Patient-centric drugs are widely recognised as being fundamental for optimising health and well-being in individuals. In addition to selecting the appropriate drug type and strength, the product design must also sufficiently address patient needs. This includes consideration of the disease, routes of administration, active pharmaceutical ingredients and drug delivery technologies. The latter has received less attention in the industry but forms a crucial element of patient centricity. Some have referred to patient-centric design as the ‘next frontier’ in drug delivery.

Despite the widespread discussion of the concept, a universal definition does not exist. For John Burke, a senior consultant specialising in parenteral drug delivery devices at Team, it is about the mentality. “Putting the patient and their needs front and centre, whether that is in the development of a novel drug or innovative delivery device,” says Burke. “Ultimately, it’s a shift in mindset; rather than developing medicines for patients we should challenge ourselves and ask how we can develop a therapy or delivery device in partnership with patients.”

Stand out

There are a huge number of advantages in embracing the concept, both for patients and pharmaceutical companies. “It stands to reason that putting the patient at the centre should lead to therapies, devices and services that better meet their specific needs and capabilities,” says Burke. “In an increasingly competitive marketplace, getting this right will make your product stand out.”

This means embracing a collaborative approach. “Consider two competing biosimilars, which from a purely therapeutic standpoint are identical,” Burke continues. “By working with patients to better understand their capabilities, challenges and concerns, we can offer a much improved user experience.”

Small changes can make a big difference in this context. “This might be a small iterative change such as improving the quality and clarity of the information provided through the packaging or instructions for use,” explains Burke. “Alternatively, it could involve tailoring the delivery device to meet the needs of challenging groups such as paediatric or geriatric patients with reduced physical capabilities or limited dexterity.”

Ultimately, the idea is to think holistically about what it is that matters to patients and making adjustments accordingly. “The aim is to provide a better and more relevant product or service in order to deliver a great user experience,” says Burke. “This may in turn lead to patient preference or improved compliance.”

Navigate challenges

Adopting a patient-centric approach also involves navigating a number of various challenges. One of these is the distance from patients. “Earlystage R&D within pharma is traditionally quite disconnected from the realities and real-world experiences of patients self-administering therapies,” says Burke. “This can lead to molecules being developed with little to no consideration as to how this can be consistently administered outside of a controlled clinical setting.”

This separation is the norm in the industry, but it does not have to be. Companies are already starting to change their ways of working to facilitate better communication and collaboration with patients. “Increasingly we’re seeing large pharma companies setting up user experience groups responsible for championing the end user within the organisation,” says Burke. “They can provide invaluable insights from existing marketed products and patients, and feed this into early-stage drug discovery and development. Opening up these channels and lines of communication within large global organisations is challenging but early input from both clinical and user perspectives is critical in order to identify potential opportunities and maximise the benefits of a new therapy.”

“Rather than developing medicines for patients, we should challenge ourselves and ask how we can develop a therapy or delivery device in partnership with patients.”

There are certain drug delivery devices where it is particularly difficult to implement a patientcentric approach. One example is platform delivery devices, which are becoming increasingly popular within the industry.

“Putting your drug into a proven device goes a long way towards mitigating development risk, and can significantly reduce time to clinic and eventual commercial launch,” explains Burke. “However, there are potential downsides to this approach as platform products offer limited opportunity for customisation or changes. In this case, tailoring the device to your patients may be prohibitively expensive in terms of cost and development time.”

“We try to involve the user as early as possible within any development. Typically, we undertake exploratory design research with patients to understand their experiences, attitudes to the condition, therapy and existing product offerings.”

This was demonstrated in 2015 with the battle to see which PCSK9 (Proprotein Convertase Subtilisin Kexin Type 9) would be first to market: Amgen or Sanofi-Regeneron. Sanofi-Regeneron won with Praluent, which was followed by Amgen’s Repatha a few months later.

“Surprisingly, these competing drugs were initially launched in the same device,” says Burke. “The opportunity to differentiate these competing therapies through device design or user experience improvements wasn’t possible in the race to launch.”

The way to success

When it is possible and practical to implement a patient-centric approach, there are a number of best practices to increase the chance of success.

“We try to involve the user as early as possible within any development,” explains Burke. “Typically, we undertake exploratory design research with patients to understand their experiences, attitudes to the condition, therapy and existing product offerings.”

This sometimes means going above and beyond the call of duty. “We often visit patients in their homes to see first-hand the realities of living with and managing a chronic condition,” says Burke. “It’s not uncommon to see a fridge dedicated to storage of temperature-sensitive medications or huge boxes containing different medications required to manage a complex condition.”

It is also key to continue to ask questions throughout the entire process. “We seek to identify the key ‘pain points’ or challenges faced by users and turn these into opportunities,” adds Burke. “For example, ‘How might we simplify the management of this condition’ or ‘How might we reduce the pain experienced during injections’. This in turn guides the concept generation activities, and informs the key decisions and forward strategies.”

What does the data say?

In addition to engaging with patients directly, big data can also provide value throughout the process. This includes vast volumes of information as well as predictive medical analytics. Electronic medical records from hospitals and medical data from social network services can elucidate patient needs and help to improve the efficiency of drug development.

A good example where patientcentricity is working well in practice is Amgen’s Neulasta OnPro device. Following extensive research with patients, Amgen relaunched its market-leading white cell boosting Neulasta in a custom version of the OmniPod device, called OnPro.

“In doing so, they not only raised the bar for biosimilars in development but offered clear benefits to users,” explains Burke. “Following a strong dose of chemotherapy, users are able to stay home, rather than visit their doctor the following day to receive the injection.”

It is not always easy to implement this approach but it is clearly worth the time and cost investment. With momentum growing within the industry, this will become increasingly common, bringing benefits to industry and patients alike.

“I believe the outlook is positive, we’re seeing more and more companies engaging with users early in the development and translating these insights into their marketed products,” says Burke. “My hope is that as the launch of innovative patient-centric delivery devices and therapies start to shape the market, other companies will see the value and opportunities offered by engaging users early.”

Principles for increasing patient involvement in drug development

  • Acknowledge the importance of patient involvement: define what it means to truly incorporate patients as partners.
  • Take inventory: review existing efforts and work with patients to determine when patients should be more included.
  • Be transparent: own up to instances when this could have been done better, both internally and to external stakeholders such as patients, payers and regulators.
  • Trust patients: invest in them and take the time to educate them on issues and needs.
  • Involve patients continuously, early and often: encourage involvement across all phases of product development.
  • Treat patients as collaborators: they should be seen and treated as equal stakeholders and not as a target market.
  • Display leadership: be ambassadors for this cause for the rest of the industry.
  • Be the change you want to see: proactively provide patient-generated perspectives and evidence to the regulators, payers and market access gatekeepers.
  • Share your lessons learned: document failures as well as successes.
  • Collaborate to develop best practices: share tool kits with your peers.

Source: Value in Health