FDA Clears Test To Identify Organisms that Cause Bloodstream Infections and Provide Antibiotic Sensitivity Results

 

Bacterial or yeast blood infections can occur in patients of all ages, but are particularly severe in infants, the elderly and those with weakened immune systems. If not treated rapidly, such bloodstream infections can lead to severe complications, such as septic shock and death.

 

The FDA has marketing clearance of the PhenoTest BC Kit, performed on the Pheno System. This is the first test to identify organisms that cause bloodstream infections and provide information about which antibiotics the organism is likely to respond to (antibiotic sensitivity). The test also reduces the amount of time it takes to provide this important information, which can guide antibiotic treatment recommendations more quickly. Unlike traditional identification and antibiotic susceptibility tests that may take 24 to 48 hours after detection in a positive blood culture to provide test results, the PhenoTest BC Kit can identify bacteria or yeast from a positive blood culture in approximately 1.5 hours. For certain organisms, the test also provides important information to guide treatment recommendations in approximately 6.5 hours after the organisms are detected from blood cultures.

 

The test can identify 14 different species of bacteria and two species of yeast that cause bloodstream infections, while also providing antibiotic sensitivity information on 18 selected antibiotics for a subset of the identified organisms as appropriate. The test can also identify the presence of two indicators of antibiotic resistance, which can occur when potentially harmful bacteria change in a way that reduces or eliminates the effectiveness of antibiotics.

 

The PhenoTest BC Kit works by measuring the similarity of the infection-causing organism’s genetic material to DNA known to be unique to specific bacteria or yeast. Once the organism is identified, it is mixed with antibiotics and the growth of the bacteria is measured by time-lapse images. If the organism does not grow when the antibiotic is present, this means that an antibiotic can possibly be used for treatment. The FDA reviewed the data for the PhenoTest BC Kit through the de novo premarket review pathway, a regulatory pathway for devices of a new type with low-to-moderate-risk that are not substantially equivalent to an already legally marketed device and for which special controls can be developed, in addition to general controls, to provide a reasonable assurance of safety and effectiveness of the devices. The FDA’s decision to allow marketing was based largely on its review of the sponsor’s primary clinical study of 1,850 positive blood cultures. In this study, the PhenoTest BC Kit provided correct identification of the bacteria or yeast in the positive blood culture more than 95% of the time. Results for testing whether the bacteria were sensitive to antibiotics were also accurate when compared to traditional tests.

 

Risks associated with use of the PhenoTest BC Kit include false positive findings, which can occur when an individual not infected with organisms that cause bloodstream infections receives a test result that incorrectly indicates that he or she is infected. Results obtained from the test should always be interpreted alongside additional laboratory test results. The PhenoTest BC Kit and the Pheno System are manufactured by Accelerate Diagnostics Inc. in Tucson, Arizona.

FDA Approves Siliq to Treat Psoriasis

 

Psoriasis is a skin condition that causes patches of skin redness and flaking. Psoriasis is an autoimmune disorder that occurs more commonly in patients with a family history of the disease, and most often begins in people between the ages of 15 and 35. The most common form of psoriasis is plaque psoriasis, in which patients develop thick, red skin with flaky, silver-white scales.

 

The FDA has approved Siliq (brodalumab) to treat adults with moderate-to-severe plaque psoriasis. Siliq’s active ingredient (brodalumab) binds to a protein that causes inflammation, inhibiting the inflammatory response that plays a role in the development of plaque psoriasis. The drug is intended for patients who are candidates for systemic therapy (treatment using substances that travel through the bloodstream, after being taken by mouth or injected) or phototherapy (ultraviolet light treatment), and have failed to respond, or have stopped responding to other systemic therapies.

 

Siliq’s safety and efficacy were established in three randomized, placebo-controlled clinical trials with a total of 4,373 adult participants with moderate-to-severe plaque psoriasis who were candidates for systemic therapy or phototherapy. More patients treated with Siliq compared to placebo had skin that was clear or almost clear, as assessed by scoring of the extent, nature and severity of psoriatic changes of the skin.

