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2.
Healthc Policy ; 17(3): 81-90, 2022 02.
Article in English | MEDLINE | ID: covidwho-1761263

ABSTRACT

Regulatory and reimbursement decisions for drugs and vaccines are increasingly based on limited safety and efficacy evidence. In this environment, life-cycle approaches to evaluation are needed. A life-cycle approach grants market approval and/or positive reimbursement decisions based on an undertaking to conduct post-market clinical trials that address evidentiary uncertainties, relying on the collection and analysis of post-market data. In practice, however, both conditional regulatory and reimbursement decisions have proven problematic. Here we discuss some of the regulatory implications and unsettled ethical and pragmatic issues, taking lessons from the recent experiences of Israel in rapidly approving the Pfizer-BioNTech COVID-19 vaccine.


Subject(s)
Drug Approval , Insurance, Health , COVID-19/prevention & control , Canada , Drug Approval/legislation & jurisprudence , Humans , Insurance, Health/legislation & jurisprudence
7.
Pharmaceut Med ; 35(4): 203-213, 2021 07.
Article in English | MEDLINE | ID: covidwho-1375861

ABSTRACT

The Emergency Use Authorization (EUA) originated in 2004 because of the need for emergency medical countermeasures (MCMs) against potential bioterrorist attacks. The EUA also proved useful in dealing with subsequent pandemics and has emerged as a critical regulatory pathway for therapeutics and vaccines throughout the Coronavirus Disease 2019 (COVID-19) pandemic. With the EUA process in the USA, we witnessed emergency authorizations, their expansions, as well as withdrawal of previously authorized products, which exemplifies the dynamic nature of scientific review of EUA products. EUAs proved vital for the first group of COVID-19 vaccines, including the temporary pause of one vaccine while emergency safety issues were evaluated. Although this review on the EUA is primarily focused on the USA, distinctions were made with other jurisdictions such as Europe and Canada with respect to the emergency authorizations of the vaccines. Finally, we discuss some important differences following EUA and formal new drug/vaccine application (NDA/BLA) approvals.


Subject(s)
Antiviral Agents/standards , COVID-19 Vaccines/standards , COVID-19/prevention & control , Drug Approval/legislation & jurisprudence , Emergencies/history , Antiviral Agents/administration & dosage , Antiviral Agents/adverse effects , Bioterrorism/history , Bioterrorism/prevention & control , COVID-19/drug therapy , COVID-19/epidemiology , COVID-19 Vaccines/administration & dosage , COVID-19 Vaccines/adverse effects , Canada/epidemiology , Civil Defense/history , Drug Approval/history , Emergencies/epidemiology , Europe/epidemiology , History, 21st Century , Humans , Pandemics/prevention & control , United States/epidemiology
8.
Am J Law Med ; 47(2-3): 157-175, 2021 07.
Article in English | MEDLINE | ID: covidwho-1361581

ABSTRACT

The COVID-19 pandemic has revealed myriad and complex challenges for our national health care system spanning preparedness, response, access, costs, infrastructure, coordination, and medical innovation. These challenges implicate federal, state, and local agencies and actors, as well as international collaborative bodies. One constant throughout the pandemic has been the pressing need for safe and effective diagnostics, prophylactic vaccines, and drug treatments to counter the virus.1 Inarguably, significant problems with the multi-faceted system of drug and vaccine innovation and regulation manifested long before the COVID-19 pandemic.2 The pandemic, however, has laid bare the inextricable connections among federal funding, patents, product review and approval mechanisms, and the eventual medical products and resulting costs.


Subject(s)
Biological Products/economics , COVID-19/drug therapy , Drug Approval/legislation & jurisprudence , Government Agencies , Patents as Topic , Therapies, Investigational/economics , Humans , Information Dissemination , Intellectual Property , Research Support as Topic , SARS-CoV-2 , United States
9.
Br J Cancer ; 125(11): 1477-1485, 2021 11.
Article in English | MEDLINE | ID: covidwho-1360190

ABSTRACT

Important breakthroughs in medical treatments have improved outcomes for patients suffering from several types of cancer. However, many oncological treatments approved by regulatory agencies are of low value and do not contribute significantly to cancer mortality reduction, but lead to unrealistic patient expectations and push even affluent societies to unsustainable health care costs. Several factors that contribute to approvals of low-value oncology treatments are addressed, including issues with clinical trials, bias in reporting, regulatory agency shortcomings and drug pricing. With the COVID-19 pandemic enforcing the elimination of low-value interventions in all fields of medicine, efforts should urgently be made by all involved in cancer care to select only high-value and sustainable interventions. Transformation of medical education, improvement in clinical trial design, quality, conduct and reporting, strict adherence to scientific norms by regulatory agencies and use of value-based scales can all contribute to raising the bar for oncology drug approvals and influence drug pricing and availability.


