Clinical supplies & logistics
options are included; a clear definition of the programme’s objectives; adherence to local regulatory requirements, which vary significantly between countries; and alignment with the interests and expectations of all stakeholders, including patients, healthcare providers, pharmaceutical companies, and regulatory bodies. Despite efforts to address urgent medical needs, access to these drugs in various stages of development can be a significant challenge for patients. “It’s quite nice to think ‘if I fit this criteria, I’ve got an opportunity to get the drug,’ but fitting the criteria is pretty tough,” explains Sheela Upadhyaya, an independent consultant in rare diseases. With over 20 years in the healthcare industry, Upadhyaya started her career in the NHS and has spent the past 15 years focusing on rare diseases, in roles such as commissioner of rare disease service provision at the NHS and leading a specialised technology programme at NICE to evaluate new therapies. She is a leading authority on the intricacies of rare diseases and the complexities of introducing new therapies. “No company wants to give their stuff away for free; they have to think carefully about criteria and manage the safety concerns around that compassionate approach,” she continues. Companies must consider the practicality of these programmes as they require clinicians, staff, and infrastructure to deliver treatments. They often demand significant investment even before delivering the drug to a single patient, without any guarantee of Health Technology Assessment (HTA) approval. “I think it’s really important that those access programmes are not just about ‘here’s a free drug,’ but about the wider implications for the services, clinicians, and healthcare professionals involved,” she continues. While some may view access programmes as purely philanthropic endeavours to ensure patients have the best chance for treatment, Upadhyaya emphasises the importance of broader objectives.
“I think it’s really important that those access programmes are not just about ‘here’s a free drug,’ but about the wider implications for the services, clinicians, and healthcare professionals involved.”
“From a commercialisation and market access perspective they should be thinking ‘will it provide life-saving treatment or a significant quality of life improvement for patients’, but they should also be asking what are the opportunities here from a commercial perspective?” she says. “Can we, for example, get a better understanding of the therapy delivery mechanism and pathway service configuration
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challenges? Can we understand how people will actually access the therapy and whether there would be any refinement on the eligibility criteria?” Good intentions are valuable, she says, but overlooking what an access programme can do for companies to support the next stage of a product life cycle is a massively missed opportunity. As Upadhyaya points out, it is a nice idea to think that pharmaceutical companies are in it just for the patients, but the reality is that they have investors and must achieve a return to continue investing in and creating life-changing drugs.
Optimising objectives and opportunities “If nothing else, [EAPs] provide hope,” says Upadhyaya. Patients have the opportunity to receive potentially life-saving treatments that would otherwise be unavailable, leading to improved health outcomes and enhanced quality of life for those with rare or severe diseases. But for the stakeholders involved, these programmes also offer the chance to gain a deeper understanding of the condition and engage clinicians and healthcare professionals in meaningful ways. “It’s not just the trials per se, it’s an opportunity to engage with a wider community and understand what the treatment pathway is, what challenges are associated with it, and how we can ensure that healthcare professionals, clinicians, and others in that space can use it effectively,” explains Upadhyaya. For example, while a tablet is relatively simple to administer, a more complex treatment requires ensuring that healthcare professionals and facilities are equipped and confident to deliver it at a high standard to avoid adverse events. Engaging with the entire healthcare system allows companies to not only help patients but also raise the profile of their product and company. It also supports the wider system through the implementation process once the treatment is approved for use. If planned well, EAPs offer the opportunity to collect evidence and data that add value, such as identifying centres and trained clinicians capable of delivering the treatment once approved. However, designing an EAP is one area that Upadhyaya highlights as needing a fresh approach to clearly define programme objectives and stakeholder needs to help maximise these benefits. “[Companies] need to think about ‘what will we achieve from it, why do we want to achieve it, and will it add value to our value proposition?’” explains Upadhyaya. To achieve this, it is crucial to involve all stakeholders in the design process. This includes clinicians, healthcare professionals, and patient groups who can provide invaluable insights into the practicalities of access programmes for specific patient groups. It is essential to account for the availability of expert clinicians and centres, as well as the geographic and logistical challenges that patients might face. “It’s
Clinical Trials Insight /
www.worldpharmaceuticals.net
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