The United States Food and Drug Administration is a federal agency of the Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food safety, tobacco products, caffeine products, dietary supplements, prescription and over-the-counter pharmaceutical drugs (medications), vaccines, biopharmaceuticals, blood transfusions, medical devices, electromagnetic radiation emitting devices (ERED), cosmetics, animal foods & feed and veterinary products.
A medication is a drug used to diagnose, cure, treat, or prevent disease. Drug therapy (pharmacotherapy) is an important part of the medical field and relies on the science of pharmacology for continual advancement and on pharmacy for appropriate management.
Multiple myeloma (MM), also known as plasma cell myeloma and simply myeloma, is a cancer of plasma cells, a type of white blood cell that normally produces antibodies. Often, no symptoms are noticed initially. As it progresses, bone pain, anemia, renal insufficiency, and infections may occur. Complications may include hypercalcemia and amyloidosis.
Chronic myelogenous leukemia (CML), also known as chronic myeloid leukemia, is a cancer of the white blood cells. It is a form of leukemia characterized by the increased and unregulated growth of myeloid cells in the bone marrow and the accumulation of these cells in the blood. CML is a clonal bone marrow stem cell disorder in which a proliferation of mature granulocytes and their precursors is found; characteristic increase in basophils is clinically relevant. It is a type of myeloproliferative neoplasm associated with a characteristic chromosomal translocation called the Philadelphia chromosome.
Fabry disease, also known as Anderson–Fabry disease, is a rare genetic disease that can affect many parts of the body, including the kidneys, heart, brain, and skin. Fabry disease is one of a group of conditions known as lysosomal storage diseases. The genetic mutation that causes Fabry disease interferes with the function of an enzyme that processes biomolecules known as sphingolipids, leading to these substances building up in the walls of blood vessels and other organs. It is inherited in an X-linked manner.
Muscular Dystrophy Association (MDA) is an American nonprofit organization dedicated to supporting people living with muscular dystrophy, ALS, and related neuromuscular diseases. Founded in 1950 by Paul Cohen, who lived with muscular dystrophy, MDA accelerates research, advances care, and works to empower families to live longer and more independent lives. Renowned for The MDA Labor Day Telethon, the annual telecast aired live every Labor Day weekend, with comedian and filmmaker Jerry Lewis as its host and national chairman. Don Rickles, Frank Sinatra, Sammy Davis Jr., Milton Berle, Wayne Newton, Norm Crosby, Don Francisco, Tony Orlando, Johnny Carson, Aretha Franklin, Maureen McGovern, Diana Ross, Angela Lansbury and others have supported MDA over the years. The organization's headquarters is in Chicago, Illinois.
Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy predominantly affecting boys. The onset of muscle weakness typically begins around age four, with rapid progression. Initially, muscle loss occurs in the thighs and pelvis, extending to the arms, which can lead to difficulties in standing up. By the age of 12, most individuals with Duchenne muscular dystrophy are unable to walk. Affected muscles may appear larger due to an increase in fat content, and scoliosis is common. Some individuals may experience intellectual disability, and females carrying a single copy of the mutated gene may show mild symptoms.
Telehealth is the distribution of health-related services and information via electronic information and telecommunication technologies. It allows long-distance patient and clinician contact, care, advice, reminders, education, intervention, monitoring, and remote admissions. Telemedicine is sometimes used as a synonym, or is used in a more limited sense to describe remote clinical services, such as diagnosis and monitoring. When rural settings, lack of transport, a lack of mobility, conditions due to outbreaks, epidemics or pandemics, decreased funding, or a lack of staff restrict access to care, telehealth may bridge the gap as well as provide distance-learning; meetings, supervision, and presentations between practitioners; online information and health data management and healthcare system integration. Telehealth could include two clinicians discussing a case over video conference; a robotic surgery occurring through remote access; physical therapy done via digital monitoring instruments, live feed and application combinations; tests being forwarded between facilities for interpretation by a higher specialist; home monitoring through continuous sending of patient health data; client to practitioner online conference; or even videophone interpretation during a consult.
Expanded access or compassionate use is the use of an unapproved drug or medical device under special forms of investigational new drug applications (IND) or IDE application for devices, outside of a clinical trial, by people with serious or life-threatening conditions who do not meet the enrollment criteria for the clinical trial in progress.
Fast track is a designation by the United States Food and Drug Administration (FDA) of an investigational drug for expedited review to facilitate development of drugs that treat a serious or life-threatening condition and fill an unmet medical need. Fast track designation must be requested by the drug company. The request can be initiated at any time during the drug development process. FDA will review the request and attempt to make a decision within sixty days.
