Question 1: An overview of the Orphan Drug Act.
Research on pharmaceutical products is funded both by the federal government and private investors. This funding goes into the science in the initial stages of developing the product and clinical research and the actual manufacture of the drug. A drug developed to fight a disease affecting a huge proportion of the population can attract huge investment owing to the profit potential that can be attained. Some diseases, however, affect just a small proportion of the population and it might not be possible to develop a drug for such, or if it is developed then the process might drag for a long time. This scenario is mostly common in instances where investors do not see the possibility of reaping much return for their resources. The Congress passed the Orphan Drugs Act, which provides incentives for investment in the pharmaceutical industry for the development of drugs to combat rare diseases.
The incentives this Act offers include funding in the form of grants and contracts from the federal government to conduct clinical trials of orphan drugs, a tax credit of half the cost incurred in clinical testing activities, and seven years exclusive rights to market the orphan drugs effective from the date of marketing approval. These exclusive marketing rights shield rare orphan drug manufactures from competition for that duration of seven years.
Question 2: Key differences between small molecule drugs and biologics.
Differences in small molecule drugs and biologics can best be understood using the dimensions of size and the manufacturing processes involved. Small molecule drugs can be administered orally. These drugs work in the body within cells. Biologics, on the other hand, are substantially larger in size and are administered through injections. They work in the bloodstream and on the surface of cells, as opposed to within the cells.
Small molecule drugs are generally synthesized by the application of chemical processes. On the other hand, biologics are manufactured within specially designed cells. Analysis and purification of small molecule drugs can easily be done using the normal lab tests. This, however, is not the case for biologics as they are composed of diverse molecules which are closely similar to each other, making characterization a lot more difficult.
Question 3: FDAs Breakthrough Designation Program.
FDAs breakthrough designation program was birthed in July 2012. The intention of the program was to expedite the review and testing of drugs for serious, life-threatening diseases and conditions. The program recommends the use of drugs that have an upper hand in the treatment of life-threatening diseases or conditions over existing therapies.
A breakthrough program incorporates all features of the fast track program in addition to FDA guidance on drug testing and development program as well as the commitment of the senior management of the organization.
Question 4: Potential value of conducting a Pre-NDA meeting with the FDA for your product.
You might have incurred millions of dollars just to make sure that your drug is safe and effective. This can only be probable if your product gets approved by the FDA.
A pre-NDA meeting will increase the chances of your product getting approved by the FDA as you get to know the requirements and standards a product should satisfy before it meets their threshold.
Question 5: Advisory Committee Meeting.
Advisory committees come in to offer independent professional advice regarding human and veterinary drugs, vaccines, medical equipment and food.
FDA may choose to have products reviewed during advisory committee meetings so as to keep abreast with the latest developments associated with the product and its implications. FDA may also choose to carry out product review so as to gauge the effectiveness of the product Vis a Vis a new but similar product in the market. Also, product review may be conducted so as to meet new laws developed regarding the product in question.
Question 1: Goals of non-clinical research and identify the various IND enabling studies.
Non-clinical research for any drug is conducted in all phases of the drug development process to ensure that it adheres to all the safety standards. Also, non-clinical research aims at maximizing the chances of success of a product if the process is performed well.
An Investigational New Drug (IND) is required to perform a clinical trial of a drug that has not been approved or that has been approved for a new patient population.
Question 2: An overview of the Pre-IND meeting, including timing and objectives
Meetings with regulatory boards are generally meant to guide researchers on the regulatory standards and to ease the process of drug approval.
Interested parties should seek a meeting with the FDA so as to build rapport and obtain any information regarding drug development. They must surrender an official letter to the respective project management staff, detailing the name of the product, application number, etc.
In addition to pre-IND meetings, the FDA also offers other forums for guidance on development programs. Type A meetings are held to speed up stalled programs. Type C meetings are any other meetings held between the FDA and a sponsor regarding product development.
Question 3: IND maintenance submissions (i.e. IND amendments)
A formal submission should be submitted to the FDA to carry out drug test for those drugs to be administered to humans. The forms for submitting an IND include:
Form 1571 should be submitted in triplicate and should contain information on the name of the sponsor, date of submission, the name of the drug, etc.
Form FDA 1572 should contain the type of information that should be used to a specific application or submission.
Form FDA 3674 should include a report of the results of the tests conducted with human drugs.
Question 4: Different phases of clinical trials and brief summary of the goals of each phase.
Phase one majors on safety and pharmacology of a compound. This is where small doses of a drug are administered to a group of volunteers who are closely watched.
Phase two examines the effectiveness of the compound. This phase seeks to establish the effective dose and the best method of delivery.
In phase three, researchers try to confirm previous findings in a large population. These studies determine the safety and the effective doses of the drugs.
Question 5: Required elements of an orphan designation request and indicate which are considered the key elements
A sponsor submitting an orphan designation request should be able to prove that the new drug has been verified to be clinically superior to the existing drug in the market.
The request should also give a description of the rare disease or condition for which the drug will be tested and an explanation as to why the drug is deemed superior.
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