When faced with a diagnosis of a chronic condition, such as diabetes, hemophilia, rheumatoid arthritis or multiple sclerosis, a patient starts a life-long journey of care, compliance and mostly likely, complaints. After the initial shock has worn off, patients may experience a sense of relief that the cause of their health issues has been found. But many will respond with deep-seated emotions, such as anger or depression. The need for life-long medication may be met with denial, fear or anxiety. To learn the tasks associated with caring for the condition can be daunting, and the difficulty level can increase, depending on severity.

Home-based care offers patients a certain amount of freedom, which may help them feel more comfortable. However, difficulties with delivery and administration may also lead to non-compliance. In order for a drug product to be truly effective, it must be administered properly and the appropriate treatment regimen must be adhered to. Yet adherence to long-term therapy for chronic conditions is a painful and stressful daily routine for many patients. According to the World Health Organization, adherence to long-term treatment recommendations hovers at just 50% in developed countries. The cost to the healthcare system can be significant, and the impact on pharma companies can be huge, not least in terms of lost revenue due to non-compliance.

For most, an easy-to-use, integrated delivery and administration system will be essential. An integrated system combines the drug, its primary containment system and its delivery system. While many products do this reasonably well, a truly successful combination product must also consider the needs of the end-user at a variety of stages during the patient journey. With an initial diagnosis, a patient may feel relief or shock — attitudes that can improve or impair the likelihood of adherence. With early treatment, the patient is acclimating to the prescribed therapy, so instructions must be clear and a delivery device or administration system simple to use. For a patient with hemophilia, a single slip of a needle may cause a life-threatening situation and should be avoided through the use of needle-free preparation techniques. All of these needs must be understood and incorporated into requirements for effective devices.

As the patient becomes more comfortable, needs change. Aspects of device use that may have been appreciated during earlier stages, such as ease of learning and demonstrability, will likely wane in importance when users become more experienced with operating the device or reconstituting the drug product. Patients will become likely to make new demands of the device or administration system around convenience of use, and the impact that the prescription therapies have on lifestyle and quality of life. Devices that are safe, quick and efficient to use, especially regarding the time required to prepare and actuate delivery, stand a better chance of encouraging adherence because the impact on daily routines becomes negligible. Discreetness of the device also becomes more important. A discreet device provides its user with a greater range of options for how to integrate the dosing regimen into daily life. It also enables use without calling undue attention to the device, creating distractions to others or feelings of stigmatization. Delivery and administration systems deemed inconvenient or overly conspicuous can negatively affect a patient’s emotional attitude and motivation to sustain adherent behavior. Later, as the patient ages or degrades, lapses in therapy may occur due to the physical and emotional burdens of the condition.

By focusing on the relationship between the delivery system design and the patient interface at various stages, and by designing a device or system that meets end-user needs at these stages, pharma manufacturers stand a better chance of satisfying the emotional and physical needs of the intended user/patient throughout the course of treatment. By applying best-practice user-research methodology, pharma manufacturers can gain insight into a user’s preferences and emotional requirements, and those findings can translate into feature sets and design elements of the combination product. By gaining detailed information of the emotional and physical needs at different points in the patient journey, pharma manufacturers can design a product that not only suits a patient’s need for medication, but is also aesthetically, functionally and emotionally relevant. Such a shift from a product-centric focus to a patient-centric focus may help manufacturers design a product that encourages adherence — and brand loyalty — in crowded market segments.

Building a Better Delivery System
Many newer biologic drugs face significant challenges, which can impact container and device selection. Sensitivity to certain materials such as silicone oil, metal ions or extractables/leachables will drive the need for a container system that minimizes adverse impact of containment materials over time. Quality concerns regarding glass particles and delamination are driving increased interest in polymer-based systems. In addition, many newer biologics require higher concentrations, leading to higher viscosity and/or higher dosage volumes. In such cases the options include multiple doses of a smaller volume within 10 seconds with a syringe or auto-injector, or a slow injection of a higher volume through a patch injector technology. These factors should be considered at an early phase in the development process; they can be critical in ensuring the optimum patient experience, highest quality, and adherence to a treatment regimen. 

