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2012 Repo r t

Medicines in Development HIV/AIDS presented by america’s biopharmaceutical research companies

Biopharmaceutical Researchers Are Testing More Than 70 Medicines and Vaccines For HIV

Medicines in Development for HIV/AIDS

40 1.2 million Americans

25 are living with HIV, 50,000 are newly diagnosed each year 4 4

s Biopharmaceutical research companies vaccine given to only a third of the population are developing 73 medicines and vaccines, could reduce new HIV by 24 percent Antivirals Therapy Vaccines focusing on improved treatment regimens, over 15 years. more effective therapies and promising new The medicines in the development pipeline preventative vaccines. Cell/Gene Immunomodulator include: Although HIV/AIDS is one of the most • A gene therapy that uses genetic material to Medicines in Development devastating diseases affecting people around remove disease-causing aspects of the virus. for HIV/AIDS by Phase of the world, the number of new infections has Development been steadily declining. In the United States, • A transdermal vaccine that helps suppress vi- rus replication and destroys HIV-infected cells. 35 the AIDS-related death rate has fallen by 79 percent due to antiretroviral therapy. 28 • A first-in-class medicine intended to prevent the Over the past 30 years, nearly 40 medicines HIV virus from breaking through the . have been approved to treat HIV/AIDS. Despite the incredible progress to date, the Testing for the disease also has advanced HIV/AIDS epidemic remains a complex prob- dramatically, enabling earlier treatment. While lem. America’s biopharmaceutical research 5 4 2 these medicines have helped to prolong the companies are continuing their efforts to de- lives of HIV-infected patients, making HIV a velop novel and more effective therapies, vac-

manageable chronic disease, opportunities for cines to prevent the disease, and potentially a even greater progress remain. Phase I cure, so the millions of patients suffering today Phase IIPhase III For example, biopharmaceutical companies have hope for a better tomorrow. UnspecifiedApplicationSubmitted are intensifying their efforts to develop vac- * Some medicines are listed in more than one cines that would help prevent HIV. Current phase of development. estimates show that a 50 percent effective Medicines in Development for HIV/AIDS

Antivirals

Product Name Sponsor Indication Development Status*

// ViiV Healthcare HIV infection therapy in Phase III fixed-dose combination Rsch. Triangle Park, NC treatment-naive patients (877) 844-8872 ( inhibitor/reverse transcriptase inhibitor)

(DAPD) RFS Pharma HIV-1 infection treatment Phase II Tucker, GA (404) 601-1430

BI-224436 HIV infection treatment Phase I completed () Foster City, CA (800) 445-3235

CB1922 Canopus BioPharma HIV infection treatment Phase II (synthetic steroidal lactone) Studio City, CA www.canopusbiopharma.com

Tobira Therapeutics HIV-1 infection treatment Phase II (CCR5 antagonist) South San Francisco, CA (650) 741-6625

CMX157 Merck HIV infection treatment Phase I completed (tenofovir PIM conjugate) Whitehouse Station, NJ (800) 672-6372

Gilead Sciences HIV infection treatment application submitted (PK enhancer) Foster City, CA (800) 445-3235

cobicistat/ Gilead Sciences HIV infection Phase I fixed-dose combination Foster City, CA (800) 445-3235 (PK enhancer/protease Janssen Therapeutics (800) 526-7736 inhibitor) Titusville, NJ

cobicistat/darunavir/ Gilead Sciences HIV-1 infection Phase II /GS-7340 Foster City, CA (800) 445-3235 fixed-dose combination Janssen Therapeutics (800) 526-7736 Titusville, NJ

cobicistat// Gilead Sciences HIV-1 infection Phase II emtricitabine/GS-7340 Foster City, CA (800) 445-3235 fixed-dose combination

dapivirine International Partnership for HIV infection prevention Phase I/II completed (NNRTI) Microbicides (vaginal ring) (301) 608-2221 Silver Spring, MD ------HIV infection prevention Phase I/II completed (vaginal gel) (301) 608-2221

dolutegravir Shionogi HIV-1 infection treatment Phase III (S/GSK1349572) Florham Park, NJ (973) 966-6900 (integrase inhibitor) ViiV Healthcare (877) 844-8872 Rsch. Triangle Park, NC

*For more information about a specific medicine in this report, please call the telephone number listed. 2 Medicines in Development HIV/AIDS 2012 Medicines in Development for HIV/AIDS