 

Suicidal ideation and behavior, including completed suicides, have occurred in patients treated with Siliq during clinical trials. Siliq users with a history of suicidality or depression had an increased incidence of suicidal ideation and behavior compared to users without this history. A causal association between treatment with Siliq and increased risk of suicidal ideation and behavior has not been established. Because of the observed risk of suicidal ideation and behavior, the labeling for Siliq includes a Boxed Warning and the drug is only available through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Siliq REMS Program. Notable requirements of the Siliq REMS Program include the following:

 

Prescribers must be certified with the program and counsel patients about this risk. Patients with new or worsening symptoms of depression or suicidality should be referred to a mental health professional, as appropriate.

Patients must sign a Patient-Prescriber Agreement Form and be made aware of the need to seek medical attention should they experience new or worsening suicidal thoughts or behavior, feelings of depression, anxiety or other mood changes.

Pharmacies must be certified with the program and must only dispense to patients who are authorized to receive Siliq.

 

Siliq is also approved with a Medication Guide to inform patients of the risk of suicidal ideation and behavior, and that because Siliq is a medication that affects the immune system, patients may have a greater risk of getting an infection, or an allergic or autoimmune condition. Patients with Crohn’s disease should not use Siliq. Health care providers should also evaluate patients for tuberculosis (TB) infection prior to initiating treatment with Siliq. Health care providers should not administer Siliq to patients with active TB infection, and should avoid immunizations with live vaccines in patients being treated with Siliq.

 

The most common adverse reactions reported with the use of Siliq include joint pain (arthralgia), headache, fatigue, diarrhea, throat pain (oropharyngeal pain), nausea, muscle pain (myalgia), injection site reactions, influenza, low white blood cell count (neutropenia) and fungal (tinea) infections. Siliq is marketed by Bridgewater, New Jersey-based Valeant Pharmaceuticals.

FDA Approves Drug to Treat Duchenne Muscular Dystrophy

 

Duchenne muscular dystrophy (DMD) is the most common type of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. The first symptoms are usually seen between 3 and 5 years of age and worsen over time. The disease often occurs in people without a known family history of the condition and primarily affects boys, but in rare cases it can affect girls. DMD occurs in about one of every 3,600 male infants worldwide. People with DMD progressively lose the ability to perform activities independently and often require use of a wheelchair by their early teens. As the disease progresses, life-threatening heart and respiratory conditions can occur. Patients typically succumb to the disease in their 20s or 30s; however, disease severity and life expectancy vary.

 

The FDA approved Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 years and older with DMD. Emflaza is a corticosteroid that works by decreasing inflammation and reducing the activity of the immune system and this class of drugs are commonly used to treat DMD across the world. This is the first FDA approval of any corticosteroid to treat DMD and the first approval of deflazacort for any use in the United States. The effectiveness of deflazacort was shown in a clinical study of 196 male patients who were 5 to 15 years old at the beginning of the trial with documented mutation of the dystrophin gene and onset of weakness before age 5. At week 12, patients taking deflazacort had improvements in a clinical assessment of muscle strength across a number of muscles compared to those taking a placebo. An overall stability in average muscle strength was maintained through the end of study at week 52 in the deflazacort-treated patients. In another trial with 29 male patients that lasted 104 weeks, deflazacort demonstrated a numerical advantage over placebo on an assessment of average muscle strength. In addition, although not statistically controlled for multiple comparisons, patients on deflazacort appeared to lose the ability to walk later than those treated with placebo.

 

The side effects caused by Emflaza are similar to those experienced with other corticosteroids. The most common side effects include facial puffiness (Cushingoid appearance), weight gain, increased appetite, upper respiratory tract infection, cough, extraordinary daytime urinary frequency (pollakiuria), unwanted hair growth (hirsutism) and excessive fat around the stomach (central obesity). Other side effects that are less common include problems with endocrine function, increased susceptibility to infection, elevation in blood pressure, risk of gastrointestinal perforation, serious skin rashes, behavioral and mood changes, decrease in the density of the bones and vision problems such as cataracts. Patients receiving immunosuppressive doses of corticosteroids should not be given live or live attenuated vaccines.

 

The FDA granted this application fast track designation and priority review. The drug also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The sponsor is receiving a rare pediatric disease priority review voucher under a program intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. A voucher can be redeemed by a sponsor at a later date to receive priority review of a subsequent marketing application for a different product. This is the ninth rare pediatric disease priority review voucher issued by the FDA since the program began. Emflaza is marketed by Marathon Pharmaceuticals of Northbrook, Illinois.

