Known by many regulatory terms—named patient programs, expanded access, compassionate use and others—the practice of providing drugs to patients prior to regulatory approval has been relatively disorganized. Now, a company experienced in clinical trial sourcing and consulting, Luxembourg’s Multipharma Group, is setting up a business to organize this activity globally. Called Inceptua Medicines Access, the company has three business units: Pharma (to work directly with manufacturers); Healthcare (to provide pre-aproved medicines on demand); and Consulting (to work with manufacturers, patient advocacy groups and others).
“The sector has not adapted nor kept pace with the needs of the biopharmaceutical industry, healthcare professionals and their patients worldwide,” said Mark Corbett, EVP, in a statement. “Current demand combined with a complex and dynamic regulatory and political landscape means we need a new way of thinking about ethical and compliant access to pre-approval and unlicensed medicines. Inceptua Medicines Access offers unsurpassed understanding and a service customized to your needs.”
Obtaining drugs while still in the research stage is fraught with moral and legal issues. Besides the simple fact that the purpose of FDA and comparable national regulators is to keep unlicensed drugs off the market, clinical trials are set up with strict controls to prevent what amounts to human experimentation. Who gets access to such drugs (if their efficacy is established and patients are facing life-altering decisions) puts manufacturers in a difficult spot. Meanwhle, manufacturers themselves might find their clinical development of a drug compromised either by the difficulty in supplying a new drug, or the unregulated use of them even as safety and efficacy are being established.
There is language in the newly passed 21st Century Cures Act that requires US manufacturers to have “publicly accessible compassionate use policies” in place; other countries have various formalized processes for providing unlicensed medicines. A common occurrence is a drug approved for use in one country, but not yet granted approval in others. There are also various efforts to loosen up drug access under “right to try” concepts, although this has not taken root in the US. Even so, with the number of drugs under development today, and the multiplication of disease states being studied, the desire to gain access seems to be expanding.
Corbett tells Pharmaceutical Commerce that the company will work with manufacturers who want to set up compassionate use programs; to procure drugs on behalf of patients or providers; and to coordinate the delivery of the drugs across borders. It is not a dispensing pharmacy, however. Revenue will be generated either by fees from manufacturer clients, or adding a service charge on a direct purchase. Corbett notes that in most cases, a compassionate use situation first must get past clinical trial protocols (i.e., the program is applicable only if a patient has already been determined not to quality for a trial); and in carrying out this process, Inceptua will also be helping manufacturers recruit patients for trials.