FDA regulation and application submission process for new drugs - Term Paper Example

Download file to see previous pages Developing a drug for wide use such as a breast cancer drug follows a certain method. The development of any medication is test intensive. We send evidence from these tests to prove the drug is safe and effective. Experts will review our data. Once this independent review establishes the products health benefits outweigh the risks, the drug may be approved. Before a drug can be tested in people, we perform laboratory and animal tests to investigate how it works and how likely it is to be safe. The human testing is conducted to investigate effectiveness. Orphan drug development is similar in some ways but different in others.
To apply for orphan drug designation, we must complete a number of additional steps. First, we have to specify the disease. Next, we must submit the appropriate documents including our name and address, our contact person with title, address, and phone number as well as the name of the drug. Furthermore, we need to provide a description of the target disease, the indications for use of the drug, and the reasons why the drug is necessary to treat the disease. We must provide specific scientific rationale including all data from cynical and nonclinical studies and additional sources available whether positive, negative, or inconclusive. Copies of pertinent unpublished and published papers are required. We must document how the disease affects fewer than 200,000 people in the United States. For a drug intended for a disease affecting 200,000 or more people, we must provide rationale why there would be no expectation that costs of research and development of the drug for the indication could be recovered by sales of the drug in the United States. ...
tionale why there would be no expectation that costs of research and development of the drug for the indication could be recovered by sales of the drug in the United States. In essence, we would follow all of the protocols of developing the drug for non-orphan status but with a few additional considerations.
Questions remain as to why it might be prudent to develop the new drug as an orphan. First, there are certain government incentives to following this route. Government provides tax breaks, enhanced patent protection and marketing rights, as well as clinical research financial subsidization. Furthermore, it provides slightly relaxed statistical standards under the assumption that subjects with the disease in question may not be plentiful enough to derive a very large sample for testing. Still, there are ethical considerations. What if more people are helped otherwise We have a social responsibility to distribute a health benefiting drug to as many people who need it as possible. To develop under orphan drug protocol would limit the number breast cancer patients who might benefit from the drug. Although there are operational and financial motivations for developing the drug under the orphan guidelines, they should not necessarily be the only considerations that play into our decision.
Thinking on certain questions about the prudence of following one drug development protocol versus another has been reported here. We have examined how ways to submit a drug application for a novel anticancer molecule showing evidence of efficacy in leiomyosarcoma. We briefly outlined the differences in approaches to develop this product. One approach is that of applying for orphan drug development. The other is that of developing the drug for breast cancer. We also explored certain ...Download file to see next pagesRead More