Newborn Screening System for 4 Rare Metabolic Disorders

 

Target Health is very pleased that it worked closely with Protalix Biotherapeutics for the approval of Taliglucerase alpha for the treatment of Gauche disease, we and continue to work with Protalix in Fabry disease.

 

Lysosomal Storage Disorders (LSDs) are a group of rare, inherited metabolic disorders in which enzymes (proteins) that normally eliminate unwanted substances in the body’s cells are not at normal levels or functioning properly. According to the U.S. Department of Health and Human Services’ Advisory Committee on Heritable Disorders in Newborns and Children, MPS I, Pompe, Gaucher and Fabry occur in approximately 1 in 1,500 to no more than 1 in 185,000 newborns and children, depending on the disorder. If not detected and treated in a timely manner, these disorders may cause organ damage, neurological disability or death.

 

The FDA has permitted marketing of the Seeker System for the screening of four, rare Lysosomal Storage Disorders (LSDs) in newborns. The Seeker system is designed to detect Mucopolysaccharidosis Type I (MPS I), Pompe, Gaucher and Fabry. It is the first newborn screening test permitted to be marketed by the FDA for these disorders.

 

Alberto Gutierrez, Ph.D., director of the Office of In Vitro Diagnostics and Radiological Health in CDRH, said that the Secretary of HHS s recently added Pompe and MPS I to the list of routine recommended newborn screening programs, and it is anticipated that additional states will begin requiring use of screening tests to detect these disorders. Several states currently mandate LSD screening in all newborns, including Arizona, Illinois, Kentucky, Michigan, Missouri, New Jersey, New Mexico, New York, Ohio, Pennsylvania and Tennessee. However, there were there were no FDA-authorized devices for screening of these disorders. Availability of the Seeker System provides laboratories with a screening tool that has been reviewed by the FDA for clinical and analytical validity.

 

The Seeker System, consisting of the Seeker LSD Reagent Kit- IDUA|GAA|GBA|GLA and Seeker Instrument, works by measuring the activity level of proteins required for healthy lysosomal storage found in dried blood samples collected from the prick of a newborn’s heel 24 to 48 hours after birth. The Seeker Instrument is a device that automates the analysis of dried blood spots. Reduced enzyme activity of proteins associated with any of the four LSDs detected by the kit may indicate presence of a disorder. Results showing reduced enzyme activity must be confirmed using other testing methods, such as biopsies, genetic and other laboratory tests.

 

The FDA reviewed the data for the Seeker System through the de novo premarket review pathway, a regulatory pathway for devices of a new type with low-to-moderate-risk that are not substantially equivalent to an already legally marketed device and for which special controls can be developed, in addition to general controls, to provide a reasonable assurance of safety and effectiveness of the devices. During this process, the FDA evaluated data from a clinical study of 154,412 newborns in Missouri whose dried blood samples were tested for protein activity associated with MPS I, Pompe, Gaucher and Fabry. Efficacy was determined because the system was able to accurately identify at least one of each of these four LSDs in 73 of the screened newborns. Risks associated with use of the screening system include false negative findings.

 

As part of this study, the Missouri State Public Health Laboratory conducted active surveillance of four of the state’s metabolic clinical centers for new diagnoses of these disorders. The state laboratory’s surveillance activities extended 15 months following the study’s completion to determine cases of false negatives that had not been identified during the study. No false negative results were identified either through the study or the state’s 15-month surveillance program.

 

The Seeker System was created with funding from the Small Business Innovation Research program in National Institutes of Health’s Eunice Kennedy Shriver National Institute of Child Health and Human Development. It is manufactured by Baebies Inc., located in Durham, North Carolina.

Elevated Levels of Belladonna in Certain Homeopathic Teething Products

 

Homeopathic teething products have not been evaluated or approved by the FDA for safety or effectiveness. The agency is unaware of any proven health benefit of the products, which are labeled to relieve teething symptoms in children. In September 2016, the FDA warned against the use of these products after receiving adverse event reports.

 

The FDA has announced that its laboratory analysis found inconsistent amounts of belladonna, a toxic substance, in certain homeopathic teething tablets, sometimes far exceeding the amount claimed on the label. The agency is warning consumers that homeopathic teething tablets containing belladonna pose an unnecessary risk to infants and children and urges consumers not to use these products.

