By Jeannie Baumann and Alex Ruoff | July 16, 2019 05:41AM ET | Bloomberg Law<\/p>\n
Lawmakers and conservative advocates who fought to make it easier for Americans to use experimental medicines are floating a new twist: permitting drugmakers to sell unapproved drugs at a profit.<\/p>\n
The \u201cfree to choose\u201d<\/a> idea is in early stages of conversation, largely among conservatives. It would shortcut clinical trials, which are the costliest of the decade-long drug development process and can run into the billions.<\/p>\n The free-to-choose push seeks to build upon the Right to Try Act<\/a>, which President Donald Trump signed into law last year. It allows patients with life-threatening diseases to petition a drug company for an experimental product if they can\u2019t get into a clinical trial, and they can do so without having to go through the FDA.<\/p>\n \u201cWe are supportive of Right to Try,\u201d Christina Herrin, director of Free to Choose Medicine for the Heartland Institute, said in an interview. \u201cBut we think it\u2019s missing a market incentive.\u201d<\/p>\n The Heartland Institute, the Chicago-based think tank behind conservative messaging that downplays the impacts of climate change, is also a major force behind the free-to-choose proposal. The group says the change could clear up a bureaucratic bottleneck that stifles access to innovative treatments and limits patients\u2019 health-care options. Opponents say it would expose patients to potentially dangerous medicines and that options for gaining access to experimental drugs already exist.<\/p>\n The free-to-choose idea would set up a parallel system to the FDA\u2019s current drug approval process. Pharmaceutical companies could market drugs that have passed smaller clinical trials that test for toxicity and efficacy but not the larger studies that determine new drug approvals. It would also expand the eligible conditions to include debilitating diseases such as ALS and Alzheimer\u2019s.<\/p>\n \u201cGetting the right treatment to the right patient and at the right time is the goal,\u201d said Naomi Lopez Bauman, director of health-care policy at the Goldwater Institute in Phoenix. \u201cThis is an important issue and the Free to Choose Medicine proposal deserves serious consideration.\u201d<\/p>\n It\u2019s also gotten the attention of members of the ultra-conservative Freedom Caucus in the U.S. House. A dozen Republicans, including several Freedom Caucus members, last month asked<\/a> acting FDA Commissioner Ned Sharpless to expand the Right to Try track to diseases beyond HIV\/AIDS.<\/p>\n Rep. Thomas Massie (R-Ky.), one of the letter\u2019s signatories, said FDA officials have the discretion to go further when it comes to experimental medicines but aren\u2019t using it. He wants to know why.<\/p>\n \u201cIn the instances where they\u2019ve done it, it\u2019s been a very positive thing, so let\u2019s do more of it,\u201d Massie told Bloomberg Law. \u201cWe\u2019re asking them to use the discretion they have. And if they don\u2019t, we can pass legislation\u2014I\u2019m all for that.\u201d<\/p>\n A spokesperson for the FDA said the agency will review the lawmakers\u2019 letter and respond directly to them. It may be a tough sell because FDA officials have told Congress in other contexts that the agency isn\u2019t holding up the drug development process. It has incorporated faster reviews and broken records for the number of drugs approved.<\/p>\n Under current policies, manufacturers can recoup costs they\u2019ve incurred for providing experimental medicines, but they aren\u2019t allowed to profit from the transactions.<\/p>\n Free to choose would allow marketing of early-stage drugs. It also would create a database to track genetic data, relevant biomarkers, and clinical treatment results from the use of free-to-choose drugs. The data would allow drug sponsors, researchers, physicians, and patients to judge what treatment options might be best for them, the Heartland Institute says.<\/p>\n \u201cThe current system is overly paternalistic and it violates patient\u2019s rights of self-medication,\u201d Jessica Flanigan, a medical ethicist at the University of Richmond, told Bloomberg Law. Free to choose \u201cis a step in the right direction towards respecting patient\u2019s rights of self medication.\u201d<\/p>\n The FDA already has a program called \u201cparallel track<\/a>,\u201d which it created at the height of the AIDS epidemic to allow access to experimental HIV\/AIDS drugs. Since that time, the FDA has established other programs<\/a> to allow the use of an unapproved medicine, including expanded access and emergency uses.<\/p>\n Gregg Gonsalves, a longtime HIV\/AIDS activist who cofounded a nonprofit<\/a> that pushed for earlier and broader access to experimental therapies in the 1990s, raised alarms about the freedom-to-choose proposals.<\/p>\n Gonsalves, a Yale University epidemiologist, cautioned against weakening the FDA\u2019s oversight of investigational drugs. He said such a move could have tragic consequences. \u201cIn the case of pharmaceutical drugs, this also means dead patients too,\u201d he said.<\/p>\n Tying a marketing go-ahead to Phase III clinical evidence is the agency\u2019s \u201cmost important tool in getting industry to do the studies we need to know if drugs work,\u201d he said.<\/p>\n Investigational medicines typically must pass three clinical trial phases<\/a> to demonstrate they are safe and effective enough to market.<\/p>\n Phase I uses healthy volunteers to determine dosage and toxicity. Phase II studies are typically smaller ones that assess some level of effectiveness. Phase III studies are bigger trials to gather enough information about safety and effectiveness to warrant an FDA approval that also gives drugmakers the authority to market the medicine. Phase III trials also provide data that is used for labels physicians rely on.<\/p>\n Without Phase III testing, doctors and patients are missing important, high-quality data that they need to make an informed decision about treatment, University of Pennsylvania bioethicist Holly Fernandez Lynch told Bloomberg Law.<\/p>\n Nearly 70 percent<\/a> of medicines in development fail in Phase III, which means patients could end up taking unnecessary risks, especially for conditions that aren\u2019t life threatening and for which other treatment options exist, she said.<\/p>\n Flanigan acknowledged that objections to free to choose may arise \u201con the grounds that it would allow people to access drugs that have not fully completed efficacy testing. But people can already access drugs that have not been tested for efficacy when they use drugs off-label.\u201d<\/p>\n Allowing biotech companies to charge for experimental drugs under free to choose would allow smaller drug companies to develop new drugs, the Heartland Institute said.<\/p>\n To contact the reporters on this story: Jeannie Baumann in Washington at jbaumann@bloomberglaw.com<\/a>; Alex Ruoff in Washington at aruoff@bgov.com<\/a><\/p>\n To contact the editors responsible for this story: Fawn Johnson at fjohnson@bloomberglaw.com<\/a>; Brent Bierman at bbierman@bloomberglaw.com<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" Access to Trial Meds Needs Market Incentive, Conservatives Say By Jeannie Baumann and Alex Ruoff | July 16, 2019 05:41AM ET | Bloomberg Law Lawmakers and conservative advocates who fought to make it easier for Americans to use experimental medicines are floating a new twist: permitting drugmakers to sell unapproved drugs at a profit. The…<\/p>\n","protected":false},"author":1,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"..\/public\/views\/revslider-page-template.php","meta":{"footnotes":""},"yoast_head":"\nMaking a Profit<\/h3>\n
Raising Alarms<\/h3>\n
Making Informed Decisions<\/h3>\n