In this feature, Anke-Peggy Holtorf (Health Outcomes Strategies, Basel, Switzerland) discusses her presentation from HTAi (15–19 June 2019, Cologne, Germany) on the current and future states of value-based decision making.
Please could you introduce yourself and your institution?
I am the Managing Director and Market Access Consultant at Health Outcomes Strategies (Basel, Switzerland) as well as an Assistant Professor at the University of Utah (UT, USA).
Together with Diana Brixner (University of Utah), ZoltÃ¡n KalÃ³ (Center for Health Technology Assessment at the Semmelweis University; Budapest, Hungary), Nikos Maniadakis (National School of Public Health; Athens, Greece), and other global health policy and health economics experts, I helped develop the Initiative on Value-Based Decision Making for Pharmaceuticals in Emerging Markets. Specifically, we were interested in improving decision making concerning off-patent products [1, 2].
What is the importance of value-based decision making with regards to pharmaceutical policies, particularly in countries with expanding healthcare coverage?
Countries with expanding healthcare coverage, which are mostly low- and middle income countries, work under extremely tense conditions: they would like to expand patients’ access to healthcare, including pharmaceutical products, but traditionally had relatively small budgets available for this. These budgets can only be slowly expanded if the economic situation allows.
In addition, the public health systems of countries with emerging healthcare systems often do not have the full range of regulatory frameworks available to guide them, which have been built up in mature healthcare systems over a long time.
…the more that prices are downregulated in these environments, the more compromises we observe in the quality or availability of products…”
Further, whilst most marketed pharmacotherapies in countries with expanding healthcare coverage are established (off-patent) pharmaceuticals, there is an increasing demand for more expensive, innovative therapeutics — for addressing medical needs that cannot be met with off-patent pharmaceuticals. Therefore, many countries introduce measures for regulating prices down to a minimum, especially for products with generic alternatives where competitive market forces can be applied.
On top of all of this, the desire for countries’ economic growth and independence can lead to their prioritizing production of local products, which often have lower acquisition cost.
However, the more that prices are downregulated in these environments, the more compromises we observe in the quality of products and/or their production processes, manufacturing reliability, supply reliability, services supporting the effectiveness of the product, and other factors. Shortcomings in these areas may compromise health outcomes for patients. Thus, even if the initial acquisition cost of purchasing a drug may have been low, the secondary costs due to harms to patients or system inefficiencies may outweigh the intended savings.
We have therefore concluded that a tool should be developed to help countries with expanding healthcare coverages make listing, purchasing or tendering decisions — according to their healthcare priorities — in a more balanced manner, considering more than acquisition costs only.
What are some of the challenges associated with assessing the value of pharmaceutical products?
In my experience, it is much more complicated to look for value than for a low price. The definition of ‘value’ differs between specific categories of healthcare products. Defining value for each category requires agreement between the various stakeholders in the healthcare system and, as pointed out, a country’s economic and political systems too. Subsequently, instruments must be developed to help assess a products’ value; these must be able to be applied in a practicable and transparent manner, and be independent from the individual who conducts the value assessment.
…even if the initial acquisition cost of purchasing a drug may have been low, the secondary costs due to harms to patients or system inefficiencies may outweigh the intended savings.”
Agreement is also required on the data to be used for the value assessment — clinical trial data, real-world data, patient or provider experiences or other data sources. Hence, the development of value frameworks and processes to apply them in value assessment and decision making are long processes requiring input from many stakeholders. Such complex methodologies are often not feasible in the context of emerging markets. Therefore, countries with expanding healthcare coverage need much easier, more applicable instruments to help ensure balanced decision making.
What are some of the current barriers to the widespread adoption of criteria to ensure value-based decision making?
Value-based decision making has been discussed in mature healthcare systems for a long time, and many ideas and approaches for its implementation have been evaluated, tested or applied. All of these approaches are strongly influenced by the increasing consideration of stakeholder’s healthcare value in each country.
To change behaviors is a challenging endeavor.”
As our work focuses on the needs of emerging markets, we have started to explore some of the barriers to the implementation of processes for value-based decision making proposed by us. To change behaviors is a challenging endeavor. There are many advantages to using standardized decision tools, such as one we have developed , and other tools that allow for higher transparency, better standardization, better documentation, better foundation for quality control, and process improvement. However, these may not sufficiently meet the needs of all stakeholders in order to motivate their implementation as standard procedures.
Going forward, it will be increasingly important to develop instruments that are easy to use, easy to implement in the current decision-making environment, and reflect the specific, local needs and values of healthcare recipients [4, 5]. This will allow us to monitor improvements, familiarize users through pilot programs and trainings, and publish our learnings to increase awareness of the importance of these processes.
