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Casey McDonald is Senior Editor, Pharmaceutical Executive.
Some 20 states could have new right to try laws on the books by this time next year, though time will tell what kind of impact they'll have for patients or drug makers.
Expect a wave of states adopting “right to try laws” this year, according to David Farber partner in the FDA & Life Sciences Practice Group at King & Spalding. Some 20 states could have new laws on the books by this time next year, though time will tell what kind of impact they’ll have for patients or drug makers.
Expanded access has been a volatile topic in social and mainstream media and is consequently on the minds of state and national legislators. With such a fervor surrounding the issue, the industry should expect a wave of votes in state capitols.
Support for such laws has been near unanimous so far, Farber noted. Five states, Colorado, Louisiana, Michigan, Missouri and most recently Arizona have such laws on the books.
There is significant discussion whether these laws have substance or are simply “feel good” laws, said Farber.
Right to try laws allow terminally ill patients to try experimental drugs without the need for the FDA to stamp its approval, while offering doctors, hospitals and manufactures some protection from prosecution. Though where in place, the laws are not likely to offer companies much defense from attack by pundits and social media outlets crying foul, like in the well trodden case of #SaveJosh Hardy versus Chimerix and now-ejected CEO Kenneth Moch.
FDA offers expanded access for patients with serious or immediate life-threatening conditions, with no comparable or satisfactory alternative therapy, where potential benefit of the treatment outweighs potential risk and in situations that will not interfere with a compromise a drug’s clinical investigation, explained Preeya Noronha Pinto, also a partner in the FDA & Life Sciences Practice Group at King & Spalding.
To get to the point of requesting FDA-approved expanded access, a patient and/or their physician must successfully petition the drug manufacturer for compassionate use. The company must then pour over a host of reasons for and against allowing access to the drug.
FDA gives its stamp of approval, which it proudly claims to do, in 99% of cases. The high level of FDA approvals in expanded access cases should be expected, as any case that gets to this point, has been thoroughly vetted by the doctor, the hospital and the drug company, added Farber.
Surely the 1% that the FDA denies would be an interesting group to analyze, but a greater question is to better understand how many patients and doctors navigate before it comes in front of FDA. How many patients do companies deny? And what are the some issues companies need to understand before granting access to desperate patients, such as liabilities, costs, benefits to populations versus individuals and overall ethics?
Federal Level Changes
Congress is engaging on the issue of expanded access, said Farber. Introduced yesterday, Sections 1121-25 of “21st Century Cures” Legislation Discussion Draft looks directly at the expanded access issue, and would require an Expanded Access Plan submitted 30 days after an investigational drug becomes a “covered investigational drug”, explained Farber. The draft requires that a company provide a point of contact, description of process, process criteria, and anticipated timelines. The rules only apply to orphan drugs, breakthrough therapy or fast track approval drugs, or qualified infectious disease products, he added.
If a request is denied, written explanation must be provided, he noted.
The good news is that the rules would not be mandatory or restrictive making companies grant access, but would require firms to be thoughtful and to exchange information.
Also on the federal level, FDA is looking for greater transparency via a rule proposed in November. The rule would require the “responsible party to submit an “Expanded Access Record” with a unique ClinicalTrials.gov Identifier. HHS is taking comments on the rule; so if you have something to say, be sure to make your voice heard.
Another suggestion that has been suggested by Dr Arthur Caplan at New York University would be a tax on manufacturers and to create a fund. The idea does not have a lot of support, but it is important that the issue of costs, especially for smaller, more bootstrapped, biopharma firms, be open for discussion, noted Farber.
One key lesson for biotech and pharma companies on the issue of expanded access, is to be ready. Just looking at the social media outcries, it will happen again, said Farber. Whether its child cancer, Ebola, or another issue, expanded access will continue to challenge drug makers.
To wit, this Bloomberg article and this from Fierce Biotech points out that BIO is working with its member companies in efforts to train companies to be more savvy and responsive surrounding issues like patient access.
Farber suggested, if possible, have an ethicist on hand to look at expanded access policies. Have a plan in place and be ready to respond, he added.