Antivirals

Product Name Sponsor Indication Development Status

/lamivudine/ Mylan Laboratories HIV-1 infection treatment application submitted tenofovir fumarate Canonsburg, PA (724) 514-1800 fixed-dose combination

elvitegravir Gilead Sciences HIV-1 infection treatment application submitted (integrase inhibitor) Foster City, CA (800) 445-3235

Achillion Pharmaceuticals HIV infection treatment Phase II (NRTI) New Haven, CT (203) 624-7000

GS-7340 Gilead Sciences HIV infection treatment Phase II (NtRTI) Foster City, CA (800) 445-3235

HIV attachment inhibitor Bristol-Myers Squibb HIV infection treatment Phase II Princeton, NJ (800) 332-2056

HIV Bristol-Myers Squibb HIV infection treatment in clinical trials Princeton, NJ (800) 332-2056

TaiMed Biologics USA HIV-1 infection treatment Phase II (TMB-355) Irvine, CA (intravenous) (Fast Track) (949) 769-6543 (fusion inhibitor) ------HIV-1 infection treatment Phase I (subcutaneous) (949) 769-6543

Intelence® Janssen Therapeutics HIV infection combination therapy in Phase II Titusville, NJ treatment-naive patients (800) 526-7736 (NNRTI) (Fast Track)

KD-247 Kaketsuken HIV-1 infection treatment Phase I () Kumamoto, Japan www.kaketsuken.or.jp

KP-1461 Koronis Pharmaceuticals HIV infection treatment Phase II (replication inhibitor) Redmond, WA (425) 825-0240

lamivudine (3TC)// Abbott Laboratories HIV-1 infection treatment in clinical trials fixed-dose Abbott Park, IL (847) 937-6100 combination

lamivudine (3TC)// GlaxoSmithKline HIV infection Phase I completed fixed-dose Rsch. Triangle Park, NC (888) 825-5249 combination

lersivirine (UK-453061) ViiV Healthcare HIV infection treatment Phase II (NNRTI) Rsch. Triangle Park, NC (877) 844-8872

Lexiva® Vertex Pharmaceuticals HIV infection treatment in Phase II Cambridge, MA adolescents, children and infants (617) 444-6100 (PI) ViiV Healthcare (877) 844-8872 Rsch. Triangle Park, NC

Medicines in Development HIV/AIDS 2012 3 Medicines in Development for HIV/AIDS

Antivirals

Product Name Sponsor Indication Development Status

MK-1439 Merck HIV-1 infection treatment Phase I (NNRTI) Whitehouse Station, NJ (800) 672-6273

Norvir® Abbott Laboratories HIV infection treatment in clinical trials ritonavir Abbott Park, IL (847) 937-6100 powdered formulation (PI)

NRT inhibitor Bristol-Myers Squibb HIV infection treatment Phase II Princeton, NJ (800) 332-2056

Prezista® Janssen Therapeutics HIV infection application submitted darunavir Titusville, NJ (800) 526-7736 (once-daily 800 mg)

PRO 140 CytoDyn HIV-1 infection prevention and Phase II completed (CCR5 ) Lake Oswego, OR treatment (971) 204-0382

RAP101 RAPID Pharmaceuticals HIV infection treatment Phase II (CCR5 receptor antagonist) Huenenberg, Switzerland www.rapidpharma.com

RPI-MN ReceptoPharm HIV infection treatment Phase I Plantation, FL (954) 321-8988

S/GSK1265744 Shionogi HIV infection treatment Phase II (integrase inhibitor) Florham Park, NJ (973) 966-6900 ViiV Healthcare (877) 844-8872 Rsch. Triangle Park, NC

SPL-7013 Starpharma HIV infection prevention Phase I completed (vaginal gel) Melbourne, Australia (Fast Track) www.starpharma.com

TBR-220 Tobira Therapeutics HIV infection treatment Phase I (CCR5 receptor antagonist) South San Francisco, CA (650) 741-6625

tenofovir vaginal gel CONRAD HIV infection prevention Phase I (NtRTI) Arlington, VA (703) 524-4744 International Partnership for Microbicides Silver Spring, MD

TMC310911 Janssen Therapeutics HIV infection treatment Phase II completed (PI) Titusville, NJ (800) 526-7736

