There is much talk today about patient-centricity in the clinical trials, from discussions about how to ease the burden for patients, to how to empower and engage patients. But to my mind a central aspect of patient centricity that is poorly addressed is how patients and advocacy groups get better access to medical research, or even pharmaceutical companies, or how a small advocacy group would go about building a registry.

There is very little clear, understandable information in the public domain that would help rare disease patients or their carers in their search for studies or support. Many carers are struggling at home with a critically ill child with a rare disease and no one to provide them with information or support.

In my search, I came across a guide on the Patient Engagement Management Suite site that offered some good examples, with case studies and recommendations for how pharmaceutical companies and patient groups should interact. But while it was interesting, I struggled to find relevant information with any clear actions that would be useful for a lay person. The language used was complex and sophisticated, which is great for scientists and those working in the industry, but not particularly useful for carers and patients desperately seeking help.

The reality is when the industry is designing patient information sheets or instructions, they must consider the reader age of the market they are trying to reach, and in the UK and the U.S., that’s around the age of nine. That is the lay understanding that the industry needs to cater to, which can be hard for someone who understands the subject deeply. Once someone takes the time to explain to a parent or carer what they need to know about their child’s illness and what support is available, those people are extremely proactive in expanding their knowledge and advocating for their child, but they need the support from someone with knowledge about the condition or similar conditions, who is able to educate them in a way they can easily understand, to get them started.

Certainly, there is significant support through large rare disease organisations, such as NORD and EURODIS, but often these are unknown to anyone not in the industry. The information they are generating needs to filter down to those tiny groups of patients with ultra-rare, even the rare non-life-threatening diseases (which are often completely overlooked by industry and healthcare systems).

How, then, can the industry go about changing the way information is shared so it reaches those in greatest need? The fact is, now we have big data. Collectively as an industry – companies, regulators, and so on – we must start pulling all this information together and ensure it is centrally organised with a large database that covers potential symptoms and genetic links. That information must be properly regulated and managed by the regulators or healthcare systems within each country, and there must be international collaboration to ensure those ultra-rare diseases, which may only affect 10 (or fewer) people worldwide, are accounted for.

With large, easily accessible, well-managed disease databases, carers and patients can get the support they need from their local doctor, who otherwise will likely have had no exposure to a rare disease. Certainly, I have seen instances where doctors have carried out additional research and made connections that can be life-changing for that patient. However, without that central resource, many don’t get the help they need and their carers continue to struggle to gain the support and insight that might help them to, at the very least, get answers and potentially even get treatments that would alleviate their child’s symptoms.

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In a previous blog post (Linked here), I raised the issue of patient-centricity and my concern that it is being used more as a marketing tool than a way to build truly patient-centric products. To shift that thinking, there needs to be broad commitment to bringing patients into the fold at every stage of the development process.

Have patient advocates come into the company periodically to talk to the whole team – from the chief executive officer and chief financial officer to the clinical research associates, regulatory personnel and others involved in the development of the product. Get the patient to explain how the disease affects their life and what their wants and needs are. How can you design a product and put it on the market if you don’t truly understand your customer?

The fact is that those of us in this business aren’t here primarily to make money: we’re in medical research to help bring treatment to patients who need them. So, bring them in. Have them speak to your partners in the clinical research process so we can hear from patients or their carers – even if it’s by video or webinar – what they need and what their main challenges are. In so doing, it would help clinical research organisations and others to design studies better, build recruitment plans around the needs of those patients, and establish better compliance endpoints, because trials would be designed in a way that supports the needs of patients.

While discussions with patients may well be happening in the background, if everyone involved in the clinical design process doesn’t hear and can’t contribute to them, we can’t build products and protocols that meet the needs of patients.

When that starts to happen, studies will become more patient-centric and endpoints will be geared towards the needs and concerns of patients. And when that occurs, the authorities are more likely to not only be more open to approving a product, but also to reimbursing it.

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Patient-engagement or patient-centricity is now widely spoken about, but it’s rarely brought into the process of product development early on. As a regulatory consultant, my role is to fulfil the objectives of our clients as I help them navigate the clinical trial approvals process and I strongly believe that this would be a good point to bring patient organisations into the discussion. Unfortunately, this isn’t something that I have seen very often.

This demonstrates a clear disparity with the goal and the reality of patient-centricity in the current drug development landscape. I believe that true patient-centricity comes back to ensuring good study design, with end points aimed at improving quality of life based on patient insights, and a product placed on the market that puts the customer, the patient, first. Having the patient involved during early regulatory discussions would be a true reflection of patient engagement, but these vital elements are typically missing during the design stage.

While strides are being made to improve the situation, sadly, patient-centricity is still all too often used as a marketing tool, and often in unethical ways. For example, if the National Institute for Health and Care Excellence (NICE) decides not to fund a product after it has received approval, we sometimes see companies riling up patient groups to lobby against that decision, encouraging them to write to their doctors, the press, or on social media, to demand access to a drug. To me, that’s using patients at the wrong end of the process and for the wrong reasons entirely, and it’s the kind of behaviour that gets called out time and again earning so-called ‘Big Pharma’ it’s bad reputation.

If a company is developing a product for a rare disease, for example, what they could be doing is bringing in patient advocates early on and asking what matters most to them. Which aspects of their disease do they wish they could overcome or manage better in order to improve their life? It could be something as simple as having fewer blood tests or being able to have these done from home, so they don’t have to visit their doctor so often. A simple change in formulation to make the product slower release might address an issue like that, not only improving the product for the patient, but the knock-on positive effects on study compliance and thus on the clinical data generated also seem a no-brainer.

By talking to patients from the outset, companies would be in the best position to create not only a study design with the most relevant end points, schedule and logistics, but deliver a product (from formulation, to dose schedule, to packaging, and more) based on meaningful quality of life considerations. That would be true patient-centricity.

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