Clinical trials are the backbone of medical advancement, paving the way for new treatments and potential cures. Despite this, many patients in urgent need of novel therapies face the challenge of accessing treatments that are still undergoing testing or have yet to be approved in their country. Access programmes for clinical trials, also known as early access, compassionate use, or expanded access programmes (EAPs), are designed to bridge this gap. These programmes not only hold promise for patient recovery but also offer substantial benefits for a wide range of stakeholders in the healthcare ecosystem.
Ever since the Investigational New Drug (IND) application was established in the US in 1987 in response to the AIDs crisis and growing demand for drugs in development, EAPs have evolved significantly worldwide.
This reflects a growing commitment to patient-centric healthcare. When no other options are available to patients with serious or life-threatening diseases, these programmes provide pathways for patients to access drugs in development when there are no other alternatives.
Compassion and commercialization
Typically, EAPs consist of several key components: patient eligibility criteria to ensure that those who genuinely need the treatment and have no other 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 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, access programmes 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, access programmes 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 all very well saying ‘you can access it,’ but not everyone can if it’s too far away.” If the treatment centre or trial is in a remote location, many patients who could benefit will inevitably be excluded. This underscores the importance of designing programmes that are not only inclusive but also practically accessible, ensuring the promise of EAPs translates into real-world benefits.
To define the programme’s objectives, pharmaceutical companies need to consider what can be learned from EAPs and how that information can be used. “It’s amazing how much information is captured in this drug development journey and how much of it just gets filed away and nobody thinks about it,” adds Upadhyaya. Asking the right questions and engaging with the right stakeholders is important, but the mechanism for collecting data and evidence is essential for making the programmes as efficient and streamlined as possible, while ensuring that no valuable information is lost. With the magnitude of data already available, Upadhyaya stresses that there is no need to “reinvent the wheel” but to build on existing systems where data is already being captured. “Rather than say ‘here’s another tool or another app,’ we can build on something they’re familiar with and don’t need to log in and remember another password,” she explains. “From a practical perspective, we know how frustrating it is to go from one website to another.”
Revisiting the drawing board to identify the key objectives and goals for EAPs is critical to ensuring that the programmes offer value for all stakeholders and create the best access programme for patients. “My biggest frustration is that there is a lot of stuff that goes on in pharma that they have done for years without thinking about the value that it adds to the overall opportunity of access,” Upadhyaya continues. However, despite spending money on resources, the information gathered is often not used and just adds to the cost of the product. “If we didn’t need to do it, one, why did we waste everyone’s time, effort, and energy? And two, if we didn’t do it, we wouldn’t need to add that cost onto the price of this really expensive drug.” To avoid this unnecessary cost, companies need to collaborate with their shareholders to identify needless costs and exercises. It is not enough to tick a box, Upadhyaya explains; companies need to listen to what is being said and take it on board to help improve their EAPs.
Authentic and accurate insight
Looking ahead, EAPs are likely to become more integral to the development of new treatments. To capitalise on this, programmes need to become more flexible to be accessible to everyone. Incorporating digital health technologies and data collection tools is one way Upadhyaya suggests could enhance patient access and streamline programme administration. This could include the use of mobile apps for remote monitoring, telemedicine consultations, and digital platforms for data submissions and analysis.
One example Upadhyaya uses to demonstrate the possible benefits of using digital solutions is the ability to capture real-time experiences of patients. During a clinical trial, many factors could influence a patient’s participation in some tests, especially with children, who may be coerced or bribed to engage in activities they might not usually undertake. This can lead to outcomes that might not be seen as credible or unbiased. However, remote monitoring, such as a wearable device, allows clinicians to observe and record physical activity in a natural setting free from these biases. This approach provides a more authentic and accurate insight into patient behaviour, enabling the collection of valuable and reliable data.
“My biggest frustration is that there is a lot of stuff that goes on in pharma that they have done for years without thinking about the value that it adds to the overall opportunity of access.”
Moreover, Upadhyaya notes that digital monitoring tools often yield positive unintended consequences. For instance, she recounts a case where a company involved in real-world evidence collection discovered that the therapy significantly reduced infections and emergency admissions among children. This unexpected finding indicated that the therapy not only improved health but also decreased the need for antibiotics and inpatient stays, leading to substantial cost savings and better overall patient care. “This is an underutilised opportunity,” Upadhyaya emphasises. By incorporating digital health technologies into clinical trials and patient care, companies can uncover additional benefits that extend beyond their original objectives, thereby enhancing patient outcomes and contributing to a more efficient healthcare system.
Ultimately, the future of expanded access programmes holds the promise of improved patient outcomes, accelerated medical innovation, and enhanced collaboration across the entire healthcare ecosystem. However, in order to truly capitalise on these opportunities, it is imperative that these programmes are designed to maximise their benefits for all of the stakeholders. By addressing current challenges, such as clear objectives and using data effectively, while embracing new opportunities that digital health technologies can offer, EAPs can become more effective and inclusive of the needs and contributions of all stakeholders.