 

In light of these findings, the FDA contacted Standard Homeopathic Company in Los Angeles, the manufacturer of Hyland’s homeopathic teething products, regarding a recall of its homeopathic teething tablet products labeled as containing belladonna, in order to protect consumers from inconsistent levels of belladonna. At this time, the company has not agreed to conduct a recall. The FDA recommends that consumers stop using these products marketed by Hyland’s immediately and dispose of any in their possession. In November 2016, Raritan Pharmaceuticals (East Brunswick, New Jersey) recalled three belladonna-containing homeopathic products, two of which were marketed by CVS.

 

According to FDA, consumers should seek medical care immediately if their child experiences seizures, difficulty breathing, lethargy, excessive sleepiness, muscle weakness, skin flushing, constipation, difficulty urinating, or agitation after using homeopathic teething products. The FDA also is encouraging health care professionals and consumers to report adverse events or quality problems experienced with the use of homeopathic teething products to the FDA’s MedWatch Adverse Event Reporting program:

 

1. Complete and submit the report online at www.fda.gov/medwatch/report.htm; or

2. Download and complete the form, then submit it via fax at 1-800-FDA-0178.

 

FDA Approves Trulance for Chronic Idiopathic Constipation

 

According to the National Institutes of Health, an estimated 42 million people are affected by constipation. Chronic Idiopathic Constipation (CIC) is a diagnosis given to those who experience persistent constipation and for whom there is no structural or biochemical explanation.

 

The FDA has approved Trulance (plecanatide) for the treatment of CIC in adult patients. Trulance, taken orally once daily, works locally in the upper GI tract to stimulate secretion of intestinal fluid and support regular bowel function.

 

The safety and efficacy of Trulance were established in two 12-week, placebo-controlled trials including 1,775 adult participants. Participants were randomly assigned to receive a placebo or Trulance, once daily. Participants in the trials were required to have been diagnosed with constipation at least six months prior to the study onset and to have less than three defecations per week in the previous three months, as well as other symptoms associated with constipation. Participants receiving Trulance were more likely to experience improvement in the frequency of complete spontaneous bowel movements than those receiving placebo, and also had improvements in stool frequency and consistency and straining.

 

Trulance should not be used in children less than six years of age due to the risk of serious dehydration and should be avoided in patients six years of age to 18 years of age. The safety and effectiveness of Trulance have not been established in patients less than 18 years of age and Trulance should not be used in patients with known or suspected mechanical gastrointestinal obstruction. The most common and serious side effects of Trulance was diarrhea. Patients may experience severe diarrhea. If severe diarrhea occurs, patients should stop taking Trulance and contact their health care provider.

 

Trulance is manufactured by New York, New York-based Synergy Pharmaceuticals Inc.

 

New NCI Drug Formulary Will Expedite Use of Agents In Clinical Trials

 

The National Cancer Institute (NCI) has launched a new drug formulary (the “NCI Formulary”) that will enable investigators at NCI-designated Cancer Centers to have quicker access to approved and investigational agents for use in preclinical studies and cancer clinical trials. The NCI Formulary could ultimately translate into speeding the availability of more-effective treatment options to patients with cancer.

 

The NCI Formulary is a public-private partnership between NCI and pharmaceutical and biotechnology companies. It is also one of NCI’s efforts in support of the Cancer Moonshot, answering Vice President Biden’s call for greater collaboration and faster development of new therapies for patients. The availability of agents through the NCI Formulary will expedite the start of clinical trials by alleviating the lengthy negotiation process — sometimes up to 18 months — that has been required for investigators to access such agents on their own.

 

The NCI Formulary launched today with 15 targeted agents from six pharmaceutical companies:

 

— Bristol-Myers Squibb

— Eli Lilly and Company

— Genentech

— Kyowa Hakko Kirin

— Loxo Oncology

— Xcovery Holding Company LLC

 

The establishment of the NCI Formulary will enable NCI to act as an intermediary between investigators at NCI-designated Cancer Centers and participating pharmaceutical companies, facilitating and streamlining the arrangements for access to and use of pharmaceutical agents. Following company approval, investigators will be able to obtain agents from the available formulary list and test them in new preclinical or clinical studies, including combination studies of formulary agents from different companies. The NCI Formulary leverages lessons learned through NCI’s Cancer Therapy Evaluation Program (CTEP) and the NCI-MATCH trial, a study in which targeted agents from different companies are being tested alone or in combination in patients with genetic mutations that are targeted by these drugs. As the use of genomic sequencing data becomes more common in selecting cancer therapies, requests for access to multiple targeted agents for the conduct of clinical trials are becoming more common.

