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Back To Vidyya Report On New Health Care Products Approved In 2000

The FDA Approved A Large Number Of Drugs, Biological Products And Medical Devices In The Past Year


 

Last year, the Food and Drug Administration approved a large number of drugs, biological products and medical devices, many of which represent a impressive advance in the prevention, diagnosis and treatment for serious and life-threatening diseases.

Groups that particularly benefit from the 160 approved medications and medical devices include patients with cancer, children, women, patients with heart disease and the elderly. In addition, FDA's approvals in calendar year 2000 strengthened the surgical armamentarium with several state-of-the-art devices that reduce the risks of complex surgical procedures.

In addition to approving a host of important new medical products in 2000, FDA has continued to exceed virtually all of its performance goals under the Prescription Drug User Fee Act (PDUFA). In Fiscal Years 1999 and 2000, FDA reviewed 100% of new molecular entities (NMEs, or products with ingredients never before marketed in the United States) within their PDUFA goal dates.

Last year, FDA approved 20 products classified as priority drugs -- drugs that have a real benefit beyond existing therapies -- in the median time of 6 months. Moreover, although PDUFA goals specify review times and not approval times, actual approval times have decreased substantially since the program started. Review times denote time FDA spends on an application, while approval times include the time it takes for the applicant to respond to FDA's questions and concerns.

The most important products that passed the FDA's rigorous review for safety and effectiveness last year include the first drug of a new class that addresses an emerging serious public health threat by providing treatment of vancomycin-resistant bacterial infections; a promising new surgical system that incorporates cutting-edge robotics technology; and the first treatment that slows down the progress of a disease that blinds one million Americans each year.

Another notable attribute of last year's results was an unsually high number of pediatric products, which include the first product to treat juvenile rheumatoid arthritis, the first vaccine for meningitis and bacteremia for infants and toddlers; a new drug for children with influenza; and a brand new fetal monitoring system.

In addition, 13 products for a wide spectrum of diseases have been approved for pediatric use. In recent years, Congress and FDA have been stimulating increased pediatric submissions by offering limited pediatric exclusivity to manufacturers who conduct studies for pediatric use of their products.

Listed below are examples of the newly approved products and their potential benefits for various groups of patients:

Cancer patients: Several products approved last year contributed to the prevention, early diagnosis or treatment of cancer, the second deadliest disease in the United States and one that affects eight million Americans.

Three of these products are medical devices. One of them, the Optical Biopsy System, is a laser system that improves a physician's ability to distinguish small harmless growths from pre-cancerous growths in the colon. It is operated through an endoscope and can be used to evaluate polyps less than 1 cm in diameter.

Another advanced technology for early cancer diagnosis is the Senographe 2000D, the first mammography system that produces digital images on a solid state receptor instead of analog images on a radiographic film.

Unlike radiographic film, digital images can be electronically stored and transfered, so that they can be evaluated by a specialist at a remote location. The images also can be manipulated to correct for under- or over-exposure. Early diagnosis remains the best weapon against breast cancer which annually affects 185,000 women, 46,000 of whom die of the disease.

The third new device in this category, the FocalSeal-L Surgical Sealant, was approved for sealing air leaks in lungs following the removal of cancerous tumors. The sealant, which is "painted" on the lung and activated by light, was reviewed by FDA on an expedited basis because of its potential importance for patients with lung cancer.

Among the new cancer medications, two -- Trisenox and Mylotarg -- were approved for cancers of the white blood cells. Trisenox (arsenic trioxide) was found to be safe and effective for the treatment of acute promyelocytic leukemia (APL) in patients who have not responded to, or have relapsed following, all trans-retinoic acid and anthracycline-based chemotherapy. Trisenox, a priority drug, was approved in six months. The product's total development time for this use was only 3 years.

Mylotarg was approved for the treatment of CD33 (an antigen on certain leukemia cells) positive acute myeloid leukemia for patients 60 years or older who have relapsed for the first time and are not suitable candidates for the standard but poorly tolerated cytotoxic therapy.

Nolvadex (tamoxifen citrate), a marketed oncological drug, was approved last year for a new indication of particular significance for women. The drug was approved for use to reduce the risk of invasive breast cancer for women with preinvasive cancer of the ducts.

Other FDA approvals designed to benefit cancer patients included Pacis (BCG Live) for the treatment of cancer-in-situ in the absence of associated invasive cancer of the bladder; Actimmune (interferon gamma-1b) for delaying time to disease progression in malignant osteopetrosis; and Camptosar (irinotecan hydrochloride), which was approved for a new indication as a first-line therapy in combination with 5-fluorouracil and leucovorin (5-FU/LV) to treat metastatic colorectal cancer, a disease that strikes an estimated 130,000 Americans a year. The combination showed a clear survival advantage compared to 5-FU/LV alone.

Children and infants: Several new products approved last year for pediatric and obstetric use were either specifically designed for the youngest patients or were adult drugs with pediatric indications. FDA also approved pediatric use for 13 adult medications that were already on the market.

One new obstetric product is the OxiFirst Fetal Oxygen Saturation Monitoring System, which represents the first major technological development in fetal monitoring in three decades. The system, which builds on pulse oximetry technology, measures oxygen saturation in the baby's blood as a sign of fetal health during labor and delivery.