Subject(s)
Drug Approval , Drug Costs , Medical Oncology/ethics , Antineoplastic Agents/economics , Antineoplastic Agents/therapeutic use , Bias , COVID-19/epidemiology , Cost Control/ethics , Cost Control/organization & administration , Cost Control/standards , Cultural Evolution , Drug Approval/economics , Drug Approval/legislation & jurisprudence , Drug Approval/organization & administration , Drug Costs/ethics , Drug Costs/legislation & jurisprudence , Humans , Medical Oncology/economics , Medical Oncology/organization & administration , Medical Oncology/standards , Neoplasms/drug therapy , Neoplasms/economics , Neoplasms/mortality , Organizational Innovation , Pandemics
12.
Am J Obstet Gynecol ; 225(1): 33-42, 2021 07.
Article in English | MEDLINE | ID: covidwho-1312880

ABSTRACT

Pregnant and lactating women are considered "therapeutic orphans" because they generally have been excluded from clinical drug research and the drug development process owing to legal, ethical, and safety concerns. Most medications prescribed for pregnant and lactating women are used "off-label" because most of the clinical approved medications do not have appropriate drug labeling information for pregnant and lactating women. Medications that lack human safety data on use during pregnancy and lactation may pose potential risks for adverse effects in pregnant and lactating women as well as risks of teratogenic effects to their unborn and newborn babies. Federal policy requiring the inclusion of women in clinical research and trials led to considerable changes in research design and practice. Despite more women being included in clinical research and trials, the inclusion of pregnant and lactating women in drug research and clinical trials remains limited. A recent revision to the "Common Rule" that removed pregnant women from the classification as a "vulnerable" population may change the culture of drug research and drug development in pregnant and lactating women. This review article provides an overview of medications studied by the Obstetric-Fetal Pharmacology Research Units Network and Centers and describes the challenges in current obstetrical pharmacology research and alternative strategies for future research in precision therapeutics in pregnant and lactating women. Implementation of the recommendations of the Task Force on Research Specific to Pregnant Women and Lactating Women can provide legislative requirements and opportunities for research focused on pregnant and lactating women.


Subject(s)
Drug Development , Lactation , Pregnancy , Pregnant Women , COVID-19/drug therapy , COVID-19/prevention & control , COVID-19 Vaccines , Diabetes, Gestational/drug therapy , Drug Approval/legislation & jurisprudence , Drug Development/legislation & jurisprudence , Female , Fetus/drug effects , Humans , Obstetric Labor, Premature/drug therapy , Pre-Eclampsia/drug therapy , Pregnancy/physiology , Pregnancy Complications/drug therapy , Pregnancy Complications/prevention & control , Pregnancy Complications/virology , SARS-CoV-2/immunology , Teratogenesis
13.
J Neurooncol ; 153(3): 375-381, 2021 Jul.
Article in English | MEDLINE | ID: covidwho-1279476

ABSTRACT

OBJECTIVE: Contemporary management of patients with neuro-oncologic disease requires an understanding of approvals by the US Food and Drug Administration (FDA) related to nervous system tumors. To summarize FDA updates applicable to neuro-oncology practitioners, we sought to review oncology product approvals and Guidances that were pertinent to the field in the past year. METHODS: Oncology product approvals between January 1, 2020, and December 31, 2020, were reviewed for clinical trial outcomes involving tumors of the nervous system. FDA Guidances relevant to neuro-oncology were also reviewed. RESULTS: Five oncology product approvals described outcomes for nervous system tumors in the year 2020. These included the first regulatory approval for neurofibromatosis type 1: selumetinib for children with symptomatic, inoperable plexiform neurofibromas. Additionally, there were 4 regulatory approvals for non-central nervous system (CNS) cancers that described clinical outcomes for patients with brain metastases. These included the approval of tucatinib for metastatic human epidermal growth factor receptor 2 (HER2)-positive breast cancer including patients with brain metastases, brigatinib for anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC), and pralsetinib and selpercatinib for RET fusion-positive NSCLC. Finally, two FDA Guidances for Industry, "Cancer Clinical Trial Eligibility Criteria: Brain Metastases" and "Evaluating Cancer Drugs in Patients with Central Nervous System Metastases" were published to facilitate drug development for and inclusion of patients with CNS metastases in clinical trials. CONCLUSIONS: Despite the challenges of the past year brought on by the COVID-19 pandemic, progress continues to be made in neuro-oncology. These include first-of-their-kind FDA approvals and Guidances that are relevant to the management of patients with nervous system tumors.