4 Medicines in Development HIV/AIDS 2012 Medicines in Development for HIV/AIDS

Antivirals

Product Name Sponsor Indication Development Status

UB-421 United Biomedical HIV-1 infection treatment Phase II (FI) Hauppauge, NY (631) 273-2828

VRX806 Valeant Pharmaceuticals HIV infection treatment Phase II (NNRTI) Mississauga, Canada (905) 286-3000

Cell Therapy/Gene Therapy

Product Name Sponsor Indication Development Status

HIV gene therapy Adaptimmune HIV infection Phase I Philadelphia, PA (267) 499-2066 Cardiff University Cardiff, Wales University of Pennsylvania Philadelphia, PA

lexgenleucel-T VIRxSYS HIV infection therapy in Phase II (replication inhibitor) Gaithersburg, MD treatment-experienced patients (301) 987-0480

SB-728-T Sangamo BioSciences HIV infection treatment Phase II Richmond, CA (510) 970-6000

Stealth Vector® Enzo Therapeutics HIV-1 infection treatment Phase I/II HGTV-43™ New York, NY (212) 583-0100 antisense gene medicine

Immunomodulators

Product Name Sponsor Indication Development Status

AMZ0026 Amazon Biotech HIV infection treatment Phase I/II New York, NY (212) 444-1019

CYT107 Cytheris HIV infection treatment Phase II (recombinant human Rockville, MD (301) 231-0450 interleukin-7)

Cytolin® CytoDyn HIV infection treatment Phase I anti-CD8 mAb Lake Oswego, OR (971) 204-0382

IRT-103 TNI BioTech HIV infection treatment Phase II (low-dose naltrexone) New York, NY www.tnibiotech.com

Medicines in Development HIV/AIDS 2012 5 Medicines in Development for HIV/AIDS

Vaccines

Product Name Sponsor Indication Development Status

ADVAX Aaron Diamond AIDS Research HIV infection prevention Phase I completed (DNA vaccine) Center (212) 448-5000 New York, NY (212) 847-1111 International AIDS Vaccine Initiative ------New York, NY HIV infection prevention Phase I completed Ichor Medical Systems (new delivery system) (212) 448-5000 San Diego, CA (212) 847-1111

AGS-004 Argos Therapeutics HIV-1 infection treatment Phase II (autologous Durham, NC (919) 287-6300 vaccine-intradermal injection)

AVX101 AlphaVax HIV-1 infection prevention Phase I (single gene HIV vaccine) Rsch. Triangle Park, NC (919) 595-0400

DCVax-001 Celldex Therapeutics HIV infection prevention and Phase I (recombinant protein vaccine) Needham, MA treatment (781) 433-0771 Rockefeller University New York, NY

DermaVir™ Patch Genetic Immunity HIV-1 infection treatment Phase II DNA topical patch vaccine McLean, VA (703) 879-6803

HIV vaccine Mymetics HIV infection prevention Phase I Epalinges, Switzerland www.mymetics.com

HIV recombinant vaccine GlaxoSmithKline HIV-1 infection prevention Phase I Rsch. Triangle Park, NC (888) 825-5249

HIV vaccine Crucell HIV infection prevention Phase I Leiden, The Netherlands (212) 847-1111 Beth Israel Deaconess Medical Center Boston, MA International AIDS Vaccine Initiative New York, NY

HIV vaccine GeoVax Labs HIV infection prevention Phase II Smyrna, GA (678) 384-7220

HIV vaccine GeoVax Labs HIV infection treatment Phase I/II Smyrna, GA (678) 384-7220

6 Medicines in Development HIV/AIDS 2012 Medicines in Development for HIV/AIDS

Vaccines

Product Name Sponsor Indication Development Status

HIV vaccine Massachusetts General Hospital HIV infection Phase I Boston, MA (617) 726-2000 Opal Therapeutics Parkville, Australia

HIV vaccine Vaccines & Diagnostics HIV infection Phase I Cambridge, MA (617) 871-7000 National Institutes of Health Bethesda, MD

HIV vaccine PaxVax HIV infection prevention in clinical trials San Diego, CA (858) 450-9595

HIV vaccine Profectus Biosciences HIV-2 infection prevention Phase I (MAG pDNA) Baltimore, MD (866) 938-8559

HIV vaccine Profectus Biosciences HIV infection prevention Phase I (rVSV) Baltimore, MD (866) 938-8559

HIV vaccine Sumagen HIV-1 infection Phase I (SAV001) Seoul, South Korea www.sumagen.co.kr

