Collective Statement Regarding Patient Access to Approved Therapies from the Center Directors of Parent Project Muscular Dystrophy’s Certified Duchenne Care Centers

AbstractImaging clinical trials can be burdensome and often delay patient access to novel, high-quality medical devices. Tools for in silico imaging trials have significantly improved in sophistication and availability. Here, I describe some of the principal advantages of in silico imaging trials and enumerate five lessons learned during the design and execution of the first all-in silico virtual imaging clinical trial for regulatory evaluation (the VICTRE study).

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Understanding low chemoprevention uptake by women at high risk of breast cancer: findings from a qualitative inductive study of women’s risk-reduction experiences

BMC Women s Health ◽

10.1186/s12905-021-01279-4 ◽

2021 ◽

Vol 21 (1) ◽

Author(s):

Tasleem J. Padamsee ◽

Megan Hils ◽

Anna Muraveva

Keyword(s):

Breast Cancer ◽

African American ◽

High Risk ◽

Risk Reduction ◽

White Women ◽

Future Research ◽

Structured Interviews ◽

Critical Gap ◽

High Risk Women ◽

Parent Project

Abstract Background Chemoprevention is one of several methods that have been developed to help high-risk women reduce their risk of breast cancer. Reasons for the low uptake of chemoprevention are poorly understood. This paper seeks a deeper understanding of this phenomenon by drawing on women’s own narratives about their awareness of chemoprevention and their risk-related experiences. Methods This research is based on a parent project that included fifty in-depth, semi-structured interviews with a purposive sample of African American and White women at elevated risk of breast cancer. This specific study draws on the forty-seven interviews conducted with women at high or severe risk of breast cancer, all of whom are eligible to use chemoprevention for breast cancer risk-reduction. Interviews were analyzed using grounded theory methods. Results Forty-five percent of participants, and only 21% of African American participants, were aware of chemoprevention options. Women who had seen specialists were more likely to be aware, particularly if they had ongoing specialist access. Aware and unaware women relied on different types of sources for prevention-related information. Those whose main source of information was a healthcare provider were more likely to know about chemoprevention. Aware women used more nuanced information gathering strategies and worried more about cancer. Women simultaneously considered all risk-reduction options they knew about. Those who knew about chemoprevention but were reluctant to use it felt this way for multiple reasons, having to do with potential side effects, perceived extreme-ness of the intervention, similarity to chemotherapy, unknown information about chemoprevention, and reluctance to take medications in general. Conclusions Lack of chemoprevention awareness is a critical gap in women’s ability to make health-protective choices. Future research in this field must consider complexities in both women’s perspectives on chemoprevention and the reasons they are reluctant to use it.

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P50 Exploring the Merits and Availability of Smartphone Applications Related to Melanoma

BJS Open ◽

10.1093/bjsopen/zrab032.049 ◽

2021 ◽

Vol 5 (Supplement_1) ◽

Author(s):

Jade Kabbani ◽

Jamil Kabbani ◽

Jade Kabbani

Keyword(s):

Clinical Management ◽

Literature Search ◽

Patient Access ◽

Smartphone Applications ◽

App Store ◽

Widespread Availability ◽

Smartphone Use ◽

Clinical Tools ◽

Google Play ◽

Privacy Issues

Abstract Background The increased use of smartphone applications across healthcare specialties has been particularly relevant in dermatology, with dermatology related applications widely available on mainstream application stores. We reviewed published literature regarding melanoma-related applications, and the number and types of such applications available for download. Methods A literature search of “dermatology”, “smartphone” and “melanoma” was conducted to identify publications assessing applications of interest. “Melanoma” was searched in Apple’s (iOS) “App Store” and Google’s “Google Play”, and application purposes and ratings were analysed. Results 54 of the 63 literature search results explored smartphone use in relation to melanoma, describing benefits including quicker patient access to care, reduced referrals and hence unnecessary consultations, and improved accessibility to information. However, concerns include insufficient image quality, privacy issues related to encryption, and diagnostic inaccuracy. Searches on the Google Play and iOS stores identified 249 and 51 apps respectively. 25% of Google Play results were categorised as clinical tools, 17% as educational, and 58% as recreational. The corresponding results for the App store were 92%, 6% and 2%. 81% of the educational apps and 92% of the clinical management apps related to dermatology and melanoma on Google Play, whereas all of the clinical management apps and 67% of the education apps on the App store were of relevance. Conclusion The results illustrate the widespread availability of applications related to melanoma, particularly for educational and clinical purposes. Standardising photographing techniques, improving diagnostic accuracy, and privacy issues are important aspects to consider and warrant further investigation.

