For decades, partnerships between government-funded academic institutions and private companies in the United States have led to the discovery and development of innovative medicines that improve, extend and save lives. My company’s treatment for advanced prostate cancer, Xtandi (enzalutamide), resulting from a public-private collaboration, is an example of how this system works to benefit patients.
Yet despite the obvious health benefits and wide availability of Xtandi, some people and organizations want to use it as a test case to disrupt the ecosystem of technology transfer and medical innovation that is the pathway to cures for tomorrow.
Xtandi was the first – and remains the only – new hormone therapy approved by the Food and Drug Administration (FDA) to treat three distinct types of advanced prostate cancer. It helps men with one of these three types of prostate cancer live longer. It has been prescribed to over 209,000 patients in the United States and over 678,000 worldwide.
Xtandi is widely available in the US In 2021, the majority of Medicare beneficiaries paid $20 or less per month for Xtandi. Retired military members and their families enrolled in TRICARE can access Xtandi for co-pays ranging from $0 to $14 per month, with active duty TRICARE members having no co-pays.
A small group of critics have sent a petition to the Department of Health and Human Services, urging the government to seize the intellectual property rights associated with Xtandi and license them to another party. Doing this, they say, would allow another party to manufacture and sell the drug at a lower price.
Such a decision would set a troubling precedent and jeopardize important future medical innovations, discoveries and developments.
Background to the Bayh-Dole law and entrance fees
In 1980, Congress enacted legislation, sponsored by the late Senators Birch Bayh (D-Ind.) and Bob Dole (R-Kan.), that changed the paradigm for ownership of inventions. The Bayh-Dole Act allowed inventors to retain ownership of inventions conceived or generated with the support of federal funding in exchange for granting the government certain rights in the invention.
This change provided a strong incentive for private industry to invest in the development of such inventions – including drugs – and to bring them to market for the benefit of American consumers and patients.
By any measure, the act was an astonishing success. Before it became law, less than 5% of patents resulting from public-private collaborations were transformed into commercial products benefiting the public. Since the law was passed, these rates have increased dramatically. In the 22 years since the law was passed, American universities have multiplied the number of patents tenfold. More than 200 new therapies and vaccines have been introduced to the market thanks to Bayh-Dole.
The Bayh-Dole Act provided for cases in which an invention generated in part by government funding was not commercialized by the inventor and patent holder. In these cases, the law gave the government the ability – under specifically defined circumstances – to “brand” and redirect the license fees to an entity that would bring the product to market. However, as Bayh and Dole made clear, the law that bears their name “did not intend that the government fix the prices of the resulting products. The law makes no reference to a reasonable price which should be dictated by the government. This omission was intentional[.]”
Over the years, however, the government has been repeatedly pressed to use walking rights as a means of reducing drug prices – including petitions filed against Xtandi.
In accordance with the Bayh-Dole Act’s four circumstances in which market rights may be exercised, the National Institutes of Health (NIH) has consistently and unequivocally denied any request for price-based walking, whether for Xtandi or any other drug, explicitly recognize that the parade is not the right tool to fight drug prices.
In 2016, for example, the NIH dismissed an Xtandi-specific petition that argued that practical application of the drug had not been realized because its price did not make it available to the public on reasonable terms. By denying the petition, the NIH made it clear that the award is not a legal basis for the march-in. Instead, consistent with the wording of the statute and the expressed intent of Congress, NIH Director Francis S. Collins explained that an invention in question is available on reasonable terms when it is on market, widely available to the public and used by patients.
The NIH found that “Xtandi is widely available as a prescription drug,” thus Xtandi met each of these requirements in 2016 and still meets them in 2022.
The NIH has repeatedly determined that the Bayh-Dole Act does not give the agency the authority to use “branding” rights to affect drug prices.
Discovery and development of Xtandi
Xtandi’s patents cite financial grants from the NIH and the Department of Defense that cover a number of research goals and laboratory projects, including some work that led to the initial discovery of the molecule that would become Xtandi. .
An analysis of these grants that Astellas obtained in 2016 indicates that the federal government provided initial funding of less than $500,000 to the University of California, Los Angeles, which directly contributed to the initial discovery of Xtandi. This represents a small fraction of what Astellas and its partners have invested to date to take this molecule from initial discovery to FDA approval of Xtandi and beyond.
Astellas and its partners have conducted the extensive preclinical studies and clinical trials necessary to demonstrate the safety and efficacy of Xtandi for FDA approval. To date, these private entities have invested more than $2.2 billion to bring the drug to men with advanced prostate cancer. Astellas alone has invested more than $1.4 billion, 98% of which is attributable to the company’s investments in preclinical and clinical testing; chemistry, manufacturing and controls; medical affairs; Quality control; and other expenses.
Xtandi’s development journey is not uncommon. Academic, public and private scientists contribute to the vast body of fundamental scientific discovery, and this knowledge is shared and developed through peer-reviewed publications, scientific meetings and intellectual property licensing. The unique role of the biopharmaceutical industry in this ecosystem is to employ its scientific and industrial expertise to undertake the considerable work – and necessary risk – to build on basic science and advance it further into safe and effective treatments. which can be made available to patients for a defined period of time and then become generic products.
If Astellas had known from the start of Xtandi’s development that its investment would be seized by the government, the company would not have invested the way it did. Other companies currently on the verge of entering public-private partnerships or acquiring development assets that have received public funding may well question their investments if a precedent is set.
Xtandi is accessible to patients
Proposals to charge entry fees to Xtandi have been based on misleading and misleading claims about the cost of Xtandi to patients.
This drug is widely available and its price matches that of other oral therapies for advanced prostate cancer. While there are examples of patients paying high prices as a result of decisions made by insurance companies or drug benefit managers, in 2021, 71% of US patients paid less than $100 in out-of-pocket costs for their Xtandi prescriptions, regardless of the type of insurance. . While march-in proponents have pointed to the wholesale acquisition cost of Xtandi – which Astellas makes transparently available at XtandiPricing.com – the oft-quoted list price does not reflect what is ultimately paid or reimbursed for the product, and it does not reflect the amount most patients pay for it.
The list price does not take into account lower prices paid by the government, rebates manufacturers negotiate with insurance companies, or patient assistance programs, such as the Astellas Patient Assistance Program.
In the United States, society can take many steps to improve the affordability of medicines. pharmacy benefit managers to pass discounts and rebates to patients. Such policies would help make medicines more affordable for patients, without jeopardizing the engine of collaborative R&D in this country.
Fueling New Innovations
The development of vaccines and therapies against Covid-19 has clearly shown what is possible when public-private partnerships work to meet emerging healthcare needs. The biopharmaceutical industry has developed several highly effective vaccines and treatments in record time. The country needs more of these partnerships, not less.
Likewise, Astellas applauds efforts to continue these collaborations, including through the renewed Cancer Moonshot initiative, in which President Biden called for collaboration among the private sector, foundations, academic institutions and service providers. health care to bring new innovations to defeat cancer.
Today’s collaborative ecosystem, largely enabled by the Bayh-Dole Act, has served Americans throughout the pandemic and will be instrumental in meeting future challenges — and continued progress against many other diseases we are facing.
To prevail, Astellas and healthcare system stakeholders must understand the importance of collaboration and the proven investment framework established by the Bayh-Dole Act that encourages complex, high-risk medical innovation. And together, the biopharmaceutical industry, policymakers, patients and all stakeholders must address drug affordability in a responsible and measured way, not through short-sighted proposals that would discourage future investment and public innovation. -private.
Mark Reisenauer is the president of Astellas Pharma US, Inc.