HIVAX™ GeneCure Biotechnologies HIV-1 infection Phase I replication-defective Norcross, GA (770) 263-7508 HIV-1 vaccine

ITV-1 Immunotech Laboratories HIV infection treatment in clinical trials immune therapeutic vaccine Pasadena, CA (818) 409-9091

Pennvax®-B Inovio Pharmaceuticals HIV infection prevention and Phase I DNA vaccine (clade B) Blue Bell, PA treatment (267) 440-4200

Pennvax®-G Inovio Pharmaceuticals HIV infection prevention Phase I DNA vaccine (clades A, C, D) Blue Bell, PA (267) 440-4200

TUTI-16 Thymon HIV-1 infection treatment Phase I/II (lipoprotein vaccine) Short Hills, NJ (973) 467-9558

vacc-4x Bionor Pharma HIV-1 infection treatment Phase II (intradermal vaccine) Oslo, Norway www.bionorpharma.com

Medicines in Development HIV/AIDS 2012 7 Medicines in Development for HIV/AIDS

Vaccines

Product Name Sponsor Indication Development Status

VRC-HIVADV014-00-VP GenVec HIV infection prevention Phase II completed (HIV-1 recombinant adenovirus Gaithersburg, MD www.vrc.nih.gov vaccine) Vaccine Research Center (NIAID) Bethesda, MD

VRC-HIVADV027-00-VP GenVec HIV infection prevention Phase I (HIV adenovector Ad35 vaccine) Gaithersburg, MD www.vrc.nih.gov Vaccine Research Center (NIAID) Bethesda, MD

VRC-HIVDNA016-00-VP Vaccine Research Center (NIAID) HIV infection prevention Phase II Bethesda, MD www.vrc.nih.gov

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 November 16, 2012. The information in this report may not be comprehensive. For more specific informa- tion about a particular product, contact the individual company directly or go to www.clinicaltrials.gov. The entire series of Medicines in Development is available on PhRMA’s web site. A publication of PhRMA’s Communications & Public Affairs Department. (202) 835-3460 www.phrma.org | www.innovation.org | www.pparx.org | www.buysafedrugs.info Provided as a Public Service by PhRMA. Founded in 1958 as the Pharmaceutical Manufacturers Association.

Copyright © 2012 by the Pharmaceutical Research and Manufacturers of America. Permission to reprint is awarded if proper credit is given.

Pharmaceutical Research and Manufacturers of America • 950 F Street, NW, Washington, DC 20004

8 Medicines in Development HIV/AIDS 2012 Approved Medicines for HIV Infection/AIDS

entry Inhibitors

• Selzentry® (maraviroc) ViiV Healthcare • Fuzeon® () Genentech, Trimeris

Integrase Inhibitor

• Isentress® () Merck

Nucleoside Reverse Transcriptase Inhibitors (NRTI)

• Combivir® (lamivudine/zidovudine) ViiV Healthcare

• Emtriva® (emtricitabine) Gilead Sciences

• Epivir® (lamivudine) ViiV Healthcare

• Epzicom® (abacavir/lamivudine) ViiV Healthcare

• Hivid® () Roche, marketing discontinued

• Retrovir® (zidovudine) ViiV Healthcare

• Trizivir® (abacavir/lamivudine/zidovudine) ViiV Healthcare

• Videx® () Bristol-Myers Squibb

• Videx® EC (didanosine delayed release) Bristol-Myers Squibb

• Zerit® () Bristol-Myers Squibb

• Zerit® XR (stavudine extended-release) Bristol-Myers Squibb, marketing discontinued

• Ziagen® (abacavir) ViiV Healthcare

Non-Nucleoside Reverse Transcriptase Inhibitors (NNRTI)

• Edurant™ () Janssen Therapeutics

• Intelence® (etravirine) Janssen Therapeutics

• Rescriptor® (delvaridine) ViiV Healthcare

• Sustiva® (efavirenz) Bristol-Myers Squibb

• Viramune® () Boehringer Ingelheim Pharmaceuticals

• Viramune®XR™ (nevirapine extended-release) Boehringer Ingelheim Pharmaceuticals

Medicines in Development HIV/AIDS 2012 9 Approved Medicines for HIV Infection/AIDS

Nucleotide Reverse Transcriptase Inhibitor (NtRTI)

