Exploration of Expanded Access of Investigational Products

Question 1

Should the public have a right to investigational treatment outside expanded access, defend your choice? Expanded access or the compassionate use refers to the outside usage of a clinical trial of an investigational medical product that is the medical product that has not been approved by the Food and Drug Administration FDA. Food and Drug Administration Expanded Access is legislation that aims at providing seriously ill patients who have exhausted the available treatments options and thus have no other comparable standard treatment option to gain access to biological agents and investigational drugs. FDA was enacted by the United States Congress to prohibit the sale of misbranded, mislabeled, or adulterated foods and drugs in interstate commerce. According to the author’s opinion, the public should have a right to investigational treatment outside expanded access. Some patients are not eligible for clinical trials and thus outside expanded access can lead to major inventions of medical products. Ordinarily, investigational drugs may be used under controlled trial. Investigational medical products to be used by a patient as part of the clinical trial is preferable since the trial can generate crucial data that may lead to the approval of medical products; a good example is the case of Josh Hardy (Kroll, 2015). Patients with no ongoing clinical trials or are not eligible for any clinical trials can receive the medical product through expanded access when appropriate. As much as the public have the right to investigational drugs, they should strive to balance between the need and patient safety. The right to investigational treatment outside expanded access can lead to the invention of medical products useful for healing rare diseases with no treatment options as the same time can be dangerous to the patients’ health (Mahady, 2015). That is why the balance between patient safety and need should be emphasized. There has been a lot of conflict between FDA and the Abigail Alliance about when is a drug safe for use. Abigail Alliance blames FDA by saying that it should make drugs accessible on the first stage of development. Each person, if she or he shows the willingness  to undertake investigational treatment outside expanded access should be allowed to do so under close check by a highly qualified physician (“Food and Drug Administration,” 2016).

Question 2

What are the implications of expanded access on drug development from the pharma and FDA perspective? Abigail Alliance claims that FDA makes drugs available for terminally ill patients at early stages of testing. The FDA perception on implications of expanded access on drug development is that it can lead to more useful data that may lead approval of the medical products and their improvement. FDA has facilitated access to investigational drugs to treat patients with serious and immediately life-threatening diseases or conditions with no therapeutic alternatives. Expanded access programs are majorly restricted to patients ailing from serious or life-threatening diseases and have exhausted all standard treatment options and are not eligible to enroll in clinical trials since they don’t meet the inclusion criteria. Pharma companies supply investigational drugs at no cost. This is likely to result in financial issues that are likely to face pharmaceutical companies. Pharma has the perception that supply of unapproved drugs can lead to legal acts being taken against them. The pharma and the FDA have in mind that the drug administered for trial and research purposes can benefit some patients, others experience no effect, and some patients can be harmed by the drug. The pharma and the physicians are faced with difficulties when prescribing drugs that are to be used for the first time. Therefore, from fear of overdose they may prescribe less dosage which has no impact on the ailing patients when the same medicine if properly prescribed could offer significant healing (Ross, 2009). Some patients can refuse to take drugs which have not yet been approved as safe for use. Expanded access gives crucial data that help weigh the benefits and risks of the medical product.

Question 3

Are the FDA regulations and guidance in place for expanded access sufficient in both protecting the public’s safety and also addressing the ethical concern of allowing patients with severe or life-threatening diseases the option to have access to an investigational product where the benefits and risks are still being explored? FDA has outlined set of regulations and guidance that govern on how medical products under investigation are being administered to patients with severe, life-threatening conditions. FDA has these regulations in place to ensure a balance between benefits and risk that can be associated with investigational drugs (“Food and Drug Administration,” 2016). According to the opinion of the author, the FDA regulations and guidance set in place for expanded access are sufficient in protecting the public safety and addressing the ethical concerns of patients suffering from severe, life-threatening diseases. For example, the Abigail Alliance once send a proposal to the FDA  suggesting that drugs should be made available to the patients with severe, life-threatening illness for purchase after the earliest testing stage. The FDA declined the proposal, one of the FDA regulations and guidance require three to four stages of medical product testing before it can be sold to the public (Jacobson & Parmet, 2007). The act of rejecting the proposal from Abigail Alliance shows that FDA is committed to ensuring the safety of the public. It is more dangerous to make medical products under testing and research available to the public in its initial stages; this poses more risks as compared to the benefits. Making a drug available to the public at its first stage of development poses a great risk to the public safety. The drug can have great negative impacts than positive impacts and thus harm a large group of people since it is not under controlled testing. FDA rejecting Abigail Alliance proposal proves that its regulations and guidance is sufficient to protect the public and patients with severe life threatening illness.

Cite this page