Individuals living with rare and chronic diseases, or any other serious underlying health conditions, have an acute understanding of the critical importance of patient access to the latest treatments and therapies. For the autoimmune community, pharmaceutical discovery and innovation is a lifeline to health and well-being that, for many, is nothing short of life-changing.
The United States is an indisputable global hub of medical innovation that creates breakthrough, life-saving therapies for those with autoimmune and other diseases, along with so many other devastating conditions. In fact, U.S.-based firms fund 44 percent of the world’s medical research and development and invest 75 percent of global medical capital.
Now, with the onset of COVID-19, our nation’s leadership role in medical innovation is more important than ever. It offers hope for combating this devastating novel coronavirus as it has delivered treatments for so many people with serious illnesses and chronic conditions in the past.
Take, for example, remdesivir – a compound originally invented by Gilead Sciences for the treatment of Ebola.
While it ultimately proved ineffective for that disease, remdesivir has yielded a different but critically important clinical outcome: Patients suffering from COVID-19 that are treated with remdesivir are discharged from the hospital four days faster on average. This both ensures and expands needed hospital capacity for other at-risk patients, particularly those suffering from chronic underlying health conditions. And remdesivir is just one striking example of innovation that the United States is known for, and is a comfort to the millions around the globe who seek a viable therapeutic response to the pandemic – and who know it will be the U.S. innovation ecosystem that will ultimately deliver.
Yet, a recent experience concerning remdesivir is also an example of American innovation at-risk, as this and other breakthrough medicines are potentially undermined by an organization that few are familiar with, but that wields outsized influence: the Institute for Clinical and Economic Review.
ICER has appointed itself the sole arbiter of new-drug value, and its reports and value-assessment announcements inform policymakers, industry, insurers, patient groups and the public about what ICER believes pharmaceutical manufacturers should charge for their drugs.
ICER issues these decrees asserting “patient engagement” and “increased access” – yet, a brief glance behind the curtain reveals that neither is a meaningful part of ICER’s value assessments. The organization states that collaboration with industry partners and public comment from relevant stakeholders are necessary inputs to its analyses, but the reality is much different.
So, while remdesivir provides a strong example of U.S. medical innovation, it also serves as strong example of ICER’s flawed and disingenuous approach to valuing new treatments and therapies during this time of unprecedent global pandemic and generally.
In fact, in reviewing remdesivir, ICER departed from its own established value framework, which was updated this year with input from a broad range of stakeholders and requires rigorously analyzing clinical data and convening key stakeholders to inform these value calculations. ICER did not consult with critical stakeholders – neither Gilead, nor patient groups, nor others – prior to rendering a judgment on remdesivir. Nor did they invite public comments (a customary step before finalizing any recommendations).
ICER failed to rigorously analyze available data and failed to inform key stakeholders, including the drug’s manufacturer. Based on ICER’s own standards and value framework, the remdesivir valuation was both premature and incomplete. Its wholly underwhelming follow-up report did nothing to substantively address or meaningfully respond to the important points third-party stakeholders were making.
From 2014 to 2018, U.S. companies developed about twice as many new drugs as all of Europe combined. Consider that not a single ICER review of a breakthrough rare disease therapy that emerged during this time period resulted in a “high value” rating, and one begins to perceive an agenda-driven — rather than science-driven — institution that nevertheless claims to consider patients.
If this trend continues, and we find that ICER’s treatment of remdesivir is some kind of “new normal,” patients facing rare and chronic diseases and underlying health concerns should be worried about safe and effective access to needed medications, both now and in the future.
To be clear, the need for and utility of pharmaceutical value assessment is not in question. In fact, there are nearly two dozen different frameworks available that are data-driven, comprehensive, scrubbed of the use of quality-adjusted life years and meaningfully inclusive of patients and attentive to their priorities for health and life.
But if ICER’s remdesivir analysis – one that not only misses the mark objectively, but disregards ICER’s own established value framework – becomes the standard, it poses a grave threat to the U.S. innovation ecosystem that millions of patients have come to rely on.
The best way to move beyond this agenda-driven, myopic view of value is simple: calculate and consider value the right way. This means engaging with all constituencies in good faith, considering all relevant data, and presenting a holistic view of value that goes beyond simply the point of view of an insurer.
The consequences of devaluing new treatments and therapies are simply too dire to ignore the outsize value that life-saving innovation has during a time of global pandemic – and the contributions to health equity realized by ensuring disproportionately impacted communities have access to breakthrough therapies.
Randall Rutta is president and CEO of the American Autoimmune Related Diseases Association.
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