• Viread® ( fumarate) Gilead Sciences

protease Inhibitors

• Agenerase® () GlaxoSmithKline, Vertex Pharmaceuticals

• Aptivus® () Boehringer Ingelheim Pharmaceuticals

• Crixivan® () Merck

• Fortovase® ( soft-gel) Roche, marketing discontinued

• Invirase® (saquinavir) Genentech

• Kaletra® (lopinavir/ritonavir) Abbott Laboratories

• Lexiva® (fosamprenavir) ViiV Healthcare, Vertex Pharmaceuticals

• Norvir® (ritonavir) Abbott Laboratories

• Prezista® (darunavir) Janssen Therapeutics

• Reyataz® () Bristol-Myers Squibb

• Viracept® () ViiV Healthcare

Combination Medicines

NNRTI/NRTI/NtRTI

• Atripla® (efavirenz/emtricitabine/tenofovir disoproxil fumarate) Bristol-Myers Squibb, Gilead Sciences

NRTI/NNRTI/NtRTI

• Complera™ (emtricitabine/rilpivirine/tenofovir, disoproxil fumarate) Gilead Sciences

Integrase Inhibitor/PK Enhancer/NRTI/NtRTI

• Stribild™ (elvitegravor/cobicistat/emtricitabine/tenofvoir disoproxil fumarate) Gilead Sciences

NRTI/NtRTI

• Truvada® (emtricitabine/tenofovir disoproxil fumarate) Gilead Sciences

10 Medicines in Development HIV/AIDS 2012 Glossary

application submitted—An application for in the virus’ replication, blocking it can halt promising drugs by establishing very early on marketing has been submitted by the company further spread of the virus. whether the agent behaves in human subjects to the Food and Drug Administration (FDA). as was anticipated from preclinical studies. PK enhancer—Pharmacokinetic (PK) en- —Unlike other HIV drugs that hancer increases the effectiveness of pharma- Phase I—Researchers test the drug in a small work after HIV has entered the human immune ceutical treatment. group of people, usually between 20 and 80 cell, entry inhibitors work outside the CD4 healthy adult volunteers, to evaluate its initial reverse transcriptase inhibitor (RTI)—When cell, blocking the virus from entering the cell. safety and tolerability profile, determine a HIV infects a cell, reverse transcriptase The process of HIV entry into a cell requires a safe dosage range, and identify potential side changes the single-stranded RNA into a series of steps in sequence involving several effects. double-stranded viral DNA. The new viral DNA key proteins. Different entry inhibitors target is then integrated into the human DNA cells, al- Phase II—The drug is given to volunteer separate proteins in the process. One type of lowing reproduction of the virus. RTIs block this patients, usually between 100 and 300, to see entry inhibitor blocks the attachment of the HIV action and prevent completion of synthesis of if it is effective, identify an optimal dose, and to protein gp120 to CD4 cell receptors on the cell the double-stranded viral DNA, preventing HIV further evaluate its short-term safety. surface. Another inhibitor targets the binding from multiplying. RTIs are a class of antiretro- of the virus to CCR5 or CXCR4 co-receptors Phase III—The drug is given to a larger, more viral drugs. involved in the virus entering the cell. And a diverse patient population, often involving be- third entry inhibitor interferes with the fusion of NRTI—Nucleoside reverse transcriptase tween 1,000 and 3,000 patients (but sometime the HIV virus with T-cells at the cell membrane. inhibitor. many more thousands), to generate statistically significant evidence to confirm its safety and HIV infection—The presence of antibodies in NNRTI—Non-nucleoside reverse transcriptase effectiveness. They are the longest studies, the blood to the human immunodeficiency virus inhibitor. and usually take place in multiple sites around (the virus that causes AIDS). HIV-1 refers to the world. the most common strain of the virus found in NtRTI—Nucleotide reverse transcriptase U.S. AIDS patients. inhibitor. PI—Protease inhibitors are a class of antiret- roviral drugs used to treat HIV infection. They integrase inhibitor— A class of antiretroviral Phase 0—First-in-human trials conducted prevent the HIV virus from replicating by inhib- drugs designed to block the action of integrase, in accordance with FDA’s 2006 guidance on iting the activity of proteases, such as HIV-1. an that inserts the virus into the DNA exploratory Investigational New Drug (IND) of human cells. Since integration is a vital step studies designed to speed up development of

Medicines in Development HIV/AIDS 2012 11 Selected Facts about HIV/AIDS

Overview

U.S. AIDS Diagnoses through 20101 U.S. AIDS Deaths through 20091

Adults/Adolescents 1,119,651 614,394

Pediatric (under age 13) 9,475 4,986

TOTAL 1,163,575* 641,976*

* Because totals for the estimated numbers were calculated independently of the values for the subpopulations, the subpopulation values may not equal these totals.