Subject(s)
Antineoplastic Agents/therapeutic use , Brain Neoplasms/drug therapy , Drug Approval/legislation & jurisprudence , Drug Approval/methods , Humans , United States , United States Food and Drug Administration
15.
AAPS PharmSciTech ; 22(5): 172, 2021 Jun 07.
Article in English | MEDLINE | ID: covidwho-1261286

ABSTRACT

Vaccination development and production was an essential question for the prevention and global control of COVID-19. The strong support from governing authorities such as Operation Warp Speed and robust funding has led to the development and authorization of the tozinameran (BNT162b2) vaccine. The BNT162b2 vaccine is a lipid nanoparticle-encapsulated mRNA that encodes for SARS-CoV-2 spike protein, the main site for neutralizing antibodies. Once it binds with the host cells, the lipid nanoparticles enable the transfer of the RNA, causing S antigens' expression of the SARS-CoV-2, conferring immunity. The vaccine is administered as a 2-dose regime 21 days apart for individuals 16 years and older. Pfizer-BioNTech's BNT162b2 vaccine was the first candidate to receive FDA-Emergency Use Authorization (EUA) on December 11, 2020. During phase 2/3 clinical trials, 95% efficacy was reported among 37,706 participants over the age of 16 who received the BNT162b2 vaccination; additionally, 52% efficacy was noted 12 days following the administration of the first dose of BNT162b2, reflecting early protection of COVID-19. The BNT162b2 vaccine has exhibited 100% efficacy in clinical trials of adolescents between the ages of 12 and 15. Clinical trials in pregnant women and children under the age of 12 are expected to also exhibit promising results. This review article encompasses tozinameran (BNT162b2) vaccine journey, summarizing the BNT162b1 and BNT162b2 vaccines from preclinical studies, clinical trial phases, dosages, immune response, adverse effects, and FDA-EUA.


Subject(s)
COVID-19 Vaccines/administration & dosage , COVID-19/prevention & control , Clinical Trials as Topic/methods , Drug Approval/methods , SARS-CoV-2/drug effects , Animals , Antibodies, Neutralizing/drug effects , Antibodies, Neutralizing/metabolism , COVID-19/epidemiology , COVID-19/metabolism , COVID-19 Vaccines/adverse effects , COVID-19 Vaccines/metabolism , Clinical Trials as Topic/legislation & jurisprudence , Drug Approval/legislation & jurisprudence , Drug Evaluation, Preclinical/methods , Exanthema/chemically induced , Female , Humans , Male , SARS-CoV-2/metabolism , Spike Glycoprotein, Coronavirus/drug effects , Spike Glycoprotein, Coronavirus/metabolism , Vaccination/legislation & jurisprudence , Vaccination/methods
16.
Clin Pharmacol Ther ; 111(3): 551-558, 2022 03.
Article in English | MEDLINE | ID: covidwho-1241501

ABSTRACT

The development of drugs for coronavirus disease 2019 (COVID-19) is a global challenge. In Japan, remdesivir was approved in May 2020 for COVID-19 caused by the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection. In February 2021, a vaccine against COVID-19 was approved. These two approvals were made using the Special Approval for Emergency system in Japan. This Japanese system was started in 2010 and has been used to approve four drugs to date, including remdesivir and the Pfizer COVID-19 vaccine. This paper discusses future challenges for Japan's Special Approval for Emergency system and organizes what can be learned from experiences to date. As a result, I would like to point Out the following issues. (i) Special Approval for Emergency is a system for approving drugs approved overseas, not a system for approving drugs originally developed in Japan. A system to approve drugs that have not been approved in foreign countries needs to be considered. (ii) In the Special Approval for Emergency system, it is necessary to ensure that postmarketing activities are conducted in accordance with the Risk Management Plan and the conditions of approval, to disclose the results in a timely and speedy manner, and to judge the appropriateness of continued approval based on the results of postmarketing activities.


Subject(s)
Antiviral Agents/therapeutic use , COVID-19/drug therapy , Drug Approval/legislation & jurisprudence , COVID-19/epidemiology , Drug Approval/methods , Drug and Narcotic Control/legislation & jurisprudence , Emergencies , Europe , Humans , Japan , Risk Management , United States , United States Food and Drug Administration
20.
Pharmacol Res ; 166: 105472, 2021 04.
Article in English | MEDLINE | ID: covidwho-1084633

ABSTRACT

The coronavirus disease 2019 (COVID-19) has now rapidly spread around the world, causing an outbreak of acute infectious pneumonia. To develop effective and safe therapies for the prevention and treatment of COVID-19 has become the major global public health concern. Traditional medicine (TM)/herbal medicines (HMs) have been used to treat multiple epidemics in human history, which brings hope for the fight against COVID-19 in some areas. For example, in China, India, and South Korea with traditional medication history and theory, the governments issued a series of guidelines to support TM/HMs in the medication of COVID-19. In contrast, other countries e.g. North American and European governments are typically silent on these practices, unless to warn of possible harm and overselling. Such difference is due to the discrepancy in culture, history and philosophical views of health care and medication, as well as unharmonized policies and standards in the regulation and legalization of TM/HMs among different areas. Herein, we reviewed the responses and scientific researches from seven selected countries on the policies and legalization of TM/HMs to treat COVID-19, and also analyzed the major challenges and concerns to utilize the traditional knowledge and resource.


Subject(s)
Antiviral Agents/therapeutic use , COVID-19/drug therapy , COVID-19/therapy , Complementary Therapies/legislation & jurisprudence , Drug Approval/legislation & jurisprudence , Global Health/legislation & jurisprudence , Medicine, Traditional , Plant Preparations/therapeutic use , Healthcare Disparities/legislation & jurisprudence , Humans , Policy Making
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