Many proponents of Right to Try legislation believe that the US Food and Drug Administration (FDA) is too slow and inefficient when it comes to reviewing requests for access to important investigational new drugs. They, therefore, consider FDA oversight to be an unnecessary roadblock that should be removed from the equation.
However, what these Right to Try supporters fail to recognize is that, simply removing the FDA does not improve access, as it is drug manufacturers, not the FDA, that govern whether a patient receives an investigational drug.
As such, the NYU School of Medicine Working Group on Compassionate Use and Pre-Approval Access (CUPA) is encouraging legislators to focus less on phasing out the FDA and more on finding ways to make the current Expanded Access framework more appealing to drug companies. CUPA has three main recommendations for how to go about doing this.
The first recommendation is to address the concerns raised by industry regarding the occurrence of serious adverse events. One of the main reasons companies decide not to provide their drugs through Expanded Access is that they worry that patients, who fall outside the criteria of the ideal patient for the clinical trial, and who use their drugs outside the controlled clinical-trial setting, are more likely to have a negative response to the medication. Because under the current Expanded Access guidelines, any serious adverse events must be reported to the FDA, companies are concerned that this will deter their chances of getting their drugs approved and will unsettle potential investors.
However, the FDA has produced reports, based on recently conducted audits, that show that companies very rarely face obstacles in the drug approval process due to the occurrence of serious adverse events. In light of this, CUPA is calling for legislators to require the FDA to distribute these findings and make them easily accessible to the pharmaceutical industry. This can be done by expanding the FDA’s current expanded access guidance document to include clear and specific information on how serious adverse events, occurring in the context of expanded access, will be reviewed and what the consequences of these events will be.
The second recommendation made by CUPA is to promote the use of Real World Data (RWD). The International Society for Pharmacoeconomics and Outcome Research has defined RWD as “everything that goes beyond what is normally collected in the phase III clinical trials program in terms of efficacy.” This kind of data, gathered on patient experiences outside the clinical trial, can be extremely useful, as it allows the FDA to glean insights into how an investigational drug works in the broader patient population.
Legislators are definitely beginning to recognize the importance of Real World Data, as is evidenced by Section 3022. of the 21st Century’s Cures Act, which states that the FDA must issue guidance to increase the collection, use, and reliance of real-world evidence to help support regulatory decision-making. However, continued efforts must now be made to ensure that this guidance is created promptly and is made accessible to pharmaceutical companies. Only when companies are fully aware of how this data can be collected, and how the FDA will review it, will the true value of this data source become apparent to industry.
The third recommendation is simply to support smaller pharmaceutical companies with limited resources. Oftentimes, a company is open to providing expanded access use of its drug, but it does not have the time, money, or personnel to support such a program alongside its clinical trial. Extra drug will have to be produced, a distribution channel will have to be set up and managed, and employees will have to be hired and trained to handle requests, assist requesting physicians, dispense the drug(s), field questions, and handle paperwork and reporting. Going down the expanded access path can, therefore, seem daunting and almost impossible for small companies. As such, CUPA is encouraging legislators and industry to work together to come up with new ways to make these activities feasible for these companies. Only through open and ongoing conversation can new and innovative ideas and solutions can be brought to the table.