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The Biosimilar Landscape: An Overview of Regulatory Approvals by the EMA and FDA

Pharmaceutics ◽

10.3390/pharmaceutics13010048 ◽

2020 ◽

Vol 13 (1) ◽

pp. 48

Author(s):

Ioana Gherghescu ◽

M. Begoña Delgado-Charro

Keyword(s):

Drug Administration ◽

Action Plan ◽

European Medicines Agency ◽

Patient Access ◽

Clinical Evaluations ◽

The Past ◽

The Us ◽

Improve Patient ◽

The Eu

Biosimilar medicines expand the biotherapeutic market and improve patient access. This work looked into the landscape of the European and US biosimilar products, their regulatory authorization, market availability, and clinical evaluation undergone prior to the regulatory approval. European Medicines Agency (EMEA, currently EMA) and Food and Drug Administration (FDA) repositories were searched to identify all biosimilar medicines approved before December 2019. Adalimumab biosimilars, and particularly their clinical evaluations, were used as a case study. In the past 13 years, the EMA has received 65 marketing authorization applications for biosimilar medicines with 55 approved biosimilars available in the EU market. Since the first biosimilar approval in 2015, the FDA has granted 26 approvals for biosimilars with only 11 being currently on the US market. Five adalimumab biosimilars have been approved in the EU and commercialized as eight different medicines through duplicate marketing authorizations. Whilst three of these are FDA-approved, the first adalimumab biosimilar will not be marketed in the US until 2023 due to Humira’s exclusivity period. The EU biosimilar market has developed faster than its US counterpart, as the latter is probably challenged by a series of patents and exclusivity periods protecting the bio-originator medicines, an issue addressed by the US’s latest ‘Biosimilar Action Plan’.

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Radiological Reporting of Urgencies Related to Medical Devices: Commentary on a Possible Systematic Approach

Tomography ◽

10.3390/tomography7030024 ◽

2021 ◽

Vol 7 (3) ◽

pp. 268-277

Author(s):

Andrea Contegiacomo ◽

Marco Conti ◽

Massimo Muciaccia ◽

Pietro Trombatore ◽

Michele Dezio ◽

...

Keyword(s):

Emergency Department ◽

Medical Devices ◽

Clinical Management ◽

Systematic Approach ◽

Patient Access ◽

Radiological Images ◽

Radiological Report

Most medical devices are routinely recognized on radiological images and described as normal findings in the radiological report, but sometimes they can cause patient access to the emergency department. Multiple possible complications have been described and most of them require prompt recognition by radiologists for proper clinical management. This commentary proposes a systematic approach to radiological reporting of the most common emergent complications related to medical devices with the intent to avoid the omission of important findings in the final radiological report.

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A global perspective on the issue of access to insulin

Diabetologia ◽

10.1007/s00125-020-05375-2 ◽

2021 ◽

Author(s):

David Beran ◽

Maria Lazo-Porras ◽

Camille M. Mba ◽

Jean Claude Mbanya

Keyword(s):

Type 1 Diabetes ◽

Diabetes Management ◽

Multinational Companies ◽

Global Perspective ◽

Patient Access ◽

Middle Income ◽

Middle Income Countries ◽

Wide Range ◽

Pricing And Reimbursement

AbstractThe discovery of insulin in 1921 changed the prognosis for people with type 1 diabetes. A century later, availability and affordability of insulin remain a challenge in many parts of the globe. Using the WHO’s framework on understanding the life cycle of medicines, this review details the global and national challenges that affect patients’ abilities to access and afford insulin. Current research and development in diabetes has seen some innovations, but none of these have truly been game-changing. Currently, three multinational companies control over 95% of global insulin supply. The inclusion of insulin on the WHO’s Prequalification Programme is an opportunity to facilitate entry of new companies into the market. Many governments lack policies on the selection, procurement, supply, pricing and reimbursement of insulin. Moreover, mark-ups in the supply chain also affect the final price to the consumer. Whilst expenses related to diabetes are mostly covered by insurance in high-income countries, many patients from low- and middle-income countries have to pay out of their own pockets. The organisation of diabetes management within the healthcare system also affects patient access to insulin. The challenges affecting access to insulin are complex and require a wide range of solutions. Given that 2021 marks the centenary of the discovery of insulin, there is need for global advocacy to ensure that the benefits of insulin and innovations in diabetes care reach all individuals living with diabetes. Graphical abstract

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Where Stool is a Drug: International Approaches to Regulating the use of Fecal Microbiota for Transplantation

The Journal of Law Medicine & Ethics ◽

10.1177/1073110519897729 ◽

2019 ◽

Vol 47 (4) ◽

pp. 524-540 ◽

Cited By ~ 1

Author(s):

Alexandra Scheeler

Keyword(s):

Human Cell ◽

Fecal Microbiota ◽

Regulatory Agencies ◽

Patient Access ◽

Global Survey ◽

Tissue Product

Regulatory agencies vary widely in their classification of FMT, with significant impact on patient access. This article conducts a global survey of national regulations and collates existing FMT classification statuses, ultimately suggesting that the human cell and tissue product designation best fits FMT's characteristics and that definitional objectives to that classification may be overcome.

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