scholarly journals The changing landscape of expanded access to investigational drugs for patients with unmet medical needs: ethical implications

2017 ◽  
Vol 10 (1) ◽  
Author(s):  
Eline M. Bunnik ◽  
Nikkie Aarts ◽  
Suzanne van de Vathorst
Keyword(s):  
Expanded Access ◽  
Ethical Implications ◽  
Medical Needs ◽  
Investigational Drugs ◽  
Unmet Medical Needs

scholarly journals What do patients with unmet medical needs want? A qualitative study of patients’ views and experiences with expanded access to unapproved, investigational treatments in the Netherlands

2019 ◽  
Vol 20 (1) ◽  
Author(s):  
Eline M. Bunnik ◽  
Nikkie Aarts
Keyword(s):  
Clinical Trials ◽  
Qualitative Study ◽  
Standard Treatment ◽  
Treatment Options ◽  
Safety And Efficacy ◽  
Expanded Access ◽  
Medical Needs ◽  
Investigational Drugs ◽  
Unmet Medical Needs ◽  
Access Programs

Abstract Background Patients with unmet medical needs sometimes resort to non-standard treatment options, including the use of unapproved, investigational drugs in the context of clinical trials, compassionate use or named-patient programs. The views and experiences of patients with unmet medical needs regarding unapproved, investigational drugs have not yet been examined empirically. Methods In this qualitative study, exploratory interviews and focus groups were held with patients with chronic or life-threatening diseases (n = 39), about topics related to non-standard treatment options, such as the search for non-standard treatment options, patients’ views of the moral obligations of doctors, and the conditions under which they would or would not wish to use non-standard treatment options, including expanded access to unapproved, investigational drugs. Results Respondents had very little knowledge about and/or experience with existing opportunities for expanded access to investigational drugs, although some respondents were actively looking for non-standard treatment options. They had high expectations of their treating physicians, assuming them to be aware of non-standard treatment options, including clinical trials elsewhere and expanded access programs, and assuming that they would inform their patients about such options. Respondents carefully weighed the risks and potential benefits of pursuing expanded access, citing concerns related to the scientific evidence of the safety and efficacy of the drug, side effects, drug-drug interactions, and the maintaining of good quality of life. Respondents stressed the importance of education and assertiveness to obtain access to good-quality health care, and were willing to pay out of pocket for investigational drugs. Patients expressed concerns about equal access to new and/or non-standard treatment options. Conclusion When the end of a standard treatment trajectory comes into view, patients may prefer that treating physicians discuss non-standard treatment options with them, including opportunities for expanded access to unapproved, investigational drugs. Although our respondents had varying levels of understanding of expanded access programs, they seemed capable of making well-considered choices with regard to non-standard treatment options and had realistic expectations with regard to the safety and efficacy of such options. Dutch patients might be less likely to fall prey to false hope than often presumed.

scholarly journals Factors Affecting Health-Related Quality of Life in Multimorbidity

Healthcare ◽  
2021 ◽  
Vol 9 (3) ◽  
pp. 334
Author(s):  
Eunmi Lee ◽  
Sunkyung Cha ◽  
Geun Myun Kim
Keyword(s):  
Quality Of Life ◽  
Health Behaviors ◽  
Private Insurance ◽  
Subjective Health ◽  
Health Related Quality ◽  
Medical Needs ◽  
Related Quality ◽  
Health Related ◽  
Unmet Medical Needs

