Sickle Cell Disease and Family Involvement


The purpose of this case study is to provide analysis of sickle cell disease, investigate the involvement of family in making healthcare decisions, and determine the role of grants and FDA regulations in scientific and pharmaceutical advances. Sickle cell disease is considered as one of the most dangerous conditions since the patients diagnosed with it risk not to reach their adulthood even under the conditions of appropriate care and treatment. Therefore, it is necessary to promote awareness of this disease and discover new testing and therapeutic approaches to this illness.

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Guidelines and Reasons Behind the FDA Regulations for Introducing New Pharmaceutical Agents

The major purpose of FDA’s activity is to provide the US citizens with high-quality food and medical products. The introduction of new pharmaceutical agents makes it possible to make the lives of many people better by improving their health condition. However, the process of introducing these agents is not an easy matter (Ciociola, Cohen, Kulkarni, & the FDA-Related Matters Committee of the American College of Gastroenterology, 2014). Therefore, special FDA regulations are needed to make this process easier and allow people to obtain access to new treatment opportunities sooner (Ciociola et al., 2014). Currently, the process of producing a new drug is costly and lengthy.

Thus, the FDA has introduced several new laws to simplify this process. According to Ciociola et al. (2014), the FDA Safety and Innovation Act of 2013 is aimed at enhancing the process of drug approval and making access to new drugs less complicated. Along with making the process of drug approval fasted, the FDA takes care of checking their quality (Ciociola et al., 2014). Therefore, the guidelines and reasons behind the FDA regulations for introducing new pharmaceutical agents are concerned with providing patients with better access to new drugs while making sure that nothing can threaten their health.

The Role that Money and Grants Play in Scientific Advances

Every day, scientists make incredible discoveries that are aimed at making people’s lives better. Their persistent work could give outstanding results if there were no obstacles in the way. Unfortunately, such barriers exist, and the biggest one of them is the financial burden. Over the past five decades, the federal funding of research projects in the US has decreased considerably (Loria, 2015). In 1968, 10% of the budget was given to research, while in 2015, the number was 3% (Loria, 2015). Since researchers cannot perform their work relying solely on their personal resources, they resort to philanthropists and grant programs to obtain financial support. Therefore, it is possible to conclude that the role of money in scientific advances is rather significant.

Scientists need to employ assistants and buy equipment. They perform many hours of work which should be compensated appropriately. They need to rent offices and travel to perform experiments and make tests (Weinberg et al., 2014). All of these activities are impossible to complete without financial support. Therefore, researchers rely their hopes on federal funding and private help from philanthropists. Without money, no scientific advancements can be achieved. Without research breakthroughs, thousands of people may never be cured of their diseases and have a normal lifestyle. Thus, it is crucial to launch grants programs that will help scientists to make valuable discoveries for the future of the world.

The Role and Involvement of Family in Making the Healthcare Decisions

Since sickle cell disease is an inherited condition, the role of the family in making healthcare decisions is the most significant one. The patient’s parents, siblings, and other caregivers should be aware of the disease’s development, complications, and treatment methods in order to reach the best outcomes for the child (Porter, Graff, Lopez, & Hankins, 2014). Family members need to learn all the aspects of the illness to be able to make correct and beneficial healthcare decisions for their child.

The most common concerns of family members are associated with the transition from pediatric to adult care (Porter et al., 2014). The following issues are frequently discussed in this connection:

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  • the need to change a familiar provider;
  • the barriers and challenges faced during the process of transition;
  • the possibility for patients to manage their condition by themselves;
  • the apprehension of self-management skills being not enough for a quality lifestyle (Porter et al., 2014).

In order to minimize the risks for a patient, family members need to learn about the diagnostic and treatment options. Also, the family should make sure that the patient visits doctors regularly, and they should keep notes of each new test and recommendation.

A crucial aspect of family involvement is presented by learning opportunities (Porter et al., 2014). Parents and caregivers of children diagnosed with sickle cell disease should have access to the information about the illness and should have support from the community and medical institutions in which the child is being treated. Sickle cell disease is not a condition to face alone. Strong support from family and their wise decisions are needed to eliminate the risks and increase the child’s chances to reach adulthood.

The Description of Disease, Its Prevalence, and Incidence

Sickle cell disease is a hematological disorder that impacts millions of people (Ware, de Montalembert, Tshilolo, & Abboud, 2017). The disease is caused by abnormal erythrocytes that have the shape of a sickle (hence the name). These erythrocytes obstruct blood flow in small vessels. Such vaso-occlusion causes inflammation and distal tissue ischemia (Ware et al., 2017). The cause of sickle cell disease is the inherited abnormal beta-globin alleles carrying the sickle mutation on the HBB gene (Ware et al., 2017). The disease is more prevalent in the developing countries and occurs more frequently in the people of Afro-American origin (Ware et al., 2017). Sickle cell disease may cause severe complications such as hypoxia, meningitis, anemia, or organ damage (Ware et al., 2017). Current treatment options include blood transfusion and hydroxycarbamide (Ware et al., 2017). Scientists are working on the development of better diagnostic and treatment methods.

Possible Laboratory Testing

About half of the children having sickle cell disease die before reaching adulthood (Yang et al., 2013). In order to decrease this number, there is an acute need for accurate diagnostic techniques. The best of the current methods of laboratory testing for sickle cell disease is the transcranial Doppler screening (TCD) (Ware et al., 2017). With the help of TCD, it is possible to notice the abnormality in patients’ blood (Naffaa, Tandon, & Irani, 2015). Another method of testing for the disease is magnetic resonance imaging (MRI) (Naffaa et al., 2015). Yang et al. (2013) discuss the future laboratory testing possibilities such as a cheap and fast point-of-care test that will enable physicians to notice the sickle cell carriers in patients’ blood.


Sickle cell disease is one of the most serious life-threatening health conditions the timely diagnosis and effective treatment of which may help to save a person’s life. The role of family members in making healthcare decisions is rather significant. No less important is the development of new testing options and drugs which depends on financial support from the government or philanthropists. Only when the efforts of family, society, and science are combined, is there a possibility to enhance the level of life for patients suffering from sickle cell disease.


Ciociola, A. A., Cohen, L. B., Kulkarni, P., & the FDA-Related Matters Committee of the American College of Gastroenterology. (2014). How drugs are developed and approved by the FDA: Current process and future directions. American Journal of Gastroenterology, 109(5), 620-623.

Loria, K. (2015). One chart shows serious reasons to be concerned about the future of US research. Business Insider. Web.

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Naffaa, L. N., Tandon, Y. K., & Irani, N. (2015). Transcranial Doppler screening in sickle cell disease: The implications of using peak systolic criteria. World Journal of Radiology, 7(2), 52-56.

Porter, J. S., Graff, J. C., Lopez, A. D., & Hankins, J. S. (2014). Transition from pediatric to adult care in sickle cell disease: Perspectives on the family role. Journal of Pediatric Nursing, 29(2), 158-167.

Ware, R. E., de Montalembert, M., Tshilolo, L., & Abboud, M. R. (2017). Sickle cell disease. The Lancet, 390(10091), 311-323.

Weinberg, B., Owen-Smith, J., Rosen, R., Schwartz, L., McFadde-Allen, B., Weiss, R. E., & Lane, J. (2014). Science funding and short-term economic activity. Science, 344(6179), 41-43.

Yang, X., Kanter, J., Piety, N. Z., Benton, M. S., Vignesa, S. M., & Shevkoplyas, S. S. (2013). A simple, rapid, low-cost diagnostic test for sickle cell disease. Lab on a Chip, 13(8), 1464-1467.

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