The establishment of medicinal regulatory agency’s intent is in facilitating and protecting public health interest by ensuring the safety and efficacy evaluation of medicinal products (biologics, devices, drugs) and other pharmaceutical components distributed or marketed for public use. In much respect, public opinion is necessary in regulatory issues, but in some cases, its contents are derivatives of assumptions and not based on scientific or evidence-based research. In the absence of coercion, incompetence and malpractice, the public should be able to confide and trust the regulatory agencies. At the same level of trust and confidence, the regulatory agency should be able to demonstrate and establish a high level of integrity by maintaining a transparent and unbiased regulatory pattern.

The regulatory agency’s review advisory committee, regulatory personnel, etc. makes the most important decision concerning public health in terms of the safety and efficacy of investigational medicinal products or other products. These decisions should be transparent and free from bias and undue influence by all means. Also, the public must understand that all approved medicinal products have risks, and many of the products have very serious risks (Willman, 2000). Perhaps, the debate on whether the regulatory agencies should adopt a liberal or conservative approach or both for the approval process of medicinal products has been an ongoing debate within many agencies over the years. The most interesting concern or public health issue for the lack of a better word is in determining the most effective direction (liberal/conservative or both) that is most beneficial for public health and public interests.

Take for instance, the approval decision made by the FDA regarding a flu medication called Relenza. Relenza was approved by the FDA after the FDA’s advisory committee concluded that Relenza has not been proven to be safe or effective (Willman, 2000). In other words, the FDA approved the drug against the warning and risk-benefit assessment of its own experts. In addition, the FDA withheld essential safety information from Relenza’s labeling that should have alerted and properly advised the medical professional and the end users of the drug (Relenza) (Willman, 2000).

More importantly, all regulatory agencies should maintain an environment where reviewer’s expert opinion are properly vetted and properly evaluated. A work environment that should not coerce or force experts or impose a job-security threat due to the expertise opinion on the credibility issues and evidence-based suspicion of the product’s safety and efficacy claim, arising from the approval or delay process, should not be discouraged and punished unfairly. Such threat was evidence with Dr. Frances Kelsey’s refusal to approve Thalidomide in the US. The Thalidomide catastrophe of the 1950’s was a clear reminder of the dangers in an investigational medicinal product, when vetted scientific evidence for safety and efficacy is lacking or coerced. In 1950’s, Frances Kelsey had preliminary indication that Thalidomide is not safe for pregnant women. The scientific information she had at her possession indicated substantial safety issues with Thalidomide, thus forcing her adamantly to refuse the approval of the drug despite the pressure and job threat she received from both the pharmaceutical company and the public. Apparently, her heroic action saved many lives including unborn children in the US. Thalidomide is a teratogenic compound. Her action sets altruistic standards worth emulating in all level of many regulatory agencies.

Another situation of this kind is the approval of the AZT for the treatment of HIV/AIDS patients in 1987 (Aegis.org, n.d). The death rate of AIDS patients in the US during 1987 was 4,135 (Aegis.org, n.d). After the approval of the AZT, the death rate in 1988 and 1989 was 4,855 and 14,544 respectively (Aegis.org, n.d). Years after the approval of AZT for the treatment of HIV positive patients, researcher concluded that the product was neither safe nor effective in treating HIV or AIDS patients. In fact, experts concluded that AZT treatment contributed in more adverse event and death of HIV/AIDS patients. Perhaps, medicinal product approval process still lack the parity of balance or balanced-approach in protecting public health interests and maintaining better quality of life, in some cases.

Therefore, a conservative approach may encourage the research and development aspect of pharmaceutical companies in identifying safer and efficacious medical products. In addition, it will encourage more scientific innovation, healthier competition, fewer legal litigation, lower costs, etc. associated with drug defects because, with the appropriate balanced-approach, fewer products’ recalls will occur. According to the Walden DVD on “regulatory environment,” the FDA does not approve medicinal products based on safety and effectiveness, but rather, the FDA approves medicinal products based on whether the benefits outweighs the risks (Dombey, Kaitin, & Whitmore, 2013). In contrast, if the risks of any medicinal product is measured by the number of serious adverse events, which may ultimately lead to product’s recalls, pharmaceutical industries may function better under a conservative approach.

Also, it is beneficial to introduce a conditional liberal approach under some rare occasion such as an epidemic situation. Such conditional or provisional approach will require pharmaceutical companies to continue a thorough phase 4 pharmacovigilance clinical research after any provisional or conditional approval process to ensure product’s risks-benefits assessment quality. In addition, the agency should require the users and physicians prescribing such conditional medicinal products to register with the phase 4 surveillance program in order to monitor the product and possible adverse events. Such program is currently in place with Thalidomide prescriptions.

In the developmental aspects of medicinal products, taxpayers are active investors/contributors in the pharmacological research and development. In fact, a significant portion of research and development innovations came from academic institutions, government laboratories or smaller start-ups (Relman, 2007). These institutions receive an enormous amount of grants, tax deductions and private funding (mostly from taxpayers) for research and development. The introduction of the US “Orphan Drug Acts” of 1983 follows the same incentive standards, but for rare diseases.

In this era of scientific discovery, it is possible to classify many diseases under the Orphan Drug Acts. For instance, there are several types of lung cancers; a small cell lung cancer accounts for 15% of lung cancer and squamous cell carcinoma of the lung accounts for 30% of lung cancer (about.com, n.d). Hence, the totality of patients suffering from each cancer type, if less than or equal 200,000 patients, in the US, may be considered an Orphan disease and could be eligible for Orphan act funding for the research and development purposes. In short, the Orphan act has substantially encouraged research and development in many pathological fields and rare diseases.

References

About.com. (n.d). Lung Cancer. Retrieved from http://lungcancer.about.com/od/typeso flungcance1/Types_of_Lung_Cancer.htm.

Aegis.org. (n.d). A Brief History of HIV/AIDS. Retrieved from http://ww1.aegis.org/ topics/timeline/default.asp.

Dombey, S., Kaitin, K., & Whitmore, E. (2013). Regulatory Environment. Retrieved from https://class.waldenu.edu/webapps/portal/frameset.jsp?tab_tab_group_id=_2_1&url= %2Fwebpps%2Fblackboard%2Fexecute%2Flauncher%3Ftype%3DCourse%26id %3D_2099284_1%26url%3D.

Relman, A. S. (2007, July 23). To lose trust, every day. The New Republic, 237(2), 36–42. Retrieved from http://ezp.waldenulibrary.org/login?url=http://search.ebscohost.com/login .aspx?direct=true&db=a9h&AN=25803260&site=ehost-live&scope=site.

Willman, D. (2000). New FDA. How a new policy let to seven deadly drugs. Los Angeles Times. Retrieved from http://www.pulitzer.org/archives/6473