for physicians to where and by whom the new studies will be prescribe. A company must continue to submit conducted; the chemical structure of the compound; periodic reports to FDA, including any cases of how it is thought to work in the body; any toxic adverse reactions and appropriate quality-control effects found in the animal studies; and how the records. For some medicines, FDA requires additional compound is manufactured. All clinical trials must be trials (Phase IV) to evaluate long-term effects.
reviewed and approved by the Institutional Review Discovering and developing safe and effective new Board (IRB) where the trials will be conducted.
medicines is a long, difficult, and expensive process.
Progress reports on clinical trials must be submitted at PhRMA member companies invested an estimated $33.2 billion in research and development in 2003.
Medicines in Development for AIDS is presented by PhRMA in cooperation with the following organizations: AIDS ActionAIDS Project Los AngelesAIDS Research AllianceAmerican Academy of Allergy, Asthma and ImmunologyAmerican Academy of Physician AssistantsAmerican Association of ImmunologistsAmerican College of Allergy, Asthma & ImmunologyAmerican Medical Directors AssociationAmerican Nurses AssociationAmerican Social Health AssociationAmerican Society for MicrobiologyAssociation of Nurses in AIDS CareCAVDA-Citizens AIDS ProjectCenter for Women Policy StudiesChildren’s AIDS FundClinical Immunology SocietyElizabeth Glaser Pediatric AIDS FoundationFoundation for Retrovirology and Human HealthHealth Education Resource Organization (HERO)Health Information Network/SeattleInfectious Diseases Society of AmericaInteramerican College of Physicians & SurgeonsMothers’ VoicesNational Alliance for Hispanic HealthNational Black Nurses AssociationNational Foundation for Infectious DiseasesNational Medical AssociationNational Native American AIDS Prevention CenterOffice of AIDS Research, National Institutes of HealthPlanned Parenthood Federation of AmericaWomen Alive Coalition Being listed in this report in no way implies that the above-mentioned organizations endorse or recommend the useof any of the products in development contained in this publication. For further information, patients should consulttheir physicians or health care providers.
Pharmaceutical Research and Manufacturers of America1100 Fifteenth Street, NWWashington, DC 20005

Source: http://www.fairfoundation.org/pharma02.pdf