 

By the end of 2017, NIH expects to have doubled the number of partnerships and drugs available in the NCI Formulary and CTEP staff continue to discuss the NCI Formulary with pharmaceutical companies to make additional proprietary agents available for studies initiated by investigators at NCI-designated Cancer Centers.

 

The Formulary will complement NIH’s plans for another new public-private partnership in oncology, the Partnership to Accelerate Cancer Therapies (PACT). Through PACT, the NIH, U.S. Food and Drug Administration, biopharmaceutical groups in the private sector, foundations, and cancer advocacy organizations will come together to support new research projects to accelerate progress in cancer research as part of the Cancer Moonshot. PACT research will center on the identification and validation of biomarkers of response and resistance to cancer therapies, with special emphasis on immunotherapies. PACT will also establish a platform for selecting and testing combination therapies. PACT is expected to launch in 2017.

 

FDA Approves First Drug For Spinal Muscular Atrophy

 

Spinal muscular atrophy (SMA), is a hereditary disease that causes weakness and muscle wasting because of the loss of lower motor neurons controlling movement. There is wide variability in age of onset, symptoms and rate of progression. SMA types 1 through 4 all result from a single known cause – a deficiency of a protein called SMN, for “survival of motor neuron.“ Deficiency of SMN protein occurs when a mutation is present in both copies of the SMN1 gene one on each chromosome 5. When SMA symptoms are present at birth or by the age of 6 months, the disease is called type 1 SMA (also called infantile onset or Werdnig-Hoffmann disease). Babies typically have generalized muscle weakness, a weak cry and breathing distress. They often have difficulty swallowing and sucking, and don’t reach the developmental milestone of being able to sit up unassisted. Typically these babies have two copies of the SMN2 gene, one on each chromosome 5. Over half of all new SMA cases are SMA type 1. When SMA has its onset between the ages of 7 and 18 months and before the child can stand or walk independently, it is called type 2 or intermediate SMA. Children with type 2 SMA generally have at least three SMN2 genes. Late-onset SMA (also known as types 3 and 4 SMA, mild SMA, adult-onset SMA and Kugelberg-Welander disease) results in variable levels of weakness. Type 3 SMA has its onset after 18 months, and children can stand and walk independently, although they may require assistance. Type 4 SMA has its onset in adulthood, and people are able to walk during their adult years. People with types 3 or 4 SMA generally have between four and eight SMN2 genes, from which a fair amount of full-length SMN protein can be produced.

 

The FDA has approved Spinraza (nusinersen) for use across the range of SMA patients. Spinraza is the first drug approved to treat children and adults with SMA, is administered by injection into the fluid surrounding the spinal cord. The efficacy of Spinraza was demonstrated in a clinical trial in 121 patients with infantile-onset SMA who were diagnosed before 6 months of age and who were less than 7 months old at the time of their first dose. Patients were randomized to receive an injection of Spinraza, into the fluid surrounding the spinal cord, or undergo a mock procedure without drug injection (a skin prick). Twice the number of patients received Spinraza compared to those who underwent the mock procedure. The trial assessed the percentage of patients with improvement in motor milestones, such as head control, sitting, ability to kick in supine position, rolling, crawling, standing and walking.

 

The FDA worked closely with the sponsor during development to help design and implement the analysis upon which this approval was based. The FDA asked the sponsor to conduct an interim analysis as a way to evaluate the study results as early as possible. A total of 82 of the 121 treated patients were eligible for this analysis. Results showed that 40% of patients treated with Spinraza achieved improvement in motor milestones as defined in the study, whereas none of the control patients did. Additional open-label uncontrolled clinical studies were conducted in symptomatic patients who ranged in age from 30 days to 15 years at the time of the first dose, and in presymptomatic patients who ranged in age from 8 days to 42 days at the time of first dose. These studies lacked control groups and therefore were more difficult to interpret than the controlled study, but the findings appeared generally supportive of the clinical efficacy demonstrated in the controlled clinical trial in infantile-onset patients.