Another product of benefit to the youngest children is Prevnar, the first multivalent conjugate pneumococcal vaccine for infants and toddlers under the age of two. Prevnar was approved to prevent invasive pneumococcal diseases that can cause brain damage, and, in rare cases, death.

The vaccine was designed to prevent invasive diseases caused by Streptococcus pneumoniae, including bacteremia, an infection of the blood stream that affects about 35,000 infants and toddlers in the U.S., and meningitis, an infection of the lining of the brain or spinal cord that is diagnosed in about 17,500 infants a year.

One- to eight-year old children with asthma can benefit from last year's approval for Pulmicort Respules (budesonide inhalation suspension). The product is the first anti-inflammatory corticosteroid formulated for inhalation using a nebulizer in this age group. This is an important feature because toddlers frequently cannot use metered-dose inhalers.

Another new pediatric drug is Relenza (zanamivir) for the treatment of uncomplicated influenza A and B in patients 7 years and older. Tamiflu (oseltamivir phosphate) oral suspension was approved for prophylaxis of influenza virus in adults and adolescents aged 13 years and older. Influenza causes 20,000 deaths and 100,000 hospitalizations a year.

Drugs approved last year that had both adult and pediatric indications included Kaletra, Unithroid and Protopic.

Kaletra, a combination of lopinavir and ritonavir, received accelerated approval as a protease inhibitor for the treatment of HIV, the human immunodeficiency virus that causes AIDS.

Unithroid (levothyroxine sodium), a thyroid replacement drug, is the first single-ingredient oral levothyroxine product approved for the treatment of hypothyroidism, a disease that causes mental and physical sluggishness, including stunted growth.

Protopic (tacrolimus) is an ointment for patients with moderate or severe eczema for whom standard eczema therapies present potential risks, or who are not adequately treated by or do not tolerate standard eczema therapies.

In addition, FDA last year added pediatric use to the labeling of 13 adult drugs including ibuprofens Motrin and Advil, Zantac (ranitidine) for stomach acid reflux, and Nasalcrom (cromolyn) for asthma and hay fever. The agency also approved lowering the age for the Nucleus 24 Cochlear Implant from 18-month-old to 12-month-old infants who are profoundly hearing impaired.

Women: In addition to the Senographe and Nolvadex, discussed above, FDA approved several other products to treat diseases that either exclusively or predominantly affect women.

For example, Remicade (inflixmab) was approved for the reduction in signs and symptoms of rheumatoid arthritis in patients with inadequate response to methotrexate. Three-quarters of the more than 8 million Americans with rheumatoid arthritis are women.

Another approval that primarily benefits women was for Novantrone (mitoxantrone hydrochloride), a cancer drug, for the treatment of advanced or chronic multiple sclerosis. The disease affects up to 350,000 Americans, 66-75 percent of them women.

In addition, FDA approved Mifeprex (mifepristone) for the termination of pregnancy within 49 days from the beginning of the last menstrual period. The agency also allowed continued marketing of two types of saline-filled breast implants that had been approved for breast augmentation and breast reconstruction in women 18 years or older. The implants decision was made following the conclusion of clinical studies involving 9,000 women.

Cardiac patients and the elderly: Examples of new products for patients with heart disease and the elderly include several novel devices and important medications.

Two of the devices are Cordis Checkmate™ System and Novoste Beta-Cath™ System, both of which use catheters to deliver radiation inside a coronary stent following the reopening of a blocked artery. The radiation helps reduce the risk of new tissue growth inside the coronary stent and the resulting repeated narrowing of the artery.

Another noteworthy new product for people with heart disease is TNKase (tenecteplase), a tissue plasminogen activator produced by recombinant DNA technology. The product was approved for the reduction of mortality in acute myocardial infarction, the major cause of sudden death in adults, which affects more than 350,000 Americans a year.

Among the products designed primarily for the elderly, Visudyne (verteporfin for injection) is of particular importance as the first therapy to slow vision loss in people with the classic type of "Wet Age-Related Macular Degeneration." The disease causes each year blindness of 1 million Americans over the age of 55.

In 2000, FDA also approved the Vibrant Soundbridge, a new type of surgically implanted hearing device, for use in adults with moderate to severe nerve hearing loss who cannot be helped by hearing aids or cochlear implants. About 20% of Americans -- more than 56 million -- experience some nerve deafness by the age of 55.

Other significant approvals of last year included a variety of novel drugs, biological products and medical devices. Here are several examples:

Zyvox (linezolid), the first antibacterial drug in a new class, was approved to treat infections associated with Enterococcus faecium resistant to vancomycin, which for many years has been the treatment of last resort for this organism.

Zyvox was approved for treatment of bloodstream infection, hospital-acquired pneumonia and complicated skin and skin structure infections, and for treatment of community-acquired pneumonia and uncomplicated skin and skin structure infections. These infections are often resistant to multiple antibiotics, and particularly endanger hospitalized or immuncompromised individuals.

