Health economics, as applied in the pharmaceutical industry (also commonly referred to as pharmacoeconomics), is concerned with providing evidence to demonstrate the efficiency, effectiveness and value of pharmaceutical products.
The health economic deliverables which primarily support market access are economic models (cost-effectiveness and budget impact models) and value dossiers. Cost-effectiveness and budget impact models play a key role in the assessments by many national health technology assessment (HTA) bodies . Budget impact models can also facilitate discussions with local payers and health care decision-makers. The United States Food and Drug Administration modernization Act of 1997, for example, allows pharmaceutical manufacturers to provide economic information on their drugs to formulary committees, managed care organisations and other large scale payers for health care products . Value dossiers provide information on the relevant disease state, including the unmet need and current treatment options and patterns; clinical effectiveness and safety of the drug and its comparators as well as economic analyses to demonstrate value. The value dossier forms the foundation of the pharmaceutical manufacturers' submission packages to national HTA bodies, whose job it is to assess the evidence and make recommendations as to whether a drug should be reimbursed or not. Other types of economic deliverables include real world studies, for example, of treatments patterns and resource use, as well as treatment preference studies.
A range of analytic approaches are used including systematic literature reviews (SLRs), meta-analysis, economic modelling techniques, statistical analyses applied to healthcare datasets and stated choice models. The health economist has a key role in identifying evidence gaps and producing evidence to fill the gaps. They also ensure health care resource use and health related quality of life data are collected within the pivotal clinical trials which can be leveraged to demonstrate the drug’s value. The internal expectation is that the new evidence will be published. SLRs required for HTA submissions, for example, are based on data in the public domain. In addition, most countries require promotional pieces to be based on published data rather than data on file.
Health economists working in pharma work to tight internal deadlines driven by product development and external deadlines driven by HTA bodies. The subject is complex but the audience is broad and includes non health economists, such as clinicians, payers, and lay people, who need to understand the information to make informed decisions.
Against this challenging background we offer here 10 suggestions to help the health economist be successful in their role in pharma. The suggestions are based mainly on the author’s experience of nearly two decades working in the industry.
- A Clear Health Economics Strategy
A clear strategy as to how the efficiency, effectiveness and value of the pharmaceutical products are to be demonstrated requires a plan of the health economic deliverables. The plan will be driven by HTA requirements and the market access team’s value strategy. A clear definition of the patient population of interest, the comparator and outcomes ensures clarity and consistency across the health economic deliverables. The health economic strategy needs to be aligned with that of the broader product team, requiring discussions with the market access team towards the end of Phase II and early part of Phase III of the drug development process. The plan can evolve as new insights become available. Linking the completed deliverables back to the strategy will ensure the value package is aligned internally and reduce the opportunity for the relevance of the deliverables to be challenged.
- Identify the Evidence Gaps Early
Discussions regarding the data gaps for the evidence package ideally should start towards the end of Phase II/early part of Phase III, as generating new evidence takes time. Input from the wider product team, particularly the market access team and medical affairs, is invaluable. SLRs can also help elucidate the data gaps as they provide a comprehensive summary of the current literature relevant to the question. Discussions with external thought leaders can also provide useful insight and validation of the evidence gaps. Early phase economic models can facilitate insights into the potential cost-effectiveness or budget impact implications of the drug whilst still in the earlier phases of drug development. Models will need to be populated with available evidence at the time and reasonable assumptions made where data are not available. Such an exercise can be useful to identify data gaps as well as informing the pricing strategy and expectations regarding the challenges for market access.
With limited financial budgets, skilled personnel and time, the health economic deliverables will likely need to be prioritised. Decisions need to be made as to what are the ‘must haves’ and what are the ‘nice to haves’. The ‘must haves’ are those required to meet HTA body requirements for access and reimbursement. Depending on the budget available, a decision then needs to be made as to which of the ‘nice to haves’ are most impactful and can be implemented within the budget. It is imperative to keep cross functional partners informed of expected deliverables and prioritisation in order to mitigate challenges in the future.
4. Provide Solutions
Once the evidence gaps have been identified and prioritised, solutions to fill the evidence gap need to be sought. Before any research can be conducted it is important to identify the research question(s) to be addressed, as this will drive the study protocol and type of analysis. Selecting an appropriate data source(s) and conducting the analysis can be done internally, or by an external vendor, but having the right skills and experience is key to success.
As previously highlighted, many HTA bodies require manufacturers to submit cost-effectiveness models as part of their submission package. One solution to decreasing the risk of the model and interpretation of results being challenged by the HTA body, is to have the model validated by an independent assessor prior to submission.
5. Analytical Skills
As highlighted above, health economics utilises a number of analytical approaches. Even if the health economist does not conduct the actual analyses, or have high expertise in one specific type of analysis, they need to be able to understand how the analyses are conducted and be able to interpret the evidence. It is therefore important that the appropriate training has been undertaken. This may be in the form of specialist courses, formal degree qualifications in health economics, supplemented with on the job training and self teaching.
6. Keeping up to date with developments in the external environment
Keeping up to date with the latest developments in the external environment: health policy, disease area, analytical techniques, requirements by HTA bodies will help ensure the success of an HTA submission and the evidence provided. A good way to do this is to attend relevant conferences, such as the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), and relevant disease specific conferences such as American Society for Clinical Oncology (ASCO) , explore their websites and use your internal networks with medical and marketing teams.
