Patients getting behind their disease
Written by Dr Niamh O’Reilly


Creating healthcare products to truly help improve the lives of patients should start with an understanding of their needs and the challenges they face in daily life (1). Now more than ever people have access to vast amounts of health related information due in part to advancing technology giving rise to increasingly health literate patients who are empowered to influence health care decisions (2). For their part, healthcare industry stakeholders are now recognising the benefits of involving patients throughout the product development lifecycle (2).

For example, the FDA recently launched the Patient Focused Drug Development Initiative to gain patient perspectives for more effectively informing evaluation of the risk: benefit profile of new therapies (1). While healthcare companies have traditionally involved patients mostly during postlaunch activities such as disease awareness campaigns, development of education resources and patient support programs, some companies are now engaging patients as early as the research selection phase and throughout clinical trial development, regulatory approval, reimbursement and treatment decisions (2).

There are many ways in which patients can ‘get behind their disease’ to influence improved access to quality and affordable care. Here we discuss two examples where the actions of patients and patient advocates have effectively influenced positive healthcare system changes.

Health care professionals advocating for patients

Patient advocates often have professional experience in the health industry such as in nursing and may use their knowledge of a specific disease area to help patients understand the potential benefits of various treatment options, support services and other aspects of healthcare. In this example, a team of diabetes nurse educators helped patients from Aboriginal and Torres Strait Islander communities living with type 2 diabetes to take charge of their condition (3).

A program run by the nurse educators provided patients access to continuous glucose monitoring (CGM) devices, allowing them to track the effects of food, exercise and medications on their glucose levels. As a result, patients who used CGM could access detailed graphs of their glucose levels allowing them to learn how certain foods and behaviours influenced changes in their levels throughout the day. Using CGM empowered patients to learn how to bring their glucose levels under control. The nurse educators observed many benefits among patients using CGM and have gone on to advocate for extending the program to younger patients and those with gestational diabetes (3).

Patients influencing access to therapy

Patients with rare cancers may have limited treatment options or struggle to access adequate treatment and support, which can negatively impact patient outcomes. In some cases where treatments are available, they may not be funded by payers, meaning patients need to find a way to self-fund expensive treatment or risk dying earlier from their disease. In this example one patient with a rare form of blood cancer campaigned for a decade to gain access to a potentially life saving therapy (4). Even after gaining compassionate access she continued advocating to achieve subsidised access among the wider patient community. The patient attended global conferences, lobbied politicians, rallied support from leading haematologists and a patient organisation and eventually completed a reimbursement submission (5). As a result, over one thousand patients in Australia with rare types of leukaemia will gain reimbursed access to the therapy through the Pharmaceutical Benefits Scheme (PBS) from August 2018 (6).


CRC’s experienced team provides innovative Medical Affairs solutions to assist our clients in gaining valuable patient insights that can be used to develop patient focused initiatives in achieving business goals.



  1. Lowe et al. Increasing Patient Involvement in Drug Development. 2016. Value in Health.
  2. Ings S. Pharma’s response to the patient voice. 2018. Envision Pharma. Available at: https://bit.ly/2AUBDjb
  3. Helping diabetes patients ‘see’ their blood sugar levels aids treatment. Available at: https://bit.ly/2vG0cuV
  4. Life-saving drug subsidies for thousands of patients battling rare cancers. 2018. Available at: https://bit.ly/2nzzwrw
  5. Australians living with MPN step closer to accessing Pegasys treatment. 2017. Available at: https://bit.ly/2OwZzek
  6. PBS list: https://bit.ly/2nwwQuz