Cipro® (ciprofloxacin), an antimicrobial product, was designed to reduce the incidence or progression of inhalational anthrax following exposure to Bacillus anthracis. Inhalational anthrax is an extremely rare disease, usually resulting in industrial setting from exposure to contaminated animal hides and hairs, but the causative organism can be used as a biological weapon.

The Da Vinci Surgical System, a first-of-a-kind robotic device, enables a surgeon to perform laparoscopic gall bladder and reflux disease surgery while sitting at a console with a computer and video monitor and manipulating three robotic arms with hand grips and foot pedals. The product is regarded as the forerunner of new robotic devices that eventually may revolutionize the practice of surgery.

Trufill n-BCA Liquid Embolic System, a new glue for use in a rare form of brain surgery that is performed when the blood vessels in the brain are intertwined. The glue, which is infused via a catheter before surgery, helps reduce the loss of blood by blocking blood vessels in the brain, thereby lessening the patient's risk of stroke and death.

Apligraf, a cellular, bi-layered skin substitute to be used as a new type of wound dressing to help heal diabetic foot ulcers. Open foot sores lead to thousands of amputations each year in people with diabetes.

Myobloc™ (botulinum toxin type B), the first drug to reduce the severity of neck and shoulder muscle contractions and the resulting abnormal head position and neck pain associated with cervical dystonia. Treatment with the product interrupts the cholinergic transmission between the nerve and the affected muscle, causing it to relax.

Malarone, a new combination drug for the prevention and treatment of acute, uncomplicated P. falciparum malaria. A combination of atovaquone and proguanil hydrochloride, Malarone has been shown to be effective in regions where the disease has become resistant to other anti-malarial drugs.

OssaTron, a device that uses shock waves to relieve chronic severe heel pain when conventional treatment has been ineffective. The pain is usually caused by an inflammation of the foot's tissue.

FDA also approved Positron Emission Tomography (PET) F-18 FDG drug as part of an initiative under the FDA Modernization Act of 1997, which included consultations with the public and FDAís advisory committee, a review of literature and an FDA Federal Register notice describing how the drug could be found to be safe and effective for certain indications. F-18 FDG is a radiopharmaceutical drug thatís injected in patients to localize areas with abnormal glucose metabolism to assist in cancer assessments and in identifying reversible cardiac dysfunction.

FDA performance. The products described in this report are only a small sample of the 160 new drugs, biological products and medical devices the agency approved last year. The approval statistics also do not reflect the many other ways in which the Center for Drug Evaluation and Research (CDER), the Center for Biologics Evaluation and Research (CBER), and the Center for Devices and Radiologic Health (CDRH) strengthen public health protection.

These activities include refusal of products not shown to be safe and effective; assuring adequate labeling for products that are approved; and monitoring the effects of marketed drugs. Last year, the three Centers also issued 125 draft and final guidances and other documents to clarify their requirements and facilitate industry's compliance with FDA's demanding standards.

During calendar year 2000, CDER took 238 actions on original new drug applications. Ninety-eight of them were approvals, which were completed in the total median time (including FDA's review and the sponsor's response, if any) of 11.2 months. All but two of these new drugs were processed under PDUFA, which was passed in 1992 and which was reauthorized for five more years in 1997.

In addition to the 20 products classified as priority and approved in the median time of 6 months, FDA approved 27 new molecular entities in the median total time of 15.6 months. Ninety-six percent of the NMEs and 98 percent of all new drugs were approved within their goal dates. The Center also approved eight medications for rare diseases covered by the Orphan Drug Act of 1983.

In addition, CDER received 365 applications for the approval of generic counterparts of original drugs. The Center issued 244 approvals and 61 tentative approvals for such products as Doxazosin Mesylate, the generic equivalent of Cardura for benign prostatic hyperplasia; Paclitaxel, the equivalent of the cancer drug Taxol; and Nifedipine, the equivalent of Adalat CC for hypertension.

CBER again met all of its obligations under PDUFA. The Center took 53 actions, resulting in 13 approvals for product license or biological license applications (PLA/BLAs) in the median time of 25.1 months. Six of the approvals were part of the PDUFA program, and their median approval time was 24.5 months. The 2000 cohort included one priority product, which was approved in 8.5 months.

CDRH in calendar year 2000 took 285 actions on original devices submitted for full evaluation under Product Marketing Applications (PMAs). Forty-nine PMAs were approved in the median approval time of 9.6 months, and four additional products were approved under FDA's Humanitarian Use Exemptions program for devices with a patient population of up to 4,000. These devices were approved in the median time of 3.5 months. In addition, the Center cleared for the market 3,457 so-called 510(k) devices, products that are similar to devices already in use. These notifications were reviewed in the median total time of 2.4 months.

In January 2000, CDRH took over from the Centers of Disease Control and Prevention the responsibility for the implementation of the Clinical Laboratory Improvement Amendments (CLIA) of 1988, which establishes quality standards to ensure the accuracy, reliability and timeliness of laboratory test results for patients. In addition to evaluating laboratory tests for marketing, the Center categorized 1,058 diagnostic tests according to the potential risk to public health, and allowed 158 tests to be conducted in any setting.


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Editor: Susan K. Boyer, RN
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