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About CDER - Report |
U.S. Food and Drug Administration • Center for Drug Evaluation and Research |
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About CDER - Report |
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CDER Report to the Nation: 2001 1 Drug ReviewIndex
MissionWe promote the public health by promptly and efficiently reviewing clinical research and taking appropriate action on the marketing of human drugs in a timely manner. HighlightsMany Americans benefited from last year's timely reviews of new prescription medicines, over-the-counter medicines and their generic equivalents. We met or exceeded all the performance goals we agreed to under legislation authorizing us to collect user fees for drug reviews. We approved 24 new medicines that have never been marketed before in this country and 234 generic versions of existing drugs. We authorized seven medicines to be sold over the counter without a prescription. We conducted 463 foreign and domestic inspections that help protect volunteers for clinical trials from research risks and validate the quality and integrity of data submitted to us. Highlights of new medication options for American consumers include:
Review and approval times. Review time represents the time that we spend examining the application. Approval time represents our review time plus industry's response time to our requests for additional information. Our charts show these times as "medians." The value for the median time is the number that falls in the middle of the group after the numbers are ranked in order. It provides a truer picture of our performance than average time, which can be unduly influenced by a few very long or short times. Actions and filings. An application is "filed" when we determine it is complete and accept it for review. We make a filing decision within 60 days of receiving an application. Approval is one of the actions that we can take once an application is filed. Other actions include seeking more information from the sponsor. There is no direct connection between applications filed in one year and actions in the same year. Filings provide an idea of what the workload in subsequent years will be. New drug statistics on Internet. Other statistics are available on our Web site at http://www.fda.gov/cder/rdmt/default.htm. Standard new drugs. We took 143 actions on standard new drug applications, of which 56 were approvals. These drugs have therapeutic qualities similar to those of already marketed products. We have a goal of reviewing 90 percent of these applications within 10 months. Standard new drugs
Priority new drugs. We took 27 actions on priority new drug applications, of which 10 were approvals. These drugs represent significant improvements compared with marketed products. We have a goal of reviewing 90 percent of these applications within six months. Priority new drug statistics
Priority new drug approvals (N=NME)
New molecular entities. Twenty-four of the new drugs we approved were new molecular entities, and seven received priority reviews. NMEs contain an active substance that has never before been approved for marketing in any form in the United States. New molecular entities
Standard NME statistics
Priority NME statistics
Orphan drugs. Three of the approvals were for "orphan" uses in patient populations of 200,000 or fewer. Sponsors of such products receive inducements that include seven-year marketing exclusivity, tax credit for the product-associated clinical research, research design assistance by FDA and grants of up to $200,000 per year. Orphan drugsWe approved three "orphan" products to treat disorders with patient populations of 200,000 or fewer:
Notable 2001 new drug approvalsLast year's approvals benefited people with cancer, HIV infection, heart disease and other disorders. People with cancerOne of our most important approvals last year was for imatinib mesylate (Gleevec), a new oral treatment for patients with chronic myeloid leukemia, a rare life-threatening form of cancer. We reviewed the application for the drug in two and one-half months and approved it under a special procedure that permits the marketing of important therapies on the basis of their effect on surrogate markers. The sponsor is committed to carry out additional studies demonstrating the drug's long-term safety and effectiveness. The drug was developed for use in a U.S. patient population of fewer than 200,000 and was, therefore, designated an orphan drug. Zoledronic acid (Zometa) is an intravenous bisphonsphonate for the treatment of hypercalcemia of malignancy, the most common life-threatening metabolic complication associated with cancer. This complication, which is characterized by elevated serum calcium levels, affects more than 10 percent of all cancer patients and generally occurs late in the course of the disease. People with heart and circulatory diseaseOne new approval last year for cardiac patients is nesiritide (Natrecor) for the treatment of acute decompensated congestive heart failure. The drug, which was developed