All posts tagged: Drug Development

CAC2 Webinar–“Beyond the Signature: Demystifying Informed Consent in Childhood Cancer”

In CAC2's June webinar, "Beyond the Signature: Demystifying Informed Consent in Childhood Cancer," we brought together an extraordinary panel of experts, including a clinical ethicist, a pediatric oncologist, and a parent advocate, who each offered unique and deeply informed perspectives on how we can make this informed consent process more ethical, transparent, and truly family-centered. Informed consent is more than just a signature on a form.  It is a vital process that ensures families understand, trust, and feel empowered in the face of one of the hardest decisions they may ever make: enrolling their child in a clinical trial. In [...] Read more

CAC2 Webinar–“Fertility Hope for Childhood Cancer Survivors”

In CAC2's May webinar, "Fertility Hope for Childhood Cancer Survivors" our speakers explored fertility preservation and future family building options for childhood cancer patients and survivors. Dr. Leena Nahata (Nationwide Children’s Hospital) spoke first and covered a range of topics including How and when to have fertility preservation conversations Risk stratification overview Ovarian tissue cryopreservation Testicular tissue cryopreservation Egg and sperm banking Long term storage Megan Scherer (Worth the Wait) and CAC2 Member Nick Giallourakis (Elephants and Tea) followed and provided real world stories concerning helping survivors with fertility preservation and family building, the importance of providing stories of hope [...] Read more

CAC2 Drop-In Session–“Know Before You Go, Preparing to Attend the CAC2 Summit in June”

If you weren't able to join us for the "Know Before You Go" drop-in session for CAC2 Members it isn't too late to catch up on all the action.  We held this session to help who have already registered for the CAC2 Summit in Washington DC this June prepare for the upcoming CAC2 Summit. We covered everything you need to feel informed, empowered, and ready to make the most of your summit experience. Watch the recording below and review the slide deck where we walked through the highlights and shared engagement tips to ensure a meaningful event for all. VIDEO RECORDING [...] Read more

CAC2 Webinar–“Patient Advocacy, Non-Profit Foundations, and the Changing Landscape of Pediatric Cancer Funding”

In CAC2's April pop-up webinar, "Patient Advocacy, Non-Profit Foundations, and the Changing Landscape of Pediatric Cancer Funding," our special guest, Dr. Kimryn Rathmell, shared compelling insights on the evolving pediatric cancer landscape and the role of advocates. Dr. Rathmell recently completed her appointment as the 17th Director of the National Cancer Institute and has taken a new role as CEO of the Ohio State James Cancer Hospital and Solve Research Institute. Her presentation drew from a distinguished career of scientific excellence and compassionate leadership that spanned from her acclaimed work in kidney cancer genomics to her vision for the future [...] Read more

CAC2 Webinar–“The Role of AI from Diagnosis to Treatment for Childhood Cancer”

In CAC2's April webinar, "The Role of AI from Diagnosis to Treatment for Childhood Cancer," our speakers explored how Artificial Intelligence is being used in pediatric cancer and how to harness the full potential on behalf of patients by enhancing diagnosis, treatment planning, and patient outcomes. Dr. Resnick spoke first and articulated how Artificial Intelligence (AI) will play a transformative role in pediatric cancer research by enhancing diagnosis, treatment planning, and patient outcomes. AI is being used to analyze complex datasets, improve the speed, provide new diagnostic capabilities, accuracy, and personalize treatment strategies, thereby creating new opportunities for children with [...] Read more

CAC2 Webinar–“Fit for Filing”

In CAC2's "Fit for Filing" webinar, our speakers explored designing and conducting academic or academic industry collaborative trials that produce data suitable for regulatory filing. This presentation covered best practices and considerations for conducting trials ready to submit for regulatory approval. The panel examined multiple perspectives, including academia, industry, regulatory authorities, funding organizations, and patient advocates. The speakers emphasized the importance of early, robust, and multi-directional partnerships among academic investigators, cooperative groups, regulatory agencies, and industry, and how funding organizations can help promote these collaborations that are crucial to ensure the unmet medical needs of children with cancer remains a [...] Read more

Guest Blog–Conversations on Cancer – Pediatric Cancers: Navigating the Challenges Together

In conjunction with the European Medicines Agency (EMA), the FDA Oncology Center of Excellence (OCE) held a one-hour Conversations on Cancer public panel discussion on November 19, 2024, to examine an array of challenging decisions faced by members of the pediatric oncology community. The panel discussion featured current CAC2 Board Member Nicole Scobie (Zoe4Life) and past CAC2 Board Member Donna Ludwinski (Solving Kids' Cancer). Because of the rarity of cancers arising in childhood and adolescence and high unmet medical need, pediatric oncology drug development is inherently complex. As a result, pediatric oncology clinician investigators are faced with challenges related to [...] Read more

CAC2 Webinar–Hearing Loss from Cisplatin—Multiple Perspectives on the New FDA Preventative Treatment Option

Children who receive cisplatin as part of their cancer treatment are at risk for progressive and/or permanent hearing loss as a result of their treatment.  This treatment effect can influence speech and language development, educational achievement, and social-emotional development.  Recently, the FDA approved Pedmark, a Sodium Thiosulfate Injection created by Fennec Pharmaceuticals, as a treatment to help reduce the risk of ototoxicity associated with cisplatin.  November’s CAC2 All-Member webinar provided key information about this important breakthrough in improving the quality of life for childhood cancer survivors who received cisplatin. Attendees learned about the importance of this specific drug approval, the [...] Read more

Guest Blog–The Fit for Filing Group at ACCELERATE

Blogged with permission and thanks by the ACCELERATE Fit for Filing Working Group   The ACCELERATE PLATFORM’S Fit for Filing (FFF) group was formed in 2019 to explore the issues of academic-led trials that typically do not produce data that is fit-for-filing to gain marketing approval by regulatory agencies.   The scope of Fit for Filing (FFF) is to develop best principles on how to design and deliver an academic or academic & industry collaborative trial with a dataset that can be included in a package for regulatory filing.Objectives of the FFF working group are: Define the barriers and propose solutions to ensure […]

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FDA Public Meeting: Rare Disease Day 2022

FDA will host a virtual public meeting on March 4, 2022 to join the global observance of Rare Disease Day. The theme for FDA’s Rare Disease Day is “Sharing Experiences in Rare Diseases Together.” Patients, patient advocates, researchers and medical product developers may benefit from attending this public meeting on rare disease product development. During presentations and panel discussions various stakeholders will share their perspectives on and experiences in rare disease product development.

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