 

The most common side effects found in participants in the clinical trials on Spinraza were upper respiratory infection, lower respiratory infection and constipation. Warnings and precautions include low blood platelet count and toxicity to the kidneys (renal toxicity). Toxicity in the nervous system (neurotoxicity) was observed in animal studies.

 

The FDA granted this application fast track designation and priority review. The drug also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The sponsor is also receiving a rare pediatric disease priority review voucher under a program intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. A voucher can be redeemed by a sponsor at a later date to receive priority review of a subsequent marketing application for a different product. This is the eighth rare pediatric disease priority review voucher issued by the FDA since the program began.

 

Spinraza is marketed by Biogen of Cambridge, Massachusetts and was developed by Ionis Pharmaceuticals of Carlsbad, California.

 

21st Century Cures Act: Making Progress on Shared Goals for Patients

 

The following was excerpted from FDA Voice, authored by Robert M. Califf, M.D., FDA Commissioner

 

On 13 December 2016, Today, President Obama signed into law the 21st Century Cures Act, which builds on FDA’s ongoing efforts to advance medical product innovation and ensure that patients get access to treatments as quickly as possible, with continued assurance from high quality evidence that they are safe and effective. The 21st Century Cures Act will greatly improve FDA’s ability to hire and retain scientific experts as one of FDA’s ongoing challenges has been recruiting and retaining the experts needed in specialized areas to meet FDA’s growing responsibilities. This is an especially important need given the tremendous advances in biological sciences, engineering, information technology and data science. Preventive, diagnostic and therapeutic strategies will become more complex with much greater potential for benefit and in some cases greater risk if used without adequate evidence to exclude risks that exceed potential benefits.

 

This new law rightly recognizes that patients should play an essential role in the development of drugs and devices to diagnose and treat their disease, since patients are in a unique position to provide essential insights about what it is like to live with and fight their disease. That’s been FDA’s perspective as well, and it’s why FDA has continued to advance the science of patient input through its patient-focused drug development program and its partner with patients program for medical devices. As it is, 21st Century Cures will enhance these ongoing efforts to better incorporate the patient’s voice into FDA’s decision-making.

 

21st Century Cures will also support FDA’s efforts to modernize and improve efficiency in clinical trial design. This has been an important FDA priority for decades, but exciting new approaches are now available to develop a common understanding of which designs should be used for which clinical issues. In cancer, for example, FDA is already weighing the use of common control trials, which share a control arm, involve multiple different drugs for the same indication, and may even involve different companies. One of the benefits of using a common control arm is that the overall number of patients who need to be recruited and enrolled decreases, thereby optimizing clinical trial resources and potentially shortening the time it takes to get a new study off the ground.

 

Even without the benefit of Cures, patients have been well-served by FDA’s program efficiencies, emphasis on early meetings, and use of expedited pathway programs to speed approval and delivery of new drugs and devices to patients. Rather than passively processing product applications, FDA works to advise companies and inventors from the earliest stages of the development process on the kinds of medical products needed, how to do the necessary research, and how to viably and effectively translate from concept to product. This not only means that important new products will be developed as efficiently as possible but also that medicines and devices with no chance of success are identified much earlier so that money isn’t wasted on futile development. These programs have been embraced by developers of medical products in this country, and they are making a real and positive difference.

 

In the United States, the FDA uses expedited programs (fast track, priority review, accelerated approval, and breakthrough therapy) for drugs and biologics more than comparable drug and biologic regulators in other countries use theirs and as a result FDA is the first to approve a majority of novel drugs compared to foreign counterparts. For devices, this past year was the first full year of operation for FDA’s expedited access pathway (EAP) program, which helps speed the development and availability of certain medical devices that demonstrate the potential to address unmet medical needs for life-threatening or irreversibly-debilitating diseases or conditions. So far, FDA has granted 24 devices access to this program. 21st Century Cures builds on EAP by creating the breakthrough device pathway.

 

The law establishes other new programs as well. For instance, the Limited Population pathway will help streamline the development programs for certain anti-bacterials and anti-fungals intended to treat targeted groups of patients suffering from serious or life-threatening infections where unmet need exists due to lack of available therapies. Approvals of these antimicrobials are expected to rely on data primarily targeting these limited populations. The statement “Limited Population“ will appear prominently next to the drug’s name in labeling, which will provide notice to healthcare providers that the drug is indicated for use in a limited and specific population of patients. The limited population statement, additional labeling statements describing the data, and FDA review of promotional materials, will help assure these drugs are used narrowly to treat these serious and life-threatening infections while additional evidence is generated to assess safety and effectiveness for broader use.