HIV/AIDS Worldwide 2

• In 2010, 2.7 million people became newly infected with HIV infection (including 390,000 children younger than age 15), down from 3.1 million in 2001. Although the annual number of people newly infected with HIV has dropped since its peak in the late 1990s, it is still occur- ring at an unacceptably high rate: between 2.5 million and 3 million people annually for the past five years, adding to the global number of people living with HIV that reached 34 million (including 3.4 million children younger than age 15) by the end of 2010.

• Globally, the annual number of people newly infected with HIV continues to decline, although there is stark regional variation. In sub-Saharan Africa, where most of the people newly infected with HIV live, an estimated 1.9 million people became infected in 2010. That was 16 percent fewer than the estimated 2.2 million people newly infected with HIV in 2001, and 27 percent fewer than the annual number of people newly infected between 1996 and 1998, when the incidence of HIV in sub-Saharan Africa peaked overall.

• Reductions in the number of people acquiring HIV infection, especially people ages 15–24 in the countries in sub-Saharan Africa that have a high burden of HIV, have been offset by increases in new infections in Eastern Europe and Central Asia. In those areas, where the primary mode of transmission of HIV is among people who inject drugs and their sexual networks, the number of people dying from AIDS-related causes increased 1,100 percent during the past decade: from an estimated 7,800 in 2001 to 89,500 in 2010.

• The annual number of people dying from AIDS-related causes worldwide is steadily decreasing from a peak of 2.2 million in 2005 to an estimated 1.8 million in 2010. That year, an estimated 250,000 children younger than age 15 died from AIDS-related causes, 20 percent fewer than in 2005. The number of people dying from AIDS-related causes began to decline in 2005–2006 in sub-Saharan Africa, South and Southeast Asia and the Caribbean and has continued subsequently.

• Introducing antiretroviral therapy has averted 2.5 million AIDS deaths in low- and middle-income countries globally since 1995. Sub- Saharan Africa accounts for the vast majority of the averted deaths: about 1.8 million.

• Providing antiretroviral prophylaxis to pregnant women living with HIV has prevented more than 350,000 children from acquiring HIV infection since 1995. Eighty-six percent of the children who avoided infection live in sub-Saharan Africa, the region with the highest prevalence of HIV infection among women of reproductive age.

12 Medicines in Development HIV/AIDS 2012 Selected Facts about HIV/AIDS

HIV/AIDS in the United States1

• In 2010, an estimated 48,298 people were newly diagnosed with HIV infection in the 46 states and 5 U.S. dependent areas with confidential name-based HIV infection reporting. In the 46 states, 46,912 adults and adolescents were newly diagnosed with HIV infection, with 37,045 diag- noses in males and 9,868 diagnoses in females. Among children younger than age 13, there were an estimated 217 diagnoses of HIV infection.

• At the end of 2009, an estimated 1,148,200 people age 13 and older were living with HIV infection in the United States, including 207,600 people whose infections had not been diagnosed.

• In 2009, the estimated number of deaths of people with a diagnosis of HIV infection in the 46 states and 5 U.S. dependent areas with confidential name-based HIV infection reporting was 21,601. In the 46 states only, that included 21,007 adults and adolescents and 8 children younger than age 13.

• In 2010, the estimated number of people diagnosed with AIDS in the United States and 6 U.S. dependent areas was 33,630. In the 50 states and the District of Columbia, 24,749 AIDS diagnoses were among adult and adolescent males, 8,242 were among adult and adolescent females, and 23 diagnoses were among children younger than age 13.

• In 2009, the estimated number of deaths of people with an AIDS diagnosis in the United States and 6 U.S. dependent areas was 18,234. In the 50 states and the District of Columbia, that included 17,770 adults and adolescents and 4 children younger than age 13.

