The 21st Century Cures Act (Cures Act) is going to make it easier for patients to find out important information about accessing investigational new drugs through expanded access. Under this new law, any pharmaceutical company manufacturing an investigational new drug is now required to publicly post its policy on expanded access as well as its procedure for evaluating and responding to patient requests for such drugs.
The law imposes a deadline by which this requirement must be met, and, for many pharmaceutical companies, it is fast approaching. For those companies that currently have investigational new drugs either in their pipeline, or in phase I safety studies, the deadline is the date of the first initiation of a phase II or phase III clinical trial. However, for those companies that have already initiated a phase II or phase III study, the deadline is 60 calendar days after the Enactment Date of the Cures Act. As the Cures Act was signed into law on 13th of December, 2016, the deadline for those companies is the 11th of February, 2017.
To meet this deadline successfully, a pharmaceutical company must post the following information so that it is readily available to patients:
- A statement that clearly indicates the company’s intent TO or NOT TO provide its investigational new drug(s) to patients through expanded access
- Company contact information
- Guidelines for making a request
- Criteria that will be used by the manufacturer to evaluate and respond to a request
- The anticipated response time
- A link to the clinical trial record which contains information about expanded access for drugs
The information provided by a company may be generally applicable to all investigational drugs, if a company has more than one. The policy can also be revised at any time to reflect any changes in company procedures.
It is important to note that, while this transparency requirement will help patients better understand the treatment options that are available to them, it will NOT require a company to provide its drug to patients outside of clinical trials on an expanded access or compassionate use basis.
There are several reasons a pharmaceutical company might decide not to provide its drug. Some companies do not have the time, money, or resources to support an Expanded Access Program; other companies make a strategic decision to refuse expanded access in order to focus on the clinical trial and its efforts to bring the drug to market.