The prominent US pharmaceutical trade group PhRMA has released an updated set of principles it says reflects the US pharmaceutical industry's "commitment" to provide patients with life-threatening diseases access to investigational therapies on a compassionate-use basis.
The policy is meant to accommodate patients who are otherwise not eligible to participate in a clinical trial for a new drug, either because they are too sick to participate, have a disqualifying characteristic or are afflicted by an entirely different disease.
While FDA has its own regulatory pathway for accommodating these types of requests, known as its expanded access program (also known as its compassionate use program), it largely governs the requirements for companies deciding to provide access to their drugs.
For more on FDA's expanded access/compassionate use program, please read our explainer.
Whether companies should or will provide access to their drugs is an entirely different matter.
Under federal regulations, no company is required to grant access to its investigational products. In fact, many are hesitant to do so. Expanded access to a drug can be costly to the company, dangerous to patients, delay ultimate approval for a drug, lead to unexpected adverse events (and clinical holds) and take valuable staff time and resources to administer.
Compassionate Use Policies
But thanks to increasing patient advocacy, both PhRMA and the Biotechnology Industry Organization, a biotechnology trade group, have decided to establish standards for when their member companies should consider granting access to their investigational products.
BIO's statement, released in March 2014, said its member companies "have an ethical obligation to develop safe and effective drugs and biologics and make them available to patients as quickly as possible."
PhRMA, though, had gone beyond just a statement of principles to develop a defined set of commitments its member companies must comply with.
Under a new document, "Principles on Expanded Access to Investigational Drugs," PhRMA member companies should consider five factors when deciding whether to grant a patient access to an investigational drug:
- whether the patient has a serious or life-threatening illness and what treatment options are available
- whether the patient is eligible to participate in the clinical trial
- whether there is sufficient preliminary evidence of safety and efficacy to justify the use of the investigational medicine for treatment of the patient and what the potential benefit - risk balance would be
- whether there is sufficient supply of the investigational medicine to support the ongoing clinical trial as well as expanded access
- whether the expanded access program could compromise the conduct of clinical trials needed to obtain regulatory approval
Notably, these principles largely mirror FDA's own regulatory framework for expanded access.
Better Communication with the Public
But PhRMA's new statement goes one step further, saying companies "should establish telephone or Internet-based information sources to facilitate communication about expanded access programs between a patient's qualified healthcare provider and biopharmaceutical companies."
That recommendation largely mirrors a proposed requirement in the Andrea Sloan Compassionate Use Reform and Enhancement (CURE) Act—a piece of legislation seeking to overhaul FDA's compassionate use policies. The legislation proposed requiring all pharmaceutical companies seeking an accelerated approval decision to make publicly available their corporate expanded access policies for compassionate use of their investigational drugs.
PhRMA's executive vice president of regulatory affairs, Bill Chin, said the trade group's new policy reflected the industry's commitment to "ongoing dialogue with all stakeholders to identify ways to optimize the existing federal expanded access process and modernize the clinical trial, drug development and FDA review processes."