 

21st Century Cures also creates a new program for the development of regenerative medicine products, an important and exciting new field that deserves this special focus. The program designates drugs as regenerative advanced therapies and takes appropriate actions to improve the efficiency of development and to enhance the exchange of information among FDA, researchers and developers. An especially important element of this program is the creation of a research network and a public-private partnership to assist developers in generating definitive evidence about whether their proposed therapies indeed provide clinical benefits that are hoped for.

 

Looking ahead, much still needs to be done to spur product development. There have yet to be successful therapies identified for certain diseases, such as Alzheimer’s disease, where underlying scientific knowledge is still lacking. In addition, we are only at the early stage in building a national evidence generation system based on registries, claims data, and electronic health records that will be a rich source of post-market data and an avenue for conducting more efficient research. Last week, FDA published a consensus of FDA leadership on the use of real world evidence in the New England Journal of Medicine, focusing on the misperception that randomized trials and real world data are incompatible. In fact, the use of randomization within the context of clinical practice will constitute a major advance in evidence generation and we are actively encouraging proposals with this combination of randomized trials conducted in real world practice. Cures provides support for continued exploration of the use of real world evidence in the regulatory context. The law also addresses drug firms providing healthcare economic information to payers and formulary committees. This complex area will require careful delineation of principles to guide information exchange to enable these entities to appropriately assess the value of drugs.

 

With 21st Century Cures, great progress has been made towards our shared goal of advancing regulatory science so that we can continue to speed the discovery, development, and delivery of medical products to prevent and cure disease and improve health while sustaining the evidence framework that enables assurance to the public of the safety and effectiveness of medical products. FDA now stands ready to work with Congress, other federal agencies and the medical products ecosystem to implement these important provisions as we all continue to work on behalf of all Americans to protect and promote public health and promote innovation in this exciting time.

 

FDA Takes Action Against 4 Tobacco Manufacturers for Illegal Sales of Flavored Cigarettes Labeled as Little Cigars or Cigars

 

The Tobacco Control Act, which was passed by Congress and signed by the President in 2009, banned cigarettes containing certain characterizing flavors, such as candy or fruit flavors, to reduce the number of youth who start to smoke and who become addicted to dangerous tobacco products. The FDA began enforcing that provision in September 2009.

 

The FDA has issued warning letters to four tobacco manufacturers: Swisher International Inc., Cheyenne International LLC, Prime Time International Co. and Southern Cross Tobacco Company Inc., for selling flavored cigarettes that are labeled as little cigars or cigars, which is a violation of the Family Smoking Prevention and Tobacco Control Act. The companies received warning letters for products under the “Swisher Sweets,“ Cheyenne,“ “Prime Time“ and “Criss-Cross“ brands in a variety of youth-appealing flavors, including grape, cherry, wild cherry and strawberry.

 

According to FDA, flavored cigarettes appeal to kids and disguise the bad taste of tobacco, but they are just as addictive as regular tobacco products and have the same harmful health effects. Also, because about 90% of adult daily smokers smoked their first cigarette by the age of 18, continued enforcement of the ban on cigarettes with characterizing flavors is vital to protect future generations from a lifetime of addiction.

 

The FDA determined that, although labeled as little cigars or cigars, the products meet the definition of cigarettes in the Tobacco Control Act, because they are likely to be offered to, or purchased by, consumers as cigarettes based on their overall presentation, appearance, and packaging and labeling. Additionally, since the products meet the definition of a cigarette, the FDA determined that the products are adulterated because they contain a natural or artificial characterizing flavor, or misbranded if they only purport to do so.

 

The FDA has requested the manufacturers respond to the warning letters within 15 working days of receiving the letter. Failure to obey federal tobacco law may result in the FDA initiating further action, including, but not limited to, civil money penalties, criminal prosecution, seizure, and/or injunction. The agency expects many of these products to remain available for purchase by consumers at retail establishments while the FDA works with the manufacturers to ensure the products are in compliance with the requirements of the law.

 

Consumers and other interested parties can report a potential tobacco-related violation of the FD&C Act by using the FDA’sPotential Tobacco Product Violation Reporting Form.

 

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