Daily News

FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies

FDA launches pilot program to accelerate rare disease therapies, improving communication between sponsors and FDA staff. #RareDiseaseTherapies #FDA #ClinicalTrials

Published

1 year ago

September 29, 2023

Daily News Staff

In a significant move to expedite the development of novel drug and biological products for rare diseases, the U.S. Food and Drug Administration (FDA) has introduced a pilot program aimed at fostering more frequent communication between sponsors and FDA staff. This initiative, known as the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program, aims to address clinical development challenges and generate high-quality data to support the future marketing applications of potentially life-saving therapies.

Enhancing Communication and Collaboration:
Under the START Pilot Program, a limited number of sponsors will have the opportunity to obtain regular ad-hoc communication and advice from FDA staff. This communication will focus on addressing product-specific development issues such as clinical study design, selection of control groups, and fine-tuning the choice of patient population. By facilitating improved communication, the FDA aims to streamline the development process for rare disease therapies and ensure that patients with unmet medical needs receive timely access to innovative treatments.

Eligibility and Application Process:
The program is open to sponsors of products currently in clinical trials under an active Investigational New Drug application (IND) regulated by the Center for Biologics Evaluation and Research (CBER) and/or the Center for Drug Evaluation and Research (CDER). Eligibility criteria differ between CBER and CDER-regulated products. CBER-regulated products must be gene or cellular therapies addressing an unmet medical need for a rare disease likely to cause significant disability or death within the first decade of life. CDER-regulated products must be intended to treat rare neurodegenerative conditions, including rare genetic metabolic disorders. Detailed eligibility requirements can be found in the Federal Register Notice.

The Road Ahead:
The FDA plans to accept applications for the START program between January 2, 2024, and March 1, 2024. Participants will be selected based on their application readiness and ability to progress their development programs towards a marketing application. The agency will choose up to three participants for each center. After evaluating the pilot program and receiving feedback from selected sponsors, the FDA may consider a second iteration, which would be announced in the Federal Register at a later date.

Commitment to Rare Disease Therapies:
The FDA’s efforts to advance the development of therapies for rare diseases extend beyond the START Pilot Program. The agency has recently requested feedback from stakeholders on scientific challenges and opportunities related to cellular and gene therapies for rare diseases. This input will help inform the planning of future meetings, workshops, and educational programs to facilitate the development of regulatory tools and frameworks.

Additionally, the FDA has created the Learning and Education to Advance and Empower Rare Disease Drug Developers (LEADER 3D) program under the CDER Accelerating Rare disease Cures (ARC) initiative. Stakeholder feedback gathered through this program will contribute to identifying knowledge gaps in rare disease drug development and the creation of publicly available resources to guide stakeholders in designing and conducting clinical trials.

The FDA’s launch of the START Pilot Program demonstrates its commitment to expediting the development of rare disease therapies. By facilitating enhanced communication and collaboration between sponsors and FDA staff, the program aims to accelerate the availability of potentially life-saving treatments for patients with unmet medical needs. This pilot program, along with other ongoing initiatives, highlights the FDA’s dedication to improving the regulatory framework and recommendations for rare disease product sponsors, ultimately ensuring the safety, effectiveness, and quality of these important therapies.

Read the press release: https://www.fda.gov/news-events/press-announcements/fda-launches-pilot-program-help-further-accelerate-development-rare-disease-therapies?utm_medium=email&utm_source=govdelivery

Author

Daily News Staff

View all posts

Discover more from Daily News

Subscribe to get the latest posts sent to your email.

Up Next

FDA Grants First DNA Test for Dozens of Cancer Types

Don't Miss

Ahsoka: A Thrilling Addition to the Star Wars Universe

Daily News Staff

STM Daily News

Diana Gregory Receives Lifetime Achievement Award from AmeriCorps on Behalf of President Biden

Published

2 days ago

December 19, 2024

Rod Washington, Diana Gregory

Honoring Diana Gregory

In a moment that truly embodies the essence of service and community care, Diana Gregory, the visionary founder of Diana Gregory Outreach Services, has been honored with the esteemed Lifetime Achievement Award from AmeriCorps. This award, presented on behalf of President Joe Biden, took place during a heartfelt ceremony at the VA Health Care System in Phoenix, Arizona, organized by the dedicated leadership of The Order of St. George.

