US Food and Drug Administration FDA Launches the Rare Disease Framework Initiative to Develop a Holistic, Fit-For-Purpose Framework for Rare Disease Drug Development Effort begins by Soliciting Public Feedback

US Food and Drug Administration FDA Launches the Rare Disease Framework Initiative to Develop a Holistic, Fit-For-Purpose Framework for Rare Disease Drug Development Effort begins by Soliciting Public Feedback https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/fda-rare-disease-framework-initiative-public-meeting-10282026?utm_medium=email&utm_source=govdelivery Rare Disease Framework https://www.fda.gov/industry/fda-rare-disease-innovation-hub/rare-disease-framework?utm_medium=email&utm_source=govdelivery The U.S. Food and Drug Administration, a division within the U.S. Department of Health and Human Services (HHS), today announced the Rare Disease Framework Initiative to reimagine the development of drugs and biologics for rare diseases. As outlined in a new white paper, the Initiative will develop an optimized, fit-for-purpose framework that reflects the realities of rare disease drug development by considering the nuances of each disease, patient needs, product type and the evidence that can be realistically generated in small patient populations. To solicit feedback on the Rare Disease Framework Initiative from patients and disease advocates, industry, and others, the FDA will host an October 28 meeting at the FDA’s White Oak campus. The meeting will be facilitated by the Reagan-Udall Foundation for the FDA. “Americans with rare diseases cannot afford a regulatory system that treats every condition the same,” said HHS Secretary Robert F. Kennedy, Jr. “Under President Trump, FDA is rethinking rare disease drug development by putting patients at the center and matching regulatory requirements to the realities of small patient populations. We will remove unnecessary barriers, embrace cutting-edge science, and accelerate promising treatments while upholding FDA’s rigorous standards.” “Patients and families living with rare diseases have few or no approved treatment options, and the burden of uncertainty can extend across every part of their lives,” said Acting FDA Commissioner Kyle Diamantas, J.D. “As we learn more about rare diseases and better understand the breadth of suffering they cause, we must ensure that America’s regulatory system remains at the forefront — prepared to respond to emerging science, anticipate the needs of patients and families, and help bring safe and effective therapies to those who need them. The FDA will fight hard for patients by building a more consistent, predictable, and innovative approach to rare disease drug development while maintaining the rigorous standards Americans expect from the FDA.” The FDA has long used regulatory flexibility and developed targeted programs to support innovation in the development of drugs and biologics for rare diseases. These efforts have led to meaningful advances for patients and families. Nevertheless, with only 5% or so of rare conditions having an FDA-approved treatment, the FDA agrees with patients, drug developers, and providers that more must be done to speed the development of safe and effective treatments for rare diseases. In developing the framework, the FDA is committed to the following: Providing greater clarity, consistency, and predictability in regulatory decision-making through tailored guidance and transparent communication. Embedding the patient’s voice both early, and at clearly defined milestones, in the drug development and review process. Further leveraging external expertise to support customization of regulatory review. Building shared resources, robust training, and institutional knowledge on rare disease drug development and review. Applying risk-based approaches, particularly to chemistry, manufacturing, and controls review and pre-approval inspections. Championing the use of cutting-edge science, novel methods and innovative statistical approaches in rare disease drug development. The Initiative is led by the FDA’s Rare Disease Innovation Hub, partnering with the Office of the Commissioner, the Center for Biologics Evaluation and Research, and the Center for Drug Evaluation and Research. “Patients with rare diseases and their families deserve a regulatory process that addresses the uniqueness of each of their conditions while maintaining the FDA’s rigorous standards,” said the Hub’s Director, Amy Comstock Rick, J.D. “A bold vision to reimagine the framework for rare disease drug development that builds on key learnings and milestones is necessary to meet this moment. This new framework will enhance consistency and predictability for both sponsors and for patients.” Further opportunities to provide input will follow the public meeting, including sector-specific roundtables, Requests for Information, and internal FDA feedback sessions. This feedback will inform the detailed development of a new framework, which might include potential new or revised guidance, revisions to regulations, staff training, and organizational changes.

Prescribe with Confidence Patients with Opioid Use Disorder Need You

https://www.fda.gov/drugs/drug-safety-and-availability/prescribe-confidence?utm_medium=email&utm_source=govdelivery FDA’s Prescribe with Confidence campaign supports the Trump Administration’s Great American Recovery Initiative, which focuses on preventing opioid use disorder, expanding access to treatment, and helping people stay in recovery. The campaign provides practical resources to help healthcare professionals feel more confident in prescribing medications for opioid use disorder (MOUD). Health centers play an important role in expanding access to care, particularly in rural and underserved communities. Prescribe with Confidence includes educational videos and resources on MOUD, including buprenorphine, methadone, and naltrexone. FDA is releasing the final four videos in the campaign’s 10-part series. The new videos feature Amy Techau, a nurse practitioner working in rural community mental health, and highlight evidence-based treatment, compassionate care, and patient recovery stories. https://www.fda.gov/drugs/prescribe-confidence/their-own-words-health-care-providers?utm_medium=email&utm_source=govdelivery

FDA Approves First Treatment for Certain People With BCG-Unresponsive Bladder Cancer Expanded approval of Anktiva marks a new FDA approach to using evidence across closely related forms of bladder cancer

