Maurie Markman, MD

Maurie Markman, MD on strategies to appropriately evaluate small but clinically relevant patient subsets for whom traditional phase 3 randomized clinical trials are difficult, if not impossible, to complete in a realistically timely manner.

Maurie Markman, MD, discusses how agency leaders should be encouraged to improve regulatory science, include a patient’s perspective in approval decisions, reduce unnecessary bureaucracy and costs associated with the conduct of trials, and accelerate the overall review process for drug approval.

Maurie Markman, MD, discusses the need for peer-reviewed publications to encourage further discussion and debate, challenge existing ideas or dogma, or provide support for current biological concepts or in the clinical realm for treatment of patients.

Maurie Markman, MD, highlights the ongoing regulatory concerns that demand attention of the FDA in the cancer arena amidst evolving issues regarding COVID-19.

The continued uncertainties of the current and future status of the COVID-19 pandemic have resulted in a lack of trust in the authority of the scientific establishment in the United States, and elsewhere, as it operates during these difficult times.

There has been limited discussion in the cancer literature for how peer review among the multiple oncology journals and international cancer conferences is conducted.

It is difficult to overstate the confusion associated with our nation’s messaging regarding a well-considered, rational, and scientifically based public health–focused approach to the current and future ramifications of the COVID-19 pandemic.

One year ago, few objective observers would have suggested that the percentage of individuals who are currently fully vaccinated is less than 30%, despite the well-documented real-world success data for the vaccines in preventing serious illness, hospitalizations, and death from COVID-19 infection.

Within the hierarchy of clinical trials, phase 3 randomized studies remain the gold standard, though some argue that appropriately designed meta-analysis of multiple studies provides even more definitive and meaningful insight.

The overall picture related to COVID-19 in the Unites States is encouraging, and we might suspect that the American public would be relatively united with favorable views of efforts of public health officials and organizations at the national, state, and local levels to successfully control this terrible event and return us to our prepandemic state.

Multiple FDA approvals and an increasing number of clinical trials examining molecular target–based therapeutics, including second- or even third-generation drugs against a well-defined target, present an ever-widening array of drugs for routine cancer care based on the discovery of specific molecular targets within the tumor or within the germline.

Although the COVID-19 vaccine rollout is unprecedented in speed and scope, the process of postapproval surveillance has been shown to be robust and should serve as a source of reassurance to the public regarding the effectiveness of the initial and follow-up review process.

The challenges of assessing COVID-19 vaccines shows that events in the real world may differ from those of a formal objective scientific analysis, especially in a setting where such evaluations of necessity involve very small numbers.

Although the spectacular success associated with the development of several safe and highly efficacious vaccines and therapies for COVID-19 has once again confirmed the remarkable impact of advancements on public and individual health, we must also acknowledge recent stunning examples of the failure of scientifically oriented government agencies to provide objectively valid nonpolitical recommendations, policies, and conclusions.

The current endorsement of 3 COVID-19 vaccines in the United States, with several additional products pending FDA review, in less than 1 year from the identification of the structure of the COVID-19 virus is simply remarkable.

In the cancer arena, COVID-19 information problems highlight the critical role of clear, honest, and effective communication with the public, patients, and their families regarding the increasing complexity of a multitude of topics related to malignant disease and its management.

Evolving data provide provocative support for the relevance of an optimistic vs a pessimistic perspective in influencing outcomes in serious malignant conditions.

When one is dealing with the topic of cancer, its treatment and consequences, language that clinicians and members of the cancer research community use may have an even greater impact.

Formerly, small cell lung cancer was generally considered more chemotherapy sensitive in the short term but also associated with an overall inferior survival outcome; however, the difference in prognosis compared with metastatic non–small cell lung cancer was measured in months—rarely longer.

Maurie Markman, MD, discusses the need for comparative clinical trials with PARP inhibitors in ovarian cancer.

The importance of objectively valid data is well established in clinical medicine. Such data include an accurate recording of a patient’s clinical history; evaluation of signs and symptoms of illness; and measurement of various routine indicators, such as granulocyte and platelet counts, serum glucose, electrolytes, and liver function tests.

Although recent benefits do not pertain to all cancers and “cure” remains a relatively uncommon event, oncologists have an increasing number of molecularly targeted and immunotherapeutic strategies to employ based on objectively meaningful clinical trial outcomes.

Maurie Markman, MD, discusses remaining questions regarding human papillomavirus–negative cervical cancer.

In a pandemic, the public and its leaders yearn for relatively simple answers that can lead to solutions and forceful actions such as preventing serious infection and hospitalizations, treating active illness, and developing safe and effective vaccines quickly made available to the public.

Although PARP inhibitors are generally reasonably well tolerated, certainly compared with platinum and other routinely employed cytotoxic antineoplastic drugs, the majority of patients receiving PARP inhibitors in multiple reported clinical trials reported low-grade nausea and fatigue.

Maurie Markman, MD, discusses the utility of PARP inhibitors in ovarian cancer.

Despite solid evidence that human papillomavirus vaccination is remarkably effective in preventing persistent infection by HPV types that are known to be responsible for more than 70% to 80% of cervical cancer cases worldwide, this strategy’s utility in substantially reducing the subsequent development of the malignancy itself remained an open question.

The fundamental objectivity of major scientifically oriented public health agencies has been called into question, while the issue of what constitutes sufficient evidence for a therapeutic strategy to be considered “standard” is also being challenged.

In the realm of medicine, we see enormous opportunities for truly novel technologies to revolutionize the health and welfare of all members of society.

Because it is likely that COVID-19 will be around for a considerable period of time, it is hoped that all involved in the clinical research establishment can view this experience as a process that needs substantial improvement.