7. Meeting deadlines
Health economists working within pharma have to meet the tight internal deadlines driven by product development and external deadlines driven by HTA bodies.
Tips on meeting deadlines:
- Involve cross functional partners early on in the discussions to clarify the health economic strategy, identify evidence gaps, prioritise deliverables, help interpret data and understand its relevance. This ensures engagement and alignment reducing later questioning as to the need or purpose of deliverables.
- Have a clear understanding of the drug development timelines as this will inform when the clinical data inputs will be available for health economic deliverables such as the cost-effectiveness models and value dossier.
- Plan ahead and allow enough time to produce deliverables.
- When writing study protocols, reports, dossiers and developing the economic models, plan ahead to ensure the reviewers have advance warning of their role and the timelines for review. This helps the reviewers plan their workload.
- Using a good medical writer avoids rewrites of grammar and content by or requested by reviewers.
8. Cross functional partnerships
The success of health economic deliverables depends on cross functional partnerships with statisticians, trialist, medical, marketing and market access teams. As alluded to above, collaboration with cross functional partners will help clarify the health economic strategy, identify evidence gaps, prioritise deliverables, and inform the interpretation of data and its relevance. However, the health economist may need to be prepared to help their cross functional partners understand the role of health economics and what kind of studies can demonstrate the value of a product, and the expectations of HTA bodies. Good cross functional partnerships will ease the implementation of resource use and health-related quality of life data collection in the clinical trials and the additional statistical analyses of the trial data required for HTA purposes, such as subgroup analyses and utility value estimation.
9. Good Communication of the evidence
Health economics subject matter is complex but the audience, who need to understand the information to make informed decisions, is broad. How the data are communicated is critical to their understanding and usefulness of the data, some requiring more detail than others. For example, an internal business partner may only require the objective of the study, the topline results and key takeaways, whereas an HTA body will require the detail.
10. Choosing the right medical writing team
Having a good medical writing team is critical. A good medical writer can relieve some of the timeline anxieties associated with health economic deliverables by avoiding rewrites and additional reviews. Clearly written text can ease understanding by the decision maker. A good medical writer is one who has knowledge about the topic area and the ability to make decisions and justify changes when needed. In addition, a good project manager, who may or may not also be the medical writer, can make a difference in terms of keeping the deliverable on track and meeting expectations.
We very much hope these 10 suggestions will assist you, the health economist working or seeking to work in the pharmaceutical industry, to be successful in your roles. We have provided a quick reference checklist to aid you navigate the challenges of producing ever increasingly important, but also complex, health economic deliverables to help ensure reimbursement and market access of pharmaceuticals.
- Is there a well-defined health economics strategy?
What are the health economic deliverables and timelines? Are they driven by HTA requirements and the value strategy? What is the patient population of interest? What is the treatment comparator? What are the relevant outcomes? Are these aligned with the value strategy and wider product team’s thinking? Have the completed deliverables been linked back to the health economics strategy?
- Have the evidence gaps been identified?
Have the evidence gaps been defined early to allow time to conduct the studies? Has input been sought from the wider product team, particularly the market access and medical affairs teams? Have discussions with external thought leaders considered? Has an early cost-effectiveness modelling been considered?
- What health economic deliverables have been prioritised?
What are the ‘must haves’ and the ‘nice to haves’? Is there any budget to fund any of the nice to haves? If so, which will be the most impactful?
- Have solutions been identified to fill the evidence gaps?
What research questions need to be addressed? What data sources are available? Who will conduct the analysis? Do they have the right skill set and experience? Have the economic models been validated?
- Do you have the appropriate analytical skills?
Do you have enough understanding of how the analyses are conducted and be able to interpret the evidence? Have you considered some additional training?
- Have you kept up to date with the external environment?
Have you considered attending relevant conferences? Have you explored their websites? Have you developed an internal network with medical and marketing teams?
- Can the deadlines be met?
Are cross functional partners engaged, aligned and aware of the economic deliverables? Do you have a clear understanding of the drug development timelines? Have internal reviewers been given warning of their role and the timelines for review?
- Do the cross functional partners fully understand the need and value of health economic deliverables?
Consider informing cross functional partners what kind of studies can demonstrate the value of a product, and the expectations of HTA bodies?
- How will the data be communicated?
Who is the audience? Do they require a deep understanding or topline results and key takeaways?
- Do you have a good medical writing team?
Is the medical writer knowledgeable about the topic area? Do they have the ability to make decisions and justify changes when needed? Is there someone in the writing team whose job it is to keep the project on track and meet expectations?
- Frontier A-M, Visintin E, Kanavos P. Similarities and differences in health technology assessment systems and implications for coverage decisions: evidence from 32 countries. PharmacoEconomics Open 6, 315–328 (2022). https://doi.org/10.1007/s41669-021-00311-5
- Commissioner of the FDA backgrounder on FDAMA. U.S. Food and Drug Administration. FDA; 1997. Available from: https://www.fda.gov/regulatory-information/food-and-drug-administration-modernization-act-fdama-1997/fda-backgrounder-fdama
- Asco Hub – American Society of Clinical Oncology. American Society of Clinical Oncology]. Available from: https://www.asco.org/