President Donald Trump on Wednesday signed into law the “Right-to-Try” bill, but the news of the day came in a side comment when he said drug companies in the next two weeks will announce “massive drops in drug prices.” He did not offer any specifics on the voluntary price reductions.
The new law, meanwhile, will allow terminally ill patients with limited options to ask pharmaceutical companies if they can use experimental treatments outside clinical trials.
The law is a major win for the libertarian Goldwater Institute, which has been working to undermine the US Food and Drug Administration (FDA) for years with state “Right-to-Try” legislation that has spread to 40 states, though so far only one physician has tried to use such legislation.
The one example of patients using “Right-to-Try” at the state level is with an IND
granted to a Houston-based doctor, which was put on clinical hold, though the treatment used in the trial did win FDA approval in January
The Goldwater Institute said it did not know if the data from the Houston trial was used to support the application that won approval (the FDA press release says expanded access
data was used in the approval).
Critics of the “Right-to-Try” legislation have made clear that dubious pop-up companies and doctors trying to make a quick buck are likely to try to take advantage of this new law. Supporters, meanwhile, claim the law offers an alternative “pathway” to accessing experimental treatments, though it remains unclear how this pathway will work. Sen. Ron Johnson (R-WI) did not offer any additional details on how he thought the pathway will be implemented.
FDA Commissioner Scott Gottlieb said in a statement
: “At the FDA, we stand ready to implement this legislation in a way that achieves Congress’ intent to promote access and protect patients.”
CDER Director Janet Woodcock, meanwhile, said in a letter to staff, "We are reviewing the legislation and will work to implement it in a manner consistent with Congressional intent and with FDA's public health mission. We realize that you may receive questions about this process. We will be working expeditiously to develop further information on how to respond to such inquiries. However, we believe that sponsors are in the best position to provide information on the development status of their products (which is critical to determining whether a drug or biological product is eligible for use under Right to Try) and whether a sponsor intends to make an investigational product available under Right to Try."
"In the near term, if you receive inquiries about the legislation from patients or physicians about a specific product, please refer them to the sponsor of the investigational drug or biological product. If sponsors contact you regarding their obligations under this law, we suggest that you refer them to the statute," she said.
Ahead of the Senate Health, Education, Labor and Pensions' (HELP) hearing on drug prices on 12 June, Senator Bill Cassidy (R-LA) has circulated a white paper with recommendations for lowering the cost of healthcare.
The hearing is the fourth from the HELP committee on the topic and will focus on President Donald Trump's recently announced "blueprint
" to lower drug prices. Department of Health and Human Services (HHS) Secretary Alex Azar is scheduled to testify at the hearing.
In a speech announcing the blueprint, Trump said he would "take on one of the biggest obstacles to affordable medicines: the tangled web of special interests," including drugmakers, insurers, distributors and pharmacy benefit managers.
Previous HELP committee hearings on drug pricing in the last year have focused on aspects of US Food and Drug Administration (FDA) regulation that affect drug costs
, what other factors contribute to the costs patients pay
and a report from the National Academies of Sciences, Engineering and Medicine on making drugs more affordable
In a statement last week, Committee Chairman Lamar Alexander (R-TN) said: "The cost of prescription drugs is a complex problem and the Administration has laid out a comprehensive plan to help address that problem. I welcome the President's leadership to put patients first, and look forward to hearing more about the blueprint from Secretary Azar and learning how congress can help reduce the cost of prescription drugs."
Ranking Member Patty Murray (D-WA) has criticized
Trump's blueprint, saying that rather than taking substantive action to reduce drug prices, Trump and Congressional Republicans have "handed pharmaceutical companies billions in tax breaks that they used to pad their bottom line instead of helping lower drug costs for regular families."
In a white paper circulated on Tuesday, Cassidy, one of the co-sponsors of an effort last year to repeal the Affordable Care Act
(ACA), lays out areas where Congress could act to lower healthcare costs that he aims to include in future legislation.
Though the white paper proposes actions across the spectrum of healthcare, such as repealing the employer insurance mandate and replacing ACA funding with block grants, the rising cost of prescription drugs is singled out as a major driver of spending.
In the paper, Cassidy calls for greater incentives for "truly innovative" drugs, such as expedited reviews and longer periods of exclusivity for drugs that treat costly or life-threatening diseases. According to Cassidy, this would encourage drugmakers to target innovative products rather than so called "me-too" drugs.
However, other actions presented in the white paper are ones that have already been raised by members of Congress as potential steps to increase generic drug competition, such as allowing purchasers to import generic drugs approved in other countries and preventing drugmakers from using an FDA mandated risk evaluation and mitigation strategy (REMS) to keep generic manufacturers from accessing the samples they need to conduct bioequivalence testing.