We investigated the effect of predisposing, enabling, need factors, and health behaviors on health-related quality of life (HRQoL) of patients with multimorbidity according to Andersen’s model. This study is a secondary analysis of population-based cross-sectional surveys. Data from 328 patients with multimorbidity (≥3 co-occurring chronic diseases) from the 6th/7th Korea National Health and Nutrition Examination Surveys were analyzed using logistic regression. Patients ≥65 years, without private insurance, with poor subjective health, unmet medical needs, and/or limited activity were more likely to experience mobility problems. Self-care problems were more likely among those without private insurance and/or with limited activity. Patients lacking living security, with poor subjective health, limited activity, and/or who smoked were more likely to experience problems performing usual activities. Pain/discomfort was more likely among females, Medicaid beneficiaries, and patients with limited activity and/or with poor subjective health. Patients with poor subjective health, limited activity, and/or unmet medical needs were more likely to experience anxiety/depression. The investigation of HRQoL in multimorbidity should consider predisposing, enabling, need factors, and health behaviors. Interventions addressing movement restrictions and personalized care based on HRQoL domains should be prioritized.

scholarly journals Therapeutic Antibodies for the Treatment of Respiratory Tract Infections—Current Overview and Perspectives

Vaccines ◽  
2021 ◽  
Vol 9 (2) ◽  
pp. 151
Author(s):  
Alexie Mayor ◽  
Adélaïde Chesnay ◽  
Guillaume Desoubeaux ◽  
David Ternant ◽  
Nathalie Heuzé-Vourc’h ◽  
...  
Keyword(s):  
Respiratory Tract Infections ◽  
Inflammatory Diseases ◽  
Treatment Strategies ◽  
Candida Spp ◽  
Medical Needs ◽  
Life Threatening ◽  
Rsv Infection ◽  
Syncytial Virus ◽  
Tract Infections ◽  
Unmet Medical Needs

Respiratorytract infections (RTIs) are frequent and life-threatening diseases, accounting for several millions of deaths worldwide. RTIs implicate microorganisms, including viruses (influenza virus, coronavirus, respiratory syncytial virus (RSV)), bacteria (Pseudomonas aeruginosa, Streptococcus pneumoniae, Staphylococcus aureus and Bacillus anthracis) and fungi (Pneumocystis spp., Aspergillus spp. and very occasionally Candida spp.). The emergence of new pathogens, like the coronavirus SARS-CoV-2, and the substantial increase in drug resistance have highlighted the critical necessity to develop novel anti-infective molecules. In this context, antibodies (Abs) are becoming increasingly important in respiratory medicine and may fulfill the unmet medical needs of RTIs. However, development of Abs for treating infectious diseases is less advanced than for cancer and inflammatory diseases. Currently, only three Abs have been marketed for RTIs, namely, against pulmonary anthrax and RSV infection, while several clinical and preclinical studies are in progress. This article gives an overview of the advances in the use of Abs for the treatment of RTIs, based on the analysis of clinical studies in this field. It describes the Ab structure, function and pharmacokinetics, and discusses the opportunities offered by the various Ab formats, Ab engineering and co-treatment strategies. Including the most recent literature, it finally highlights the strengths, weaknesses and likely future trends of a novel anti-RTI Ab armamentarium.

Leveraging Novel and Existing Pathways to Approve New Therapeutics to Treat Serious Drug-Resistant Infections

2016 ◽  
Vol 42 (2-3) ◽  
pp. 429-450 ◽  
Author(s):  
Thomas J. Hwang ◽  
Aaron S. Kesselheim
Keyword(s):  
New Drugs ◽  
Resistant Bacteria ◽  
Drug Resistant ◽  
Regulatory Pathways ◽  
Medical Needs ◽  
The Past ◽  
Drug Resistant Bacteria ◽  
Patient Health ◽  
Life Threatening ◽  
Unmet Medical Needs

Accelerating the development and approval of novel therapeutics has emerged as a key public health priority given the mortality, morbidity, and economic costs associated with infections caused by drug-resistant bacteria. However, there is limited empirical evidence to guide policymaking, such as the factors that may disadvantage antibiotics compared to other classes of drugs. In this Article, we empirically examine characteristics of the key clinical trials underpinning FDA's approval of antibiotics and other drugs over the past decade. Despite perceptions that antibiotic trials are larger and more difficult to conduct, we find that antibiotic trials are no larger than those conducted for drugs approved in other disease areas with high unmet medical needs, suggesting that policymakers may need to target other levers to meaningfully stimulate innovation. We discuss the risks and benefits of harnessing new and existing regulatory pathways to speed the approval of new drugs, particularly those intended to treat patients with serious and life-threatening infections, and we evaluate ways that proposals for new regulatory pathways could be improved to better prioritize and expedite the approval of therapies with the greatest potential for patient health benefits.