HIV/AIDS Economic Impact

• The lifetime treatment cost of an HIV infection can be used as a conservative threshold value for the cost of averting one infection. Currently, the lifetime treatment cost of an HIV infection is estimated at $379,668 (in 2010 dollars); therefore, a prevention intervention is deemed cost-saving if its cost-effectiveness (CE) ratio is less than $379,668 per infection averted. The average annual cost of HIV care in the antiretroviral (ART) era is estimated to be $19,912 (in 2006 dollars; $23,000 in 2010 dollars). One study has estimated the medical savings from infections averted by United States prevention programs from 1991-2006 to be $129.9 billion with 361,878 HIV infections averted.1

• Nearly 30 years into the HIV epidemic, HIV continues to take a heavy toll in the United States. More than 1.1 million people are currently living with HIV, nearly 18,000 people with AIDS still die each year, and lifetime medical care for those who become infected with HIV each year is estimated to cost $20 billion.1

• Without intervention, a perinatal HIV transmission rate of 25 percent would result in 1,750 HIV-infected infants born annually in the United States with lifetime medical costs estimated to be $282 million. The cost of intervention (HIV counseling, testing, and zidovu- dine treatment) was estimated to be $67.6 million. That intervention would prevent 656 pediatric HIV infections, saving $105.6 million in medical care costs—a net cost-savings of $38.1 million annually.3

Sources:

1. U.S. Centers for Disease Control and Prevention, HIV Surveillance Report: Diagnoses of HIV Infection and AIDS in the United States and Dependent Areas, 2010; Vol. 22., www.cdc.gov 2. World Health Organization (WHO), www.who.int/en 3. KidSource OnLine, Inc., www.kidsource.com

Medicines in Development HIV/AIDS 2012 13 The , Development and Approval Process

Developing a new medicine takes an average of 10-15 years; For every 5,000-10,000 compounds in the pipeline, only 1 is approved.

The and Approval Process The U.S. system of new drug approvals is in people. The IND shows results of previous statistically significant evidence to confirm its perhaps the most rigorous in the world. experiments; how, where and by whom the safety and effectiveness. They are the longest new studies will be conducted; the chemical studies, and usually take place in multiple sites It takes 10-15 years, on average, for an structure of the compound; how it is thought around the world. experimental drug to travel from lab to U.S. to work in the body; any toxic effects found in patients, according to the Tufts Center for the New Drug Application (NDA)/Biologic the animal studies; and how the compound Study of Drug Development. Only five in 5,000 License Application (BLA). Following the is manufactured. All clinical trials must be compounds that enter preclinical testing make completion of all three phases of clinical trials, reviewed and approved by the Institutional it to human testing. And only one of those five a company analyzes all of the data and files an Review Board (IRB) where the trials will be is approved for sale. NDA or BLA with FDA if the data successfully conducted. Progress reports on clinical trials demonstrate both safety and effectiveness. On average, it costs a company $1.2 billion, must be submitted at least annually to FDA and The applications contain all of the scientific including the cost of failures, to get one new the IRB. information that the company has gathered. medicine from the laboratory to U.S. patients, Clinical Trials, Phase I—Researchers test Applications typically run 100,000 pages or according to a 2007 study by the Tufts Center the drug in a small group of people, usually more. for the Study of Drug Development. between 20 and 80 healthy adult volunteers, to Approval. Once FDA approves an NDA or Once a new compound has been identified in evaluate its initial safety and tolerability profile, BLA, the new medicine becomes available the laboratory, medicines are usually devel- determine a safe dosage range, and identify for physicians to prescribe. A company must oped as follows: potential side effects. continue to submit periodic reports to FDA, Preclinical Testing. A pharmaceutical com- Clinical Trials, Phase II—The drug is given including any cases of adverse reactions and pany conducts laboratory and animal studies to volunteer patients, usually between 100 and appropriate quality-control records. For some to show biological activity of the compound 300, to see if it iseffective, identify an optimal medicines, FDA requires additional trials against the targeted disease, and the com- dose, and to further evaluate its short-term (Phase IV) to evaluate long-term effects. pound is evaluated for safety. safety. Discovering and developing safe and effective Investigational New Drug Application (IND). Clinical Trials, Phase III—The drug is given to new medicines is a long, difficult, and expensive After completing preclinical testing, a company a larger, more diverse patient population, often process. PhRMA member companies invested files an IND with the U.S. Food and Drug involving between 1,000 and 3,000 patients (but an estimated $49.5 billion in research and Administration (FDA) to begin to test the drug sometime many more thousands), to generate development in 2011.