Understanding all key elements of a drug delivery system provides the cornerstone that enables this system to achieve the goals of encouraging adherence in the home-care environment. The FDA has provided recommendations for medical device design optimization through human factors analysis, testing and validation. The methods offer an effective way to understand use-related hazards and optimize how people interact with the technology, as it relates to the three major components of the device-user system:

• device users
• device use environments
• device and/or device user interfaces

In-depth statistical analysis, data aggregation and synthesis techniques can yield actionable opportunities for innovation and a thorough understanding of the potential users’ behaviors, motivation and needs. Human factors experts can help define and prioritize the needs of the intended users and determine what else needs to be learned about user and device interaction. Common methods include qualitative, quantitative and human factors/ergonomics analyses. Discovery research, usability testing, and directional and preference testing offer a strong framework for the development process. Customers’ needs and desires can be confirmed through discovery. Directional research allows users to evaluate product concepts, and usability testing helps to ensure that the delivery solution is user-appropriate.

It is also helpful for human factors experts to interview the user in the proper context or environment of use. Someone sitting in a conference room will have a different user experience than a stay-at-home mother who is also taking care of an aging parent. It may be helpful for human factors experts to see the user in the midst of daily distractions such as children, pets, ambient noise, temperature and lighting. This will help those experts get the most relevant data and better understand how the patient will use a device, as well as the many factors that may affect use. Usability may also change over time as the patient becomes more accustomed to a device.

To get the richest data, human factors experts must follow the patient on his or her journey — from diagnosis to end of therapy — and then translate the multitude of qualitative and quantitative data into features of a product that will not only provide a patient with safe and effective delivery, but also meet the emotions, needs and desires of the user at different stages of disease management.

Ensuring Safety
In the home environment, patient safety extends beyond the individual to the family and anyone involved in caregiving.  For patients diagnosed with hemophilia, needle safety can be a life-or-death issue. In addition, if used, stored or disposed of incorrectly, devices containing a needle may cause a needlestick injury for caregivers and/or family members.

While the industry has seen advances in needlestick prevention thanks to legislation in both the U.S. and Europe, such mandates are limited to the clinical environment. For home-based care, ongoing development can help to create safer systems and practices. Systems that incorporate a safety mechanism, or those that are completely needle-free, can lower or eliminate the risk of accidental needlesticks, help to reduce the spread of infectious disease and reduce costs associated with care as well as help to ensure the safety of family members and those who may come in contact with the system during disposal.

The ideal system should ensure that needle protection is triggered automatically at any point after the needle is inserted into the body and before completion of the full dose. This would ensure protection even if the dose is partially administered. For those suffering from hemophilia, additional steps that require the use of a needle may place a patient in harm’s way.  The use of needle-free devices can help to prevent problems by enabling the patient to avoid contact with a needle. Vial adapters, needleless transfer devices and systems such as Mix2Vial^® can facilitate the rapid transfer of diluent into vials, syringes or intravenous infusion bags. Such products allow for convenience and ease of use without the added danger of a needle.

Patients start their journey with an initial diagnosis, but they travel a long road with a chronic condition. Pharmaceutical and delivery systems manufacturers must start the development journey with that end in mind, and create systems and options that will not only help patients learn to care for their condition, but also comply with their prescribed treatment regimens throughout their course of care. That journey begins with the patient in mind and travels a course of testing and development that will ultimately aid the patient, no matter where they are on their journey.

Drug manufacturers should seek a partner that can apply proprietary technologies, manufacturing excellence and patient understanding to their drug products and the products’ delivery and administration systems. Such partnerships will help drug marketers offer successful, integrated solutions, benefitting manufacturers, clinicians and patients alike, while helping to ensure optimum adherence  and improving patient outcomes. CP

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Graham Reynolds is vice president, Marketing and Innovation, at West Pharmaceutical Services, Inc. (westpharma.com). He can be reached at [email protected]. Mix2Vial^® is a registered trademark of Medimop Medical Projects, Ltd., a subsidiary of West Pharmaceutical Services, Inc. Daikyo Crystal Zenith^® is a registered trademark of Daikyo Seiko, Ltd. Daikyo Crystal Zenith^® technology is licensed from Daikyo Seiko, Ltd.