How may we work towards improved value-based decision making?
In our project, we are starting with the specific issues faced by countries with expanding healthcare coverage. If you can identify specific, root causes of tension or difficulty, and work to create ways to minimize or mitigate these, users and policy makers will be much more appreciative of your suggestion compared with if you were to start introducing the ‘principles of value-based decision making’ in general, which may seem somewhat academic, if not elitist, to some emerging market stakeholders.
Patients must be heard in order to understand what ‘therapeutic value’ means to them…”
Another fundamental aspect of improving value-based decision making concerns involving a range of local stakeholders in the process of creating the local solution, by adapting international developed principles [1, 6] to address the decision in a specific environment [3, 4, 5, 7].
What is the importance of patient engagement to value-based decision making and ensuring development plans meet patient needs?
Given that the essential goal of healthcare is to improve patients’ health, I feel that patients are important stakeholders in assessing whether something is benefiting them or not. Patients must be heard in order to understand what ‘therapeutic value’ means to them, which outcomes are important for them, what is feasible for them and what is occurring in the real-world compared with the clinical trial world. Without patients having needs, we wouldn’t be having this discussion. There are many initiatives being implemented to increase the involvement of patients in research, health technology assessment and their healthcare decisions. Recently, an entire book has been published on many aspects of patient involvement in HTA .
Likewise, the industry is adapting its development processes to better meet patient needs . Further, regulatory agencies — such as the US FDA  and the European Medicines Agency — have been exploring how best to involve patients into their work. Currently, most of this work focusses on mature healthcare systems, however, movement is starting in emerging markets, such as South Africa, Brazil, Taiwan, South Korea, and others.
How do you see the future of value-based decision making evolving?
If its’ value can be proven, value-based decision making will expand. If it doesn’t deliver the promised value, it may not be so valuable after all.
Interested in multiple criteria decision analysis as a way to make more value-based and rational decisions? Watch this video to learn more>>
 Brixner D, Nikos M, ZoltÃ¡n K, Shanlian H, Jie S and Kalman W. Considering multicriteria decision analysis (mcda) simple scoring as an evidence-based hta methodology for evaluating off-patent pharmaceuticals (OPPs) in emerging markets. ViHRI; 13c: 1—6; (2017);
 Kalo Z, Holtorf A-P, Alfonso-Cristancho R, Shen J, Ãgh T, Inotai A and Brixner D. Need for Multicriteria Evaluation of Generic Drug Policies. Value Health; 18(2): 346—351; (2015);
 Inotai A, Brixner D, Maniadakis N et al. Development of Multi-Criteria Analysis (MCDA) Framework for off-patent pharmaceuticals — an application on improving tender decision making in Indonesia. BMS Health Services Research; 18(1003); (2018);
 Assawamakin A, Holtorf A-K, Maniadakis N. Weighing price and performance for decisions for multisource pharmaceutical bidding in public hospitals in thailand. Evaluation of Health Programs. doi:10.5772/intechopen.83823 (Epub ahead of print) (2019);
 Abdullah AH, Holtorf A-P, AlHussaini M, Lemay J, Alowayesh M, Kalo Z. Stakeholder driven development of a multi-criteria decision analysis tool for purchasing off-patent pharmaceuticals in Kuwait. J Pharm Policy Pract. 12(9); (2019);
 Brixner D, Maniadakis N, Kalo Z, Kim K, Wijaya K. An evidence framework for off-patent pharmaceutical review (efor) for health technology assessment in emerging markets. ViHRI. 16c; 9–13; (2019);
 Inotai A, Brixner D, Maniadakis N et al. Development of multi-criteria decision analysis (MCDA) framework for off-patent pharmaceuticals — an application on improving tender decision making in Indonesia. BMC Health Serv. Res. 18; 1003; (2018);
 Patient Involvement in Health Technology Assessment. Facey KM, Hansen HP, Single ANV (Eds.) Adis, Singapore, (2017);
 Cook NS, Cave J, Holtorf A-P. Patient Preference Studies During Early Drug Development: Aligning Stakeholders to Ensure Development Plans Meet Patient Needs. Front. Med. 6(82); (2019).
 U. S. Food and Drug Administration, Department of Health and Human Services, Center for Drug Evaluation and Research. Patient-Focused Drug Development: Collecting Comprehensive and Representative Input. Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders.
[Accessed 19 November 2019]
Holtorf commented: “I have not received any funding for this interview and the content reflects my personal experience and opinion. The content of the workshop was based on research funded by Abbott Production Operation AG (Switzerland).”