Diana’s selection for this distinguished accolade highlights her extraordinary commitment to fostering positive change and improving the lives of those in her community. Over the past decade, she has passionately addressed critical issues like food insecurity and health awareness, focusing on supporting seniors and veterans. Through her organization, Diana Gregory Outreach Services, she has distributed fresh, nutritious produce to thousands, ensuring that those in need have access to healthy food options.

But Diana’s contributions extend far beyond food distribution; she has also implemented vital health education programs that empower individuals and families, helping them to embrace wellness and make informed choices about their health. This holistic approach has created a transformative impact on underserved communities, reinforcing the importance of access to both nutrition and education.

The Lifetime Achievement Award is an honorable recognition reserved for individuals who have dedicated over 4,000 hours of volunteer service. This milestone is a testament to Diana’s proactive engagement and relentless advocacy for those she serves. Her award serves not only as recognition of her achievements but also as an inspiration for others to follow in her footsteps and contribute to their communities.

The Order of St. George, a respected non-profit organization dedicated to providing humanitarian assistance to refugees, veterans, and the Boy Scouts of America, proudly presented the award. Their involvement signifies the collaborative spirit of various organizations working together to uplift and empower individuals in need, underscoring how interconnected our communities truly are.

As we celebrate Diana Gregory’s well-deserved honor, we recognize the countless lives she has touched and the paths she has paved for a brighter, healthier future. Her remarkable efforts highlight the immense power of community service and the difference every individual can make when they dedicate their time and passion to helping others.

To learn more about Diana Gregory Outreach Services and how you can get involved in her inspiring mission, please visit dianagregory.com. Together, let’s continue the legacy of compassion and service that Diana has so beautifully embodied.

Congratulations, Diana Gregory, on this remarkable achievement! Your dedication is a shining example of what it means to be a true leader and advocate for community well-being. Your work inspires us all to be better, do better, and serve better.

To learn more about Diana Gregory, visit https://dianagregory.com/

Discover more from Daily News

Subscribe to get the latest posts sent to your email.

unknown

Mysterious Lights Over Oregon: Pilots Report Unusual Aerial Phenomena

Published

3 days ago

December 19, 2024

Rod Washington

This past weekend, the skies of Oregon became the backdrop for an extraordinary event that has both puzzled and dazzled observers. At least four commercial pilots reported encountering unexplained bright lights displaying incredible speeds and erratic movements, renewing interest and debate about aerial phenomena.

The Encounter Details

The first report came from a pilot operating an air ambulance who described a startling experience. A bright, red light zoomed towards his Life Flight plane, only to reverse direction and head back towards the Pacific Ocean at bewildering speeds. The event left the pilot and his crew astounded by the light’s velocity and abrupt maneuvers.

Meanwhile, the pilot of a United Airlines flight over Eugene also reported seeing multiple mysterious objects. These unidentified lights moved vertically and horizontally in patterns that defied easy explanation, with no evidence of military activity in the area at the time.

Adding to the intrigue, the Life Flight pilot noted that one of the lights engaged in a corkscrew pattern, an observation that was corroborated by the aircraft’s collision avoidance system. Air traffic control, maintaining a cautious approach, authorized the pilot to maneuver as necessary to maintain safety.

@stmblog
Spotted strange lights in Oregon’s skies? You’re not alone! UFO sightings are on the rise. #OregonUFOSightings #MysteriousLights 🛸✨ ♬ original sound – STMDailyNews

https://www.tiktok.com/t/ZTYVWd4ew

Visual Evidence and Continued Sightings

Throughout these encounters, several pilots managed to capture brief video clips. These clips, although hard to discern due to the dark conditions, show glimpses of the unusual red, circular lights. Another report from two Horizon Airlines pilots further confirmed that whatever was out there wasn’t isolated to a single incident or observer.

Experts Weigh In: Possible Explanations

In the wake of these mystifying sightings, experts have been quick to offer explanations. Douglas Buettner, a scientist who has researched similar incidents, suggests that these lights could likely be flares from Starlink satellites. Starlink, a constellation designed by SpaceX to provide global broadband coverage, has been known to produce vivid optical flares when its panels reflect sunlight.

Buettner’s research, which includes previous pilot reports and observations, supports the idea that this natural reflection of sunlight could explain the phenomena observed. However, he also admits the current data is insufficient to conclusively rule out other possibilities.

