Name: Exploring the future: Opportunities and challenges in cell and gene therapy for rare diseases
Start: 2025-03-05T15:00:00Z
End: 2025-03-05T15:01:03.000Z
Location: BrightTALK
Rating: 5

pharmaphorum provides a digital podium for communicating thought leadership and innovation within pharma. Our webinars bring together different stakeholders from across the healthcare spectrum to drive fresh thinking and novel solutions that deliver better outcomes for patients.

Our experts will delve into the evolving landscape of life sciences and patient care, driven by cutting-edge technology, data insights, and a renewed commitment to inclusive, patient-centred solutions.

Health Trends 2025: Reimagining What's Possible

Discover how AI is transforming the pharma commercial strategy. Unlock real-time insights & actions from data.

The AI effect in pharma commercial strategy: From data to real-time insight & actions

In this webinar, you’ll hear how to identify physicians who are prescribing competing drugs or treating a high number of patients, and discover which medications and treatment combinations are being used by physicians in key therapeutic areas.

Decoding physician prescribing behaviour:  A data-driven journey

In a new webinar with Atlas Clinical Research’s Chief Executive Officer Mark Scullion and Batisha Anson, Global Head of Patient Diversity and Health Equity at Syneos Health, learn more about the site experience for clinical research sites – including the important need for a partner who understands the challenges and drives solutions that can ease burden, create a better experience, and expand access and representation in clinical research for patients.

Enabling site success: Key insights to reducing burden and increasing access

Discover how to balance personalised engagement with regulatory compliance across global pharma markets in an upcoming webinar presented by EVERSANA INTOUCH. Join industry leader Denise Dalton, VP of worldwide omnichannel strategy at Bristol Myers Squibb, as she explores the challenges of achieving personalisation at scale in today's interconnected pharmaceutical landscape.

Global reach, personal touch: Mastering customer engagement at scale

The success of clinical trials depends on various factors beyond traditional clinical care, including social determinants of health (SDOH) like housing, health literacy, transportation, and social support. These factors can impact participant recruitment, retention, and the quality of data collected. In this webinar, panelists will explore how industry leaders are using non-clinical data to improve recruitment, retention, and evidence generation in clinical trials.

Beyond clinical: Boosting recruitment and retention with SDOH data

Pharma marketing is going through an evolution, scaling mass messaging with personalised outreach powered by complex systems. In this deep dive into innovation in content transformation, pharmaceutical companies can learn how to move from pilot projects to scalable models. We'll share innovative approaches to personalise customer engagement, accelerate content generation, and drive the greatest impact for your brand.

Shifting the paradigm in pharma marketing with content transformation

Learn why storytelling is crucial in scientific publications. The webinar will showcase examples of storytelling for clinical data, discuss trends, and explore opportunities for data storytelling to enhance health equity through plain-language content and publication extenders.

Unlocking the Power of Storytelling in Medical Publications

The new PDURS framework is poised to change the way pharma thinks about companion apps, digital medicines, and digital therapeutics. However, to unlock its potential, digital leaders in the pharma industry need to know what the framework is, how it works, and how it can be of use to them. Benefit from our speakers’ unique perspectives at the intersection of pharma and government as you get the skinny on this new way of regulating drug-related apps.

PDURS explained: The FDA’s new framework for adding software to drug labels

Explore “Innovation as Strategy” framework, providing a guide for employing innovative strategies in life sciences. These expert panellists will reflect on what ‘Innovation as Strategy’ means to them, offering their perspectives, along with practical advice and real-world examples.
If you are interested in learning more about the "Innovation as Strategy" framework, get in touch with Healthware's team at healthwaregroup.com.

Innovation as Strategy: Enabling life sciences of the future

Clinical trial delivery in the UK is evolving to take trial activities to the patients, delivering research in different ways, such as remote, decentralised, virtual or hybrid trials. Join the NIHR for the chance to learn from real-life examples as panellists explore how they overcame practical challenges to establish this model and highlight opportunities to achieve more accessible research for participants and real-world evidence.

The evolution of clinical research: embracing new delivery models in the UK

Discover how an upscaled brand planning approach can shape success by overcoming challenges: from crafting effective launch plans to optimising sales strategies, uncover these tools’ full potential, driving excellence in the intricate realm of pharmaceuticals.

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Experts in clinical trial technology, design and execution will come together in a moderated discussion to explore these different facets of the opportunities created by AI, both traditional and generative.

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As conversations surrounding Equality, Diversity, and Inclusion (EDI) in health and care research participation continue to broaden, we are also expanding our scope of involvement and inclusivity. However, achieving the ambitious goal of EDI remains a challenge.
In this interactive webinar, brought to you by the National Institute for Health and Care Research (NIHR), we delve beneath the surface of EDI in research to unpick common challenges in offering equal participation opportunities in the UK.

EDI in clinical research: The case for adaptive strategies at every step

A panel of industry experts will discuss how the NCVR is transforming study set-up timelines in the UK.

Revolutionising research: Inside the NCVR evolution in the UK

Bringing together industry panellists to share insights and lessons learned from their own professional lives on successful transformation with AI and omnichannel, and the hurdles that can occur along the way.

Harnessing Omnichannel, AI, and beyond: Reimagining Scientific Publications

To improve trial recruitment, engagement, and retention, and to increase the likelihood that trials reflect what matters most to patients, regulatory authorities increasingly point to methods for incorporating patient voice in the development of new therapeutics, specifically as part of clinical trials.

Voices to Trials: Engaging Patients in Clinical Trial Design

Rare diseases often leave patients, healthcare professionals, and pharma companies navigating complex challenges, from limited treatment options to high costs and difficult clinical trial development. Cell and gene therapies (CGT) offer a unique opportunity to address these challenges by treating diseases at their genetic or cellular roots. In this webinar, sponsored by IPG Health, discover how industry insiders are unlocking the potential of CGT in rare disease care, the unique hurdles facing pharma and patients, and the transformative opportunities these therapies bring.

Exploring the future: Opportunities and challenges in cell and gene therapy for rare diseases

Rare disease

physicians

patients

caregivers

pharma companies

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Get powerful life science insights from influential experts. Connect with thought leaders and colleagues to get the most up-to-date knowledge on strategies and initiatives to accelerate the transformation of U.S. and global health care from a volume- to value-based marketplace.

Exploring the future: Opportunities and challenges in cell and gene therapy for rare diseases

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