Events

Reflecting on the patient experience in clinical trials: Insights from a panel discussion

12th November 2024

Barcelona, Spain

The landscape of CAR-T therapies and advanced treatments is undeniably complex, requiring the collaboration of numerous stakeholders throughout the entire value creation process. As we navigate this intricate ecosystem, it's essential to remember that the ultimate focus of our efforts is the patient. In a recent panel discussion moderated by Dr. Jorge Sarroca, the importance of the patient experience emerged as a central theme, prompting vital reflections on how we communicate and engage with those affected by these therapies. Dr. Sarroca posed a critical question to a physician specializing in patient care—particularly for children with complicated cases—about the level of information patients and their families possess. Are they well-informed? Do they arrive with preconceived notions? And how easy is it to convey essential information to them? The physician shared her insights specifically regarding CAR-T therapy, highlighting its complexity and the high expectations surrounding it. She emphasized the importance of contextualizing this therapy, ensuring that both patients and their families fully understand the process. Taking the time to explain the intricacies—sometimes dedicating up to two hours—can make a significant difference in how patients perceive their options. In today’s digital age, patients often encounter a wealth of information online, which can lead to confusion. It falls upon healthcare providers to guide them through this maze. Unfortunately, many patients who appear to be candidates on paper may not qualify due to other health conditions or the aggressive nature of their diseases. This reinforces the necessity of personalized communication and education tailored to each patient's situation. Another panellist elaborated on the challenges of informing patients about clinical trials. The term ‘clinical trial’ can evoke fear, as many individuals associate it with being mere test subjects or ‘guinea pigs’. This fear is often compounded by a general scepticism regarding the pharmaceutical industry’s motivations, leading patients to question what they stand to gain from participation. Clearly explaining what a clinical trial entails—including the informed consent process and the various stages involved—is crucial. Patients frequently arrive with a mix of information and expectations derived from others’ experiences, which can complicate their understanding. Thus, managing these expectations becomes one of the greatest challenges in medicine. The physician noted that in CAR-T trials, concerns about toxicity can further amplify these issues. Therefore, having a specialized, multidisciplinary team is vital. Patients often feel more at ease discussing their concerns with trial coordinators than with physicians alone, highlighting the importance of having a supportive network in place. In this context, patient involvement is indispensable. Their insights can shed light on aspects of care and communication that professionals might overlook. As we advance in developing innovative therapies, it is imperative that we educate patients about potential risks and ensure they have access to comprehensive information and support. Ultimately, reflecting on the patient experience in clinical trials reminds us that their voices must be integrated into every stage of the process. By prioritizing patient engagement and understanding, we can create a more transparent and supportive environment that fosters trust and encourages participation in these groundbreaking therapies. As we move forward, let us commit to listening to patients, valuing their perspectives, and striving for better communication in the ever-evolving realm of advanced therapies.  

Collaborative innovations and patient-centric approaches in Cell and Gene Therapies

19th September 2024

Barcelona, Spain

On September 19, 2024, the Alcura Summit convened in Barcelona, bringing together leading experts, industry stakeholders, and patient advocates to discuss the latest advancements and challenges in cell and gene therapies (CGT). The event highlighted the critical role of collaboration across various sectors to enhance patient outcomes and drive innovation. Serving as a vital platform, the summit emphasized the importance of regulatory adaptability and patient empowerment, addressing the multifaceted challenges within the CGT landscape. Participants engaged in meaningful discussions aimed at fostering an environment conducive to innovation, culminating in a call to action for continued dialogue and partnership within the CGT community. Opening remarks Remedios Parra, Managing Director of Alliance Healthcare Spain, emphasized the need for patient-centric approaches in CGT and the importance of flexibility when it comes to unique therapies and Lung-I-Cheng, Vice President of CGT at Cencora spoke extensively about "Transforming Cell and Gene Therapy (CGT) for Global Impacte. He adressed this critical topic in detail, highlighting the advancements and potential global impact of these transformative therapies. Ten essential takeaways Global trends and policy impact Paolo Morgese, Vice President of Public Affairs at the Alliance for Regenerative Medicine (ARM), highlighted the ongoing changes in policies affecting CGT. He emphasized the importance of collaboration among industry stakeholders, including small and large companies, technology providers, and patient organizations. The need for a supportive policy environment is crucial for the growth of CGT. Investment discrepancies Paolo highlighted the significant gap in investment between the U.S. and, Europe and Asia, indicating that many European companies seek funding in the U.S. to drive their innovations. He called for stronger investment ecosystems in Europe to bridge this gap. Clinical trial landscape The summit revealed a notable gap in early-phase clinical trials between Europe and the U.S. While Europe shows strength in later-phase trials, the lack of early-stage trials may hinder the region's ability to innovate effectively. The trend toward later-stage investments encourages stakeholders to prioritize obtaining more robust data before allocating resources, underscoring the need for companies to establish thorough commercialization strategies. Patient-centric approaches A recurring theme was the critical role of patient involvement in therapy development. Discussions emphasized that understanding patient perspectives can enhance trial designs and improve outcomes, with patient advocates calling for their voices to be included in decision-making processes. Challenges in regulatory frameworks The regulatory landscape for CGT was identified as a barrier to rapid development. Participants suggested that adaptable regulatory frameworks are needed to accommodate the unique characteristics of cell and gene therapies compared to traditional pharmaceuticals. Collaboration across sectors The importance of collaboration among academia, industry, and patient organizations was stressed. Shared efforts can lead to better results in developing and distributing therapies, with successful case studies illustrating the benefits of such partnerships. Patient empowerment Patient empowerment emerged as a vital component in the CGT landscape. The need for accessible information and support for patients navigating complex treatment options was highlighted, with advocates demanding increased patient representation in clinical trials and therapy discussions. Advancements in trial design Panel discussions included insights on innovative clinical trial designs, especially in rare diseases. The focus was on creating personalized treatment plans tailored to individual patient characteristics, underscoring the need for flexibility in trial methodologies. Value-based pricing models Participants noted the increasing importance of understanding the value CGT brings to healthcare systems. Discussions on value-based pricing models highlighted the need for sustainable pricing strategies that reflect the long-term benefits of these therapies. Patient experience A dedicated panel discussion explored the patient experience, featuring healthcare professionals and patient advocates. They reiterated the emotional complexities of treatment, emphasizing the need for effective communication and support for both patients and their families. Future outlook Looking ahead, speakers expressed optimism about the future of CGT, emphasizing that continued innovation and collaboration can lead to breakthroughs that significantly impact patient lives. Ongoing education for healthcare professionals and patients was deemed crucial for successful therapy integration.

Alcura summit 2024

19th September 2024

Barcelona, Spain

‘Overcoming hurdles to promote advancements in Cell and Gene Therapies’