Government and Public Response

Following these reports, the Federal Aviation Administration (FAA) confirmed the sighting of unidentified lights by a pilot, although they have not commented further. In a recent Senate hearing, the Pentagon’s director overseeing unidentified anomalous phenomena indicated a spike in such reports since the launch of satellite constellations like Starlink, underscoring the growing interest and concern regarding these observations.

The Broader Implications

While the most likely explanation for these mysterious lights points to satellite flares, the repeated occurrences of such phenomena continue to stir the public’s imagination and scientific curiosity. As we advance in our technological capabilities, the skies above us grow increasingly populated with human-made objects that can sometimes return to us as bewildering spectacles.

In either case, these events serve as a reminder of the vast and unexplored frontiers above us and the ever-present human quest for understanding the mysteries of our universe. Whether they turn out to be mundane or truly otherworldly, the skies over Oregon this weekend have given us all something to ponder and marvel at.

Discover more from Daily News

Subscribe to get the latest posts sent to your email.

STM Daily News

New Jersey Drone Sightings: Unraveling the Mystery

Published

3 days ago

December 18, 2024

Rod Washington

In recent weeks, New Jersey has increasingly found itself under the watchful eye of mysterious drones. These sightings have captivated residents and officials alike, leading to widespread speculation and calls for action. Below is a comprehensive update on the current situation regarding drone sightings in the Garden State, including insights from government officials and community reactions.

The Surge of Sightings

Reports of large drones—up to six feet in diameter—flying over various regions of New Jersey have surged since mid-November. Assemblywoman Dawn Fantasia highlighted the unusual characteristics of these drones, often flying with their lights off and evading detection by traditional methods. Though the origin and purpose of these drones remain unclear, many are convinced that they are not the product of hobbyists.

Notably, many sightings have been concentrated near sensitive locations such as the Picatinny Arsenal, a U.S. military research facility, and Trump National Golf Club in Bedminster. Concerns have only intensified since these drones seem to be operating outside the realm of normal commercial or recreational use.

Government Investigations and Assurances

Federal and state law enforcement agencies, including the FBI and the Department of Homeland Security (DHS), have become actively involved in investigating these sightings. Despite receiving over 5,000 tips, officials state that there is currently no evidence suggesting that these drones pose a threat to national security or public safety. Instead, authorities believe that many of the reported sightings can be attributed to legal drones and other aerial phenomena, including misidentified manned aircraft and even celestial bodies like stars.

Governor Phil Murphy has reinforced this perspective, emphasizing that while drone sightings have raised valid concerns, there’s no immediate threat to public safety. The governor has called for federal assistance in the investigation and urged Congress to empower states with more authority to manage drone activities.

Congressional Response and Public Sentiment

The situation has sparked a debate among lawmakers, with some calling for stricter regulations or even a temporary ban on drone flights in New Jersey. Notably, two congressional representatives have gone so far as to request that the military intervene, highlighting the urgency felt by some constituents. U.S. Rep. Jeff Van Drew even suggested that a foreign entity could be behind the sightings, a theory that has been categorically denied by federal officials.

Public sentiment varies significantly, with many residents expressing concerns about the unknown nature of these drones. James Edwards from Succasunna has pointed out the anxiety gripping the community, noting, “There’s so much that’s unknown,” and calling for a measured response to the sightings.

The View from Washington

At the national level, President Joe Biden has responded to the growing unease, asserting that there is nothing nefarious behind the drone sightings. He emphasized the lawful presence of over one million registered drones operating across the country and assured the public that the situation is being closely monitored.

John Kirby, the White House National Security Advisor, reiterated that the majority of drone sightings do not indicate a coordinated threat and stem from common commercial, hobbyist, or law enforcement operations.

What Lies Ahead?

As investigations continue, officials stress the importance of discerning legitimate threats from unwarranted fears. The situation remains fluid, with ongoing dialogues both in Trenton and Washington to address public concerns and craft appropriate regulatory responses.

While the mystery of the New Jersey drones deepens, it’s vital for residents to remain calm and informed. As the investigation unravels the truths behind these sightings, a collective effort from the community, lawmakers, and federal agencies can help ensure safety and transparency in the skies above New Jersey.

Stay tuned for updates as this story continues to evolve, and be sure to report any new sightings or information to the appropriate authorities. Together, we can navigate the complexities of drone activity in our airspace while embracing this new technological frontier.