Good health policy requires high quality evidence. Remember that with drug pricing.

Am J Manage care. 2022;28(9):In press

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Takeaways

  • Good policy requires good research.
  • The lack of reproducibility of research results raises questions about the quality of evidence.
  • We should conduct research that informs policy to the highest possible standards. Failure to do so means that our political debate is, at the very least, misinformed. At worst, blind acceptance of misleading information could lead to policies that will harm patients, consumers and employers.
  • The National Pharmaceutical Council recently released health expenditure guidelines to support rigorous, evidence-based evaluation and discussion of health expenditure estimates and policies.
  • Our goal should be to improve the efficiency of healthcare spending and maximize patient health. Using such principles to determine the true rigor of any study gives policymakers, journalists, and others better tools for evaluation in this effort.
  • Good research, placed in the proper context and fully understanding the inherent risk, uncertainty and potential trade-offs of any recommendation, is extremely valuable.

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Healthcare spending continues to rise, with total spending in 2020 exceeding $4.1 trillion, or 19.7% of US gross domestic product.1 Concerns are growing about the sustainability of continued increases, and details of the latest proposal to allow the federal government to “negotiate” drug prices have just been released.2 At this pivotal moment in the health care spending debate, what policymakers need most is an honest, realistic and evidence-based discussion. This is not happening, mainly because policy makers are inundated with poorly designed studies, with conclusions based on unrealistic assumptions not supported by evidence.

With the right tools, policymakers and other decision-makers can better evaluate these studies to help ensure policy conversations are properly evidence-based. Quality research allows them to identify lack of rigor and methodology; it can also foster greater confidence in research that is transparent in its methodology and realistic in its assumptions.

This places a particular burden on health policy researchers, who should design studies that support claims using data, assumptions and methodologies appropriate to the question being analyzed and transparent about the issues on which they are expected to provide relevant insight. .

Unfortunately, many studies in the public arena fall far short of this. Reproducibility, the ability of an independent researcher to reproduce the results of a study, is a standard for assessing the quality of evidence. Three studies assessing reproducibility found that this standard was only met between 40% and 62% of the time, raising questions about the quality of the evidence.3-5

The challenges are not limited to the quality of the results but also lie in the interpretation of these results. An oft-cited 2015 study, for example, notes that “many of the key ideas” that led to transformative medicines first began with publicly funded basic research.6 Proponents of government price controls said the findings of this study show that as long as public investment is substantial, the pace of innovation will remain unchanged.seven

However, policymakers should be aware that the study omitted the fact that the pharmaceutical industry can invest more than a billion dollars per therapy, compared to only tens of millions of dollars in government investments. If private investment declines, what will fill the gap? The study is silent on this issue.

The point here is that we should conduct studies that inform policy to the highest possible standards. Failure to do so means that our political debate is, at the very least, misinformed. At worst, blind acceptance of misleading information could lead to policies that will harm patients, consumers and employers.

As part of our commitment to rigorous, quality research, the National Pharmaceutical Council recently released health care spending guidelines to help foster rigorous, evidence-based evaluation and discussion of health care estimates and policies. health expenditure.8

These principles can help policy makers assess methodological rigor, limitations and alignment with patient-centred care and provide a framework to guide the evaluation of policy proposals through a better process for identifying trade-offs, uncertainty and the risk of unintended consequences for these proposals.

Our goal should be to improve the efficiency of healthcare spending and maximize patient health. Using such principles to determine the true rigor of any study gives policymakers, journalists, and others better tools for evaluation in this effort.

Good research, placed in the proper context and fully understanding the inherent risk, uncertainty and potential trade-offs of any recommendation, is extremely valuable. Everyone involved in the drug pricing debate, regardless of their perspective, should welcome a good research standard.

This will allow stakeholders across the healthcare system to have an honest discussion about how best to address rising healthcare costs and foster patient-centred care.

It’s a conversation worth having.

Affiliation of authors: National Pharmaceutical Council (MC, JMO), Washington, DC.

Source of funding: None.

Author Disclosures: Mr. Ciarametaro is a former employee of the National Pharmaceutical Council, a health policy research organization supported by the biopharmaceutical industry. Dr. O’Brien is an employee of the National Pharmaceutical Council.

Author Information: Concept and design (MC, JMO); data acquisition (MC); data analysis and interpretation (MC, JMO); manuscript writing (MC, JMO); critical revision of the manuscript for important intellectual content (MC, JMO); obtain funding (MC, JMO); administrative, technical or logistical support (MC, JMO); and supervision (MC, JMO).

Address correspondence to: John Michael O’Brien, PharmD, MPH, National Pharmaceutical Council, 1717 Pennsylvania Ave NW, Ste 800, Washington, DC 20006. Email: [email protected]

REFERENCES

1. Kurani N, Ortaliza J, Wager E, Fox L, Amin K. How has US healthcare spending changed over time? Peterson-KFF Health System Tracking. February 25, 2022. Accessed July 8, 2022. https://www.healthsystemtracker.org/chart-collection/us-spending-healthcare-changed-time/#Total%20national%20health%20expenditures,%20US%20$%20Billions ,%201970-2020

2. Subtitle I – Reform of drug pricing. Senate Finance Committee. Accessed August 1, 2022. https://www.finance.senate.gov/imo/media/doc/070622%20Prescription%20Drug%20Pricing%20Reform%20Leg%20Text.pdf

3. Open scientific collaboration. Psychology. Estimation of the reproducibility of psychological science. Science. 2015;349(6251):aac4716. doi:10.1126/science.aac4716

4. Klein RA, Vianello M, Hasselman F, et al. Many Labs 2: Investigation of variations in replicability between samples and parameters. Adv Pract Psychol Sci Methods. 2018;1(4):443-490. doi:10.1177/2515245918810225

5. Camerer CF, Dreber A, Holzmeister F, et al. Assess the replicability of social science experiments in Nature and Science between 2010 and 2015. Nat Hum Behav. 2018;2(9):637-644. doi:10.1038/s41562-018-0399-z

6. Kesselheim AS, Tan YT, Avorn J. The roles of academia, rare diseases, and repurposing in the development of the most transformative drugs. Show Health (Millwood). 2015;34(2):286-293. doi:10.1377/hlthaff.2014.1038

7. Kesselheim AS, Avorn J. Letting the government negotiate drug prices will not hurt innovation. Washington Post. September 22, 2021. Accessed June 13, 2022. https://www.washingtonpost.com/outlook/2021/09/22/drug-pricing-negotiation-biden-bill/

8. Guiding principles for health expenditure. National Pharmaceutical Council. May 12, 2022. Accessed May 12, 2022. https://www.npcnow.org/hcsgp

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