FDA Approves First Treatment for Certain People With BCG-Unresponsive Bladder Cancer  Expanded approval of Anktiva marks a new FDA approach to using evidence across closely related forms of bladder cancer https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-certain-people-bcg-unresponsive-bladder-cancer?utm_medium=email&utm_source=govdelivery The U.S. Food and Drug Administration today expanded the approved use of Anktiva (nogapendekin alfa inbakicept-pmln), given with Bacillus Calmette-Guérin (BCG), to include adults with high-grade papillary-only bladder cancer that has not spread into the bladder muscle and has not responded to BCG treatment. Anktiva is the first FDA-approved treatment specifically for patients with BCG-unresponsive, high-grade papillary-only disease. The approval marks a new FDA approach to evaluating treatments for BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). The agency based its decision on evidence supporting an earlier approval of Anktiva for adults with another form of NMIBC, carcinoma in situ (CIS), with or without papillary tumors, and scientific similarities between CIS and high-grade papillary-only disease. “We listen to patients, clinicians, and researchers, and we act when the science supports a new approach,” said R. Angelo de Claro, M.D., Director of the FDA’s Oncology Center of Excellence. “For Anktiva, our review of the similarities between these cancers allowed us to build on the results in patients with CIS and expand treatment options for people with high-grade papillary tumors alone. This approach may also help guide the development of future treatments.” NMIBC is bladder cancer that has not grown into the bladder’s muscle layer and can include CIS and papillary tumors. CIS forms flat patches along the bladder’s lining, while papillary tumors grow from the lining into the bladder as small, finger-like projections. CIS is always high-grade, while papillary tumors can be low-grade or high-grade. High-grade cancers are more likely to grow and spread than low-grade cancers. Treatment for high-risk NMIBC often starts with BCG, which activates the immune system inside the bladder to fight cancer. Sometimes, however, the cancer persists or returns. Adding Anktiva gives the immune system another way to fight the cancer, and the combination can help some people whose cancer was not controlled by BCG alone. Anktiva’s CIS approval was supported by a trial (NCT03022825) that evaluated the treatment’s effectiveness in 77 adults, some of whom also had papillary tumors. Sixty-two percent achieved a complete response, meaning no high-grade cancer was detected during follow-up assessments, and of those, 58% maintained that response for at least 12 months and 40% for at least 24 months. For today’s expanded approval, the FDA considered these results alongside clinical and laboratory evidence showing that CIS and high-grade papillary tumors share important features. They often occur together, can be difficult to distinguish under a microscope and carry a high risk of returning or progressing when BCG treatment no longer works. These similarities supported applying the evidence of Anktiva’s effectiveness in people with CIS to those with high-grade papillary-only disease. To help developers apply this approach, the FDA is working toward finalizing its draft guidance, “Bacillus Calmette-Guérin-Unresponsive Nonmuscle Invasive Bladder Cancer: Developing Drugs and Biological Products for Treatment.” The guidance is intended to include discussion of using evidence from studies in patients with CIS to support approval of treatments for BCG-unresponsive, high-grade papillary-only NMIBC. Anktiva is given with BCG directly into the bladder through a catheter. It is given once a week for six weeks, with additional treatment based on how the cancer responds. Treatment may continue for up to 37 months. It should be stopped if the cancer persists after a second six-week course, returns or progresses, or side effects become unacceptable. The most common side effects and laboratory abnormalities included painful or frequent urination, an urgent need to urinate, blood in the urine, urinary tract infections, muscle or joint pain, chills, fever, and increased blood levels of creatinine and potassium. Anktiva’s prescribing information warns that delaying surgery to remove all or part of the bladder can allow the cancer to spread to other parts of the body, which can be fatal. The approval was granted to Altor BioScience, LLC.  Bacillus Calmette-Guérin-Unresponsive Nonmuscle Invasive Bladder Cancer: Developing Drugs and Biological Products for Treatment Draft Guidance for Industry August 2024 https://www.fda.gov/regulatory-information/search-fda-guidance-documents/bacillus-calmette-guerin-unresponsive-nonmuscle-invasive-bladder-cancer-developing-drugs-and?utm_medium=email&utm_source=govdelivery

Faster Aging on Blood Tests Tied to Colorectal Cancer Risk in Young Adults

https://www.medscape.com/viewarticle/faster-aging-blood-tests-tied-colorectal-cancer-risk-young-2026a10012hy?src=

Skin Radiation Dose Linked to Breast Lymphedema Risk

https://www.medscape.com/viewarticle/skin-radiation-dose-linked-breast-lymphedema-risk-2026a100128v?src= A single-center retrospective study identified a breast skin radiation-dose threshold associated with lymphedema risk and found that mammographic skin-thickness changes may help identify the complication. More specifically, breast skin receiving more than 40 Gy over a volume exceeding 90 cc was associated with nearly 4-fold higher odds of breast lymphedema after breast-conserving therapy, and mammographic skin thickness increased significantly over time in patients with breast lymphedema compared with those without it.

SMC Rejects PI3K Inhibitor for Advanced Breast Cancer Dr Sheena Meredith

https://www.medscape.com/viewarticle/smc-rejects-pi3k-inhibitor-advanced-breast-cancer-2026a10012ag?src= The Scottish Medicines Consortium (SMC) has rejected the use of inavolisib (Itovebi, Roche) within NHS Scotland to treat adults with locally advanced or metastatic breast cancer who have oestrogen receptor-positive disease and a particular gene mutation. The drug is intended to be used in combination with palbociclib and fulvestrant to treat oestrogen receptor-positive, HER2-negative breast cancer with a mutation in the PIK3CA gene and which has recurred during adjuvant endocrine therapy or within 12 months of completing it.

Senolytic CAR-T Shows Promise for Age-Related Diseases

https://www.medscape.com/viewarticle/senolytic-car-t-shows-promise-age-related-diseases-2026a10012jk?src= Chimeric antigen receptor (CAR) T-cell therapy has transformed the treatment of certain hematologic cancers. Researchers are now exploring whether this technology could also identify and eliminate senescent cells, which accumulate with age and may contribute to conditions such as diabetes, Alzheimer’s disease, and liver fibrosis.