UNMET NEED FOR ADDITIONAL MEDICAL CARE FOR SICK CHILDREN IN MOTHER’S VIEW IN RURAL BANGLADESH: IMPLICATIONS FOR IMPROVING CHILD HEALTH SERVICES

2007 ◽  
Vol 39 (5) ◽  
pp. 769-778 ◽  
Author(s):  
NURUL ALAM
Keyword(s):  
Health Services ◽  
Medical Care ◽  
Unmet Needs ◽  
Unmet Need ◽  
Chronic Illnesses ◽  
Rural Bangladesh ◽  
Medical Needs ◽  
Poor Households ◽  
Sick Children ◽  
Unmet Medical Needs

SummaryWhile a country’s health policy aims to provide health services to all who need them, very little in known about unmet need for additional medical care from users’ perspectives in Bangladesh. This study examined unmet medical need (defined as whether a mother felt that, to manage sickness, her child had required medical care that was not available, regardless of reasons and medical care sought) of 2123 under-15 sick children by illness and child’s socioeconomic characteristics in rural Bangladesh. The 1996 Health and Socioeconomic Survey conducted in Matlab recorded children’s chronic (a disease or a condition lasting 3 months or more) and acute (a disease or a condition with a rapid onset and a short, severe course) morbidity, medical care sought to combat illness and unmet needs for additional medical services in mothers’ views to manage the illness. The survey also recorded household socioeconomic data. Logistic regression was used to examine the data. The results reveal that unmet needs for additional medical care were 5·4% for children with acute illnesses, and 30·2% for children with chronic illnesses. For chronic illnesses, seeking medical care to manage illness from any health provider outside the home reduced unmet medical needs. Economic inequalities existed for both acute and chronic illnesses: the odds ratio of unmet medical needs for sick children of the least poor households was 0·42 (95% CI: 0·28–0·64) times that for sick children of the very poor households. The critically high unmet needs for children’s chronic morbidity reveal that the chronic disease control programme in Bangladesh needs urgent revisiting and strengthening.

Unmet Medical Needs in Metastatic Lung and Digestive Neuroendocrine Neoplasms

2018 ◽  
Vol 108 (1) ◽  
pp. 18-25 ◽  
Author(s):  
Jaume Capdevila ◽  
Lisa Bodei ◽  
Philippa Davies ◽  
Vera Gorbounova ◽  
Robert T. Jensen ◽  
...  
Keyword(s):  
Treatment Options ◽  
Phase Iii ◽  
Neuroendocrine Neoplasms ◽  
Limited Evidence ◽  
Medical Needs ◽  
Phase Iii Clinical Trials ◽  
Metastatic Setting ◽  
Metastatic Lung ◽  
Prospective Phase ◽  
Unmet Medical Needs

Unmet medical needs are not infrequent in oncology, and these needs are usually of higher magnitude in rare cancers. The field of neuroendocrine neoplasms (NENs) has evolved rapidly during the last decade, and, currently, a new WHO classification is being implemented and several treatment options are available in the metastatic setting after the results of prospective phase III clinical trials. However, several questions are still unanswered, and decisions in our daily clinical practice should be made with limited evidence. In the 2016 meeting of the advisory board of the European Neuroendocrine Tumor Society (ENETS), the main unmet medical needs in the metastatic NENs setting were deeply discussed, and several proposals to try to solve them are presented in this article, including biomarkers, imaging, and therapy.

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