with the use of recombinant DNA technology, is a synthetic version of a human hormone that dilates veins and arteries. Perflutren lipid microsphere (Definity) is an ultrasound contrast agent for use with suboptimal echocardiograms. It increases the power of heart ultrasounds and may provide earlier and more definitive diagnoses for millions of difficult-to-image patients at risk for heart disease. The drug is the first and only ultrasound contrast agent in the United States that is non-blood derived, eliminating the risks and concerns associated with the use of blood-based products. Fondaparinux sodium (Arixtra) is for the prophylaxis of deep-vein thrombosis, which may lead to pulmonary embolism after orthopedic surgery for hip fracture, hip replacement and knee replacement. The drug is a synthetic compound and the first in a new class of antithrombotic agents that selectively inhibit Factor Xa. Bosentan (Tracleer) is to improve exercise ability and decrease the rate of clinical worsening in patients with pulmonary arterial hypertension with significant limitation of physical activity. The drug is the first to be approved in a new class of drugs called endothelin receptor antagonists. The disease is a chronic, life-threatening disorder that can severely compromise the function of the lungs and heart. People with HIV and AIDSThe first in a new class of drugs to combat HIV was approved, a once-daily medication that blocks an enzyme involved in the replication of the disease. Tenofovir disoproxil fumarate (Viread) is for the treatment of HIV infection when taken in combination with other antiretroviral agents. The drug is the first nucleotide analogue reverse transcriptase inhibitor approved for the treatment of HIV. The drug works by blocking reverse transcriptase, an enzyme involved in the replication of HIV. As a nucleotide, the drug remains in cells longer than many other antiretroviral drugs, allowing for once-daily dosing. Valganciclovir hydrochloride (Valcyte) treats the eye infection cytomegalovirus retinitis in patients with AIDS. People with arthritisValdecoxib (Bextra), a COX-2 specific inhibitor, is for treating the signs and symptoms of osteoarthritis, adult rheumatoid arthritis and the pain associated with menstrual cramping. People with glaucomaWe approved two ophthalmic solutions for reducing elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension. Bimatoprost (Lumigan) and travoprost (Travatan) treat patients who are intolerant of, insufficiently responsive or inadequately controlled using other intraocular pressure-lowering medications. Glaucoma is a leading cause of preventable blindness. People with mental and neurological disordersZiprasidone hydrochloride (Geodon) is for the treatment of schizophrenia, a life-long illness that strikes men and women in their late adolescence or early 20s, often with multiple relapses and impaired daily functioning. Galantamine hydrobromide (Reminyl) is for the treatment of mild to moderate Alzheimer's disease. We approved two drugs for the acute treatment of migraine with or without aura in adults: almotriptan malate (Axert) and frovatriptan succinate (Frova). Infectious diseasesCaspofungin acetate (Cancidas) is for the treatment of invasive aspergillosis in patients who are refractory to or intolerant of other therapies. The drug is the first in a new class of antifungals and works by attacking the fungal cell wall. Aspergillosis is a life-threatening fungal infection in high-risk patients, especially cancer patients, organ and bone-marrow transplant recipients, and patients with HIV/AIDS. Cefditoren pivoxil (Spectracef) is a cephalosporin antibiotic for the treatment of acute exacerbations of chronic bronchitis, pharyngitis/tonsillitis and uncomplicated skin and skin structure infections in adults and children 12 years and older. Ertapenem sodium (Invanz) is a long-acting injectable antibiotic for treatment of adults with moderate to severe bacterial infections, including complicated intra-abdominal infections, complicated skin and skin structure infections, community-acquired pneumonia, complicated urinary tract infections and acute pelvic infections. People with respiratory diseases and allergiesFormoterol fumarate (Foradil) is a fast-acting, long-lasting bronchodilator for the maintenance treatment of asthma and the prevention of bronchospasm in reversible obstructive airways disease. The drug provides both long-lasting symptom control and rapid bronchodilation from a single product. Desloratadine (Clarinex) treats nasal and non-nasal symptoms of seasonal allergic rhinitis in adults and children 12 years of age and older. The drug is a once-daily, nonsedating antihistamine. People with skin diseasePimecrolimus (Elidel) is the first non-steroid cream for mild to moderate atopic dermatitis in patients 2 years and older. Commonly known as eczema, atopic dermatitis is an itchy skin condition that primarily affects children and may last until the late teen-age years or even for life. People with gastrointestinal disordersMesalamine (Canasa) is a suppository for the treatment of active ulcerative proctitis, an inflammation of the rectum. Budesonide (Entocort EC) treats mild to moderate active Crohn's disease, an inflammatory disease, involving the ileum and/or ascending colon. MenDutasteride (Dutasteride) treats benign prostatic hyperplasia, a non-cancerous enlargement of the prostate gland, which can lead to the manifestation of lower urinary tract symptoms. The pathological changes of this disorder are found in about 50 percent of men in their 50s and up to 90 percent of men in WomenWe approved three contraceptives, including two that deliver continuous doses of the hormones progestin and estrogen in new ways to prevent pregnancy:
Both the ring and the patch have to be removed one week each month when the woman menstruates. We also approved a new low-dose oral contraceptive, drospirenone/ethinyl estradiol (Yasmin). New or Expanded Use ReviewApplications for a new or expanded use, often representing important new treatment options, are formally called "efficacy supplements" to the original new drug application. When studying approved drugs in children (page 15), sponsors often learn new information about the drug's safety and the doses that should be used. An efficacy supplement changes the labeling information to reflect the new discoveries, even if there is not a new or expanded use. Last year we took action on 213 applications for new or expanded uses of already approved drugs. We approved 91, including six that were given priority reviews of six months or less. Three of the approvals were for orphan uses in patient populations of 200,000 or fewer. New or expanded uses (efficacy supplements)
Priority new or expanded uses
Orphan new or expanded uses
Notable 2001 new or expanded use approvalsWe approved capecitabine (Xeloda) when used with docetaxel for the treatment of locally advanced or metastatic breast cancer after failure to respond to anthracycline-containing cancer therapy. The drug was also approved for first-line treatment of metastatic colorectal carcinoma when treatment with fluoropyrimidine therapy alone is preferred. Letrozole (Femara) is for first-line treatment of postmenopausal women with hormone receptor positive or hormone receptor unknown locally advanced or metastatic breast cancer. Verteporfin (Visudyne) is for the treatment of patients with eye disease affecting the central, clearest portion of vision, specifically for those with predominantly classic subfoveal choroidal neovascularization due to macular degeneration, presumed ocular histoplasmosis or pathologic myopia. Pantoprazole sodium (Protonix I.V.) is an injection for the treatment of the pathological hypersecretion of stomach acid associated with Zollinger-Ellison Syndrome. Pediatric ExclusivityThe 2002 Best Pharmaceuticals for Children Act renewed our authority to grant six months of marketing exclusivity to manufacturers who conduct and submit pediatric studies in response to our written requests. It also allows us to collect user fees for reviewing these pediatric supplements and requires they be reviewed as priority supplements. The law also authorizes the federal government to contract for pediatric studies for drugs that lack patent protection or other marketing exclusivity. Pediatric exclusivity has helped us uncover important new dosing and safety information that will help pediatricians and other prescribers use drugs to treat children more confidently. As of March 2002, we had received 300 proposed pediatric study requests from manufacturers and had issued 241 written requests. These studies could potentially involve more than 32,000 children. Pediatric studies have already been conducted on more than 65 drugs. Reports from these studies have been submitted, exclusivity granted to 56 drugs, and new pediatric labeling in 31 as of March 2002. Pediatric exclusivity cumulative statistics
Internet resourceOur pediatric medicine page is at http://www.fda.gov/cder/pediatric/index.htm. Pediatric conditions with approved labelingDrugs with newly developed approved pediatric use information in their labeling are used to treat conditions such as:
Approved information to treat children's illnessesOf the 31 drugs with newly approved pediatric information in their labels, nine had significant changes for dosing, safety or use, and one drug available in several different products was not recommended for pediatric use. The nine with significant changes were:
Approved information recommends against pediatric useNewly approved labeling shows that several products with a specific topical steroid are not recommended for pediatric use. They are: Betamethasone (Diprolene AF, Diprosone Cream, Ointment, Lotion), used to treat corticosteroid-responsive skin disorders, is not recommended in patients younger than 12 because it suppressed adrenal axis activity and caused local adverse reactions, including signs of skin atrophy. Betamethasone/clotrimazole (Lotrisone), a treatment for ringworm infections, is not recommended in patients younger than 17 because it suppressed adrenal axis activity.
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