Acceptance studies: the missing link in assessing adherence?
Benoit Arnould
Senior Director, Global Patient-Centered Outcomes, Mapi HEOR and Strategic Market Access
Remi Gauchoux
Senior Director, Global Business Development Operations, Mapi Real-World Evidence
Carla Dias Barbosa
Associate Research Director, Mapi HEOR and Strategic Market Access
Hélène Gilet
Senior Research Manager, Mapi HEOR & Strategic Market Access
How do patients’ experiences regarding their medical treatments impact their willingness to adhere to them and persist with them?
The idea that patients with chronic diseases make a kind of judgment—whether or not their medications are worth taking on a long-term basis—is relatively common among healthcare professionals, doctors, pharmacists, and nurses. Patient-Centered Outcomes research specialists have explicitly assessed acceptance on several occasions.1,2 Regulatory authorities have shown interest in this concept, In fact, the EMA has made it mandatory to ensure that a child’s acceptance is assessed, with clear and explicit expectations regarding the detailed conceptual content of “acceptance.”3 In addition, there is at least one example of a regulatory authority including such an outcome in a Market Authorization Label4 (See Table 1).
In their real-world studies, Mapi teams frequently have had to identify the best available instruments that capture “something” that would be close to satisfaction and adherence. Yet this “something” should probably not be satisfaction, which is too easily modified by the management of patient expectations and has limited credibility with regulators and payers. Neither should it be adherence, which faces measurement issues and is too frequently problem oriented rather than solution oriented.
An array of methods for measuring adherence exists, but none of them meet the criteria to be a “gold standard.” Consequently, non-adherence rates are highly variable depending on the measurement methods used; that limits the interpretation of the results. Compliance measures are designed to assess how well patients take their medication but they fail to give any clue as to why they don’t. Last but not least, adherence studies face barriers in recruiting non-adherent patients.
Mapi decided to fill in this gap. Our aim was to develop a measurement tool that we could offer industry and academic researchers for their pharmaco-epidemiology studies. That tool was expected to measure a new concept explaining adherence and persistence, to focus on medical treatment attributes and to be completed by lay patients in real-world situations. That’s why Mapi has developed ACCEPT©, a generic scale assessing long-term medication acceptance and its determinants.
This has been an importance piece of work.5,6 Many challenges had to be addressed:
What is the underlying concept?
Does it really exist from a patient perspective?
Is it supported by grounded research?
In addition, we asked ourselves how do we create questions that cover all the practical features of medications yet make sense to patients; in other words, can we be concrete and also generic? How do we create answer choices that are easy for all patients to respond to, that are clearly related to the concept of interest, and that can capture differences? And how do we assess both the level of acceptance and the characteristics of medication that explain this level of acceptance?
We are proud to have now a scored, validated Acceptance measure, based on the best development standards. Today, this brief questionnaire—25 items completed in less than 10 minutes—is rich and detailed enough to assess and explain acceptance levels. There is evidence that acceptance is correlated to adherence, but is a separate concept. ACCEPT© has been validated according to Mapi standards in 16 languages (see Table 2). Studies are ongoing on specific populations (those with multiple sclerosis and who have had renal or liver transplants). The results of a large study—conducted via the social media website Carenity that included more than 3,000 patients suffering from a large variety of chronic conditions—will be shared for the first time in an international congress at ISPOR Amsterdam this fall. This study confirms that acceptance levels and their determinants vary substantially according to the disease.
ACCEPT© is expected to help researchers, clinicians, and industry professionals in several ways:
- To identify patient priorities and unmet needs
- To compare treatment performance in real life, from a patient’s perspective
- To provide clues as to why patients show limited willingness to follow their treatment
- Ultimately, to explain adherence and predict persistence.
There is a growing recognition of the importance of involving patients in the evaluation of medicines and taking into account the patient’s voice during a drug’s development, particularly in the benefit-risk assessment (BRA) of medicines. ACCEPT© is a promising tool to assess, at the individual level, how perceived risks and benefits of medicines are balanced by patients to form drug acceptance.
For more information about ACCEPT©, please contact: Benoit Arnould, benoit.arnould@iconplc.com
References
1. Chevat C, Viala-Danten M, Dias-Barbosa C, Nguyen VH. Development and psychometric validation of a self-administered questionnaire assessing the acceptance of influenza vaccination: the Vaccinees’ Perception of Injection (VAPI) questionnaire. Health Qual Life Outcomes 2009;7:21.
2. Schlarb AA, Brandhorst I. Mini-KiSS Online: an Internet-based intervention program for parents of young children with sleep problems—influence on parental behavior and children’s sleep. Nat Sci Sleep 2012;4:41-52.
3. European Medicines Agency. Guideline on pharmaceutical development of medicines for paediatric use. EMA/CHMP/QWP/805880/2012 Rev. 2. London, August 2013. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2013/07/WC500147002.pdf (accessed on August 20, 2014).
4. Therapeutic Goods Administration. INTANZA Product Information – ARTG ID 150130. Australian Register of Therapeutic Goods. http://search-au.funnelback.com/s/search.html?collection=tga-artg&profile=record&meta_i=150130 (accessed on September 3, 2014).
5. Marant C, Longin J, Gauchoux R, Arnould B, Spizak C, Marrel A, Patrick DL, Van Ganse E. Long-term treatment acceptance: what is it, and how can it be assessed? Patient 2012;5(4):239-49.
6. Arnould B, Gilet H, Patrick DL, Gauchoux R. Validation of the ACCEPtance by the Patients of their Treatment (ACCEPT) questionnaire. In preparation.