Written By: Paula DeCola
September 8, 2019
The medicines development lifecycle has historically been remiss with respect to incorporating patients in the process. The exception to this is the inclusion of certain groups within clinical trials. However, the engagement of patients in the process of medicines development is currently burgeoning. Patient advocacy groups have in large measure provided the impetus for this culture shift. They have been joined by the biopharmaceutical industry, healthcare professionals, regulatory authorities and academia. Furthermore, a number of national and international initiatives focused on defining aspects of patient engagement have emerged. The overarching aim of these initiatives is to define aspects of patient engagement and help to move it from an ad hoc event into a standardised practice — from idea inception through to medicine use and beyond.
The economic benefits associated with patient engagement in terms of development time and quality components have been effectively modeled. Involving patients in the medicines development lifecycle is an inherent good; indeed, the societal price of their exclusion is one too high for all stakeholders to pay. When we think of value-centred health care as a whole, it logically requires the patient to be part and parcel of the entire system.
A specific area where patient involvement has significantly lagged behind is in the evaluation of medicines. The most common approach assesses a medicine’s cost-effectiveness. This involves determining the cost per quality adjusted life-years gained.1 The analysis does provide a way to compare therapies, but it does not consider value drivers that are defined by patients and have broader implications.
Aspects of value that promote healthier lives such as symptom alleviation, retention of functionality and other well-being criteria are often cited by patients as key elements of value. These types of valuation and the priorities of patients with different diseases may not be the same as those anticipated by regulators, payers or biopharmaceutical companies.
The benefit-risk tolerability of a medicine can vary for patients across therapeutic areas and conditions. Intangible criteria, such as hope, which can take different forms, can be of significant value in light of this point. An example of hope that was relevant in the early treatment for HIV/AIDs was the ability to stay alive long enough for the development of successful treatments. While on the surface such development may be viewed as benefiting an individual alone, the benefit extends to society as a whole.
Another example of value that extends beyond the patient to others is demonstrated by the higher rates of hospital admission and mortality of partners of patients who have died.2,3 Additionally, decreased work levels and increased rates of depression have been found in providers of care to certain terminal cancer patients.4 Treatment of these cancer patients can result in a decreased burden to their care providers. It has been demonstrated that some therapies are found to be cost-effective when these aspects are taken into account in a medicine’s value assessment.5
Embedding the perspectives of patients throughout a medicine’s lifecycle into the development process will likely provide a competitive advantage since the resulting medicines should better address the needs of the intended community. It is especially important that value determinations are broadened and incorporate patient and societal considerations since a medicine’s value hinges on its accessibility to the patients who need them.
As we look to expand the role of patients in medicines development, we must be vigilant that we include a diverse and representative population that reflects the disease under study. We must not repeat the historical underrepresentation of women, certain racial and ethnic groups, and older people that we have seen within clinical trials.
As an example of the rationale for inclusiveness, let us look at women. Including women points to a recognition that gender plays a role in health and disease. Biological and sociocultural constructs can play a role in risk, presentation, treatment and management of disease. Therefore, it is important to include women in all the phases of medicine development. This holds true for other underrepresented groups. Furthermore, we know that women play a broad role within health care. In the USA, they make up more than half of the population, provide about two-thirds of caregiving within their families, and make about three-quarters of family health care decisions.6 No doubt these statistics are mirrored in other countries and regions. Their special role further makes the case for women to be appropriately represented.
Overall, there is significant interest in further involving patients across the development continuum and, as noted, this inclusion should be diverse and representative. This is the right prescription to move to a more balanced and value-centred healthcare system for all.