Challenges of Rare Disease Drug Development Programs | Rare Disease
By: Amanda Beaster, Ph.D.
2021 brought more challenges and opportunities than ever before. From the starts and stops of opening workplaces, schools and economies to managing an overflowing email inbox, it can be challenging to keep up with all the information you need. So, we rounded up important MMS news and knowledge from 2021 for reference.
We understand the critical importance of a thoughtful organizational change management methodology to guide any new technology implementation and ensure the maximum return on investment. We launched a Change Management (CM) methodology developed with extensive industry-specific knowledge to help Sponsors develop new processes, anticipate problems that could arise from any technology adoption, and mitigate risks to ensure business and project continuity during the transition.
SmartStart™ Templates are a structured authoring tool and clinical trial template to reduce time and effort in the medical writing process. SmartStart™ Templates were developed to improve consistency, time-savings, and quality while allowing for the reuse of data across deliverables such as protocol to protocol, protocol to CSR, protocol, to SAP, CSR to CSR, and SAP to SAP.
MMS announced a restructuring of their consulting services with the launch of a Project Advisory Services Unit to meet the needs of many small to mid-sized organizations in immediate, short and long-term needs for senior-level guidance, specialized consulting, and advisory services. MMS Project Advisory Services supports Sponsors’ needs across the entire product lifecycle, from early regulatory development strategy consultations to clinical trial and marketing authorization submissions.
Vendor Management as a Service
MMS offers a full suite of vendor management services as well as ad hoc vendor management support to support complying with regulatory requirements for vendor oversight. MMS has the proven expertise, knowledge, processes, and documentation library to provide organizations with regulatory-compliant Vendor Management Services or create a complete custom program.
MMS joined a historic alliance of fifty life sciences and healthcare organizations that seeks to accelerate the broad adoption of patient-focused, decentralized clinical trials and research. DTRA has plans to unite industry stakeholders, including healthcare companies, regulators, patient groups and research organizations with a singular mission to make clinical trial participation widely accessible by advancing policies, research practices, and new technologies in decentralized clinical research.
MMS was selected by Health Data Research UK (HDR UK) – the national institute for health data science – as a partner to support the International COVID-19 Data Alliance (ICODA) initiative. MMS was selected from a pool of some of the world’s top clinical research organizations to provide data engineering services to support a globally coordinated, health data-led research response to tackle the COVID-19 pandemic.
MMS provided regulatory strategy support to Praxis Bioresearch in the development of a therapeutic for apathy in Alzheimer’s disease. This comes as Praxis was awarded a $2.9 Million Fast-Track SBIR Grant to advance the company’s lead development candidate PRX-P4-003. Experts at MMS provided regulatory strategy, writing and operations support, including submission publishing, through an exploratory IND and pre-IND submission, with contributions made from a global team on a 24-hour support clock.
Rare Disease Initiatives:
We launched a pro-bono support program aimed at advancing research for rare diseases. Within this program, MMS will select from patient advocacy groups to provide complimentary regulatory assistance.
To apply for support, complete and send an application to the attention of the ‘Ultra-rare disease Assistance Program’ by emailing email@example.com. For details on MMS rare disease expertise, visit https://www.mmsholdings.com/rare-disease/.
Our MMS team shared their expertise in ten webinars this year.
Kelly Vaillant and Zach Weingarden shared how cutting-edge technology and data-driven processes combine to expedite anonymization of clinical artifacts, explored how machine learning techniques can be applied to accurately identify subject-identifying text in PDF documents, and evaluated risk assessment techniques, both qualitative and quantitative, with how they apply to each artifact.
Data scientists at MMS explain in this webinar: why data science and the use of machine learning is essential to pharmaceutical, biotech, and medical device organizations, how to use machine learning to understand outcomes before they occur, practical approaches for inference or prediction through machine learning, metrics to evaluate the performance of predictions made, and the possibilities for the future of machine learning in clinical research.
Explore with us in this webinar, as our experts discuss how Sponsors who leverage FSP partners across multiple interdependent functional lines achieve the maximum benefit and the increasing importance of the way that new innovations and technology continue to change the way we work and the future skillsets needed.
Join us as experts in project management, medical writing, and regulatory operations discuss best practices for a successful submissions process from start to finish.
This webinar explored how the new CT Regulation impacts Sponsors and reviewed changes to clinical trials processes, protocols, informed consent for minors, and impacts to lay summaries.
Explore with us as we discuss different protocol writing and study design approaches, ensuring protocols are designed to meet both the patients’ and Sponsor’s needs, and maintaining compliance with the International Council for Harmonisation requirements.
This two-part webinar focuses on challenges and areas of flexibility within the application of statutory standards as they apply to rare diseases. Particular attention will be paid not only to the laws and guidelines that specifically relate to orphan drugs but also how the general regulatory standards that govern safety and efficacy are uniquely applied to rare diseases at different levels of prevalence.
This rare disease webinar provides an overview of the FDA’s compendium of rare disease and expedited programs and provides sponsors with recommendations to leverage these opportunities and speed up the development timeline.
In this webinar, attendees will learn: the statistical and data analytics landscape related to rare diseases, information related to rare disease study design, longitudinal data volume, comparability and appropriateness of using RWD to supplement trial data, considerations for data access, quality and standardization, the selection and definition of informative endpoints, and how proper analysis techniques can help accelerate a clinical development program.
In this webinar, attendees will learn: recommendations for developing interactive, concise, and useful visualizations grouped within dashboards, the importance of understanding the intended audience and objective for data visualizations, practical tips for identifying and avoiding common pitfalls, and the development process for interactive dashboards from initial discovery through User Acceptance Testing (UAT). Additionally, presenters will share sample interactive operational dashboards developed with the MMS Datacise platform.
Join us for this free webinar to learn: the many components of an NDA submission, how to avoid the common programming downfalls in an NDA submission, and how to manage the challenges that arise when working on an NDA submission.
To keep up with what MMS does in 2022, follow us on LinkedIn!