Mantle cell lymphoma (MCL) is a type of non-Hodgkin's lymphoma, comprising about 6% of cases. It is named for the mantle zone of the lymph nodes where it develops. The term 'mantle cell lymphoma' was first adopted by Raffeld and Jaffe in 1991.
The "war on cancer" is the effort to find a cure for cancer by increased research to improve the understanding of cancer biology and the development of more effective cancer treatments, such as targeted drug therapies. The aim of such efforts is to eradicate cancer as a major cause of death. The signing of the National Cancer Act of 1971 by United States president Richard Nixon is generally viewed as the beginning of this effort, though it was not described as a "war" in the legislation itself.
Amyotrophic lateral sclerosis (ALS), also known as motor neurone disease (MND) or Lou Gehrig's disease in the United States, is a rare, terminal neurodegenerative disorder that results in the progressive loss of both upper and lower motor neurons that normally control voluntary muscle contraction. ALS is the most common form of the motor neuron diseases. ALS often presents in its early stages with gradual muscle stiffness, twitches, weakness, and wasting. Motor neuron loss typically continues until the abilities to eat, speak, move, and, lastly, breathe are all lost. While only 15% of people with ALS also fully develop frontotemporal dementia, an estimated 50% face at least some minor difficulties with thinking and behavior. Depending on which of the aforementioned symptoms develops first, ALS is classified as limb-onset or bulbar-onset.
An Emergency Use Authorization (EUA) in the United States is an authorization granted to the Food and Drug Administration (FDA) under sections of the Federal Food, Drug, and Cosmetic Act as added to and amended by various Acts of Congress, including by the Pandemic and All-Hazards Preparedness Reauthorization Act of 2013 (PAHPRA), as codified by 21 U.S.C. § 360bbb-3, to allow the use of a drug prior to approval. It does not constitute approval of the drug in the full statutory meaning of the term, but instead authorizes the FDA to facilitate availability of an unapproved product, or an unapproved use of an approved product, during a declared state of emergency from one of several agencies or of a "material threat" by the Secretary of Homeland Security.
Right-to-try laws are United States state laws and a federal law that were created with the intent of allowing terminally ill patients access to experimental therapies that have completed Phase I testing but have not been approved by the Food and Drug Administration (FDA). Prior to the passage of right to try laws, patients needed FDA approval to use experimental drugs. As of 2018, 41 U.S. states had passed right to try laws. The framers of these laws argue that this allows for individualized treatments that are not permitted under the FDA's current regulatory scheme. The value of these laws was questioned on multiple grounds, including the fact that pharmaceutical manufacturers would have no obligation to provide the therapies being sought. A federal right to try law was passed in May 2018. Very little data is available about the number of patients who have used the right-to-try pathway, but available sources indicate that since the signing of the bill only a handful of patients have used this pathway to access experimental therapies, as most physicians and sponsors prefer the more traditional, FDA approved, Expanded Access route. According to Scott Gottlieb, who served as commissioner of the FDA under President Donald Trump, the FDA had already approved 99% of patient requests for access to experimental drugs, either immediately over the phone or within a few days, prior to the passage of right to try legislation.
Abemaciclib, sold under the brand name Verzenio among others, is a medication for the treatment of advanced or metastatic breast cancers. It was developed by Eli Lilly and it acts as a CDK inhibitor selective for CDK4 and CDK6.
The 21st Century Cures Act is a United States law enacted by the 114th United States Congress in December 2016 and then signed into law on December 13, 2016. It authorized $6.3 billion in funding, mostly for the National Institutes of Health. The act was supported especially by large pharmaceutical manufacturers and was opposed especially by some consumer organizations.
Artificial intelligence in healthcare is the application of artificial intelligence (AI) to copy human cognition in the analysis, presentation, and understanding of complex medical and health care data. It can also augment and exceed human capabilities by providing faster or new ways to diagnose, treat, or prevent disease. Using AI in healthcare has the potential improve predicting, diagnosing and treating diseases. Through machine learning algorithms and deep learning, AI can analyse large sets of clinical data and electronic health records and can help to diagnose the disease more quickly and precisely.
Aid Access is a nonprofit organization that provides access to medication abortion by mail to the United States and worldwide. It describes its work as a harm reduction strategy designed to provide safe access to mifepristone and misoprostol for those able to become pregnant in the United States who may not otherwise have access to abortion or miscarriage management services. People are able to manage their own abortion with remote access to a physician and a help-desk for any questions. The website is available in English, Spanish, and Dutch.
The n-Lorem Foundation is a non-profit organization based in Carlsbad, California, established to develop personalized medicines using antisense RNA in order to treat patients of rare diseases.
