The pharmaceuticals world is accustomed to being at the center of raging debates on the morality of drug benefit vs. associated risks. Following President Trump’s mention of “Right to Try” in the State of the Union speech in late January, the topic of the compassionate use (also known as expanded access) of unapproved drugs for terminally ill patients has suddenly become a mainstream issue.
The U.S. Senate passed the Right to Try Act of 2017 last August, and leaders in the U.S. House of Representatives have pledged to quickly bring back an updated version of their Right to Try bill that failed last week to pass a super majority vote. With the President’s vocal support on this matter, it is highly probable that the House will pass some version of the bill.
An abundance of views is being offered by proponents and opponents in this debate, but there is very limited public discussion of pharma’s roles and responsibilities. Will a federal Right to Try law move the spotlight away from the U.S. Food and Drug Administration (FDA) and onto individual pharmaceutical companies whenever a patient is denied access to an experimental drug? It’s more critical than ever for every company to define policies, roles and processes that are consistent, transparent and clearly communicated to the public.
What Will Change; What Will Not
The core dynamic between patient and physician will continue to initiate and drive the Right to Try process. The physician plays several active roles in this regard:
- Advocating for the compassionate use of experimental drugs on behalf of his or her patients.
- Engaging the pharmaceuticals company to ensure that the potential benefit clearly outweighs the risks.
- Completing an extensive FDA form when the sponsor says yes.
- Overseeing administration of the drug.
A federal Right to Try law may streamline the process for the physician, but it is unlikely that the role of physicians will fundamentally change between compassionate use and Right to Try programs.
On the other hand, once the Right to Try Act is passed, the obligations of pharmaceutical companies will certainly change. Today, companies are not required to report on any aspect of their compassionate use or expanded access programs. Under the latest House version of the Right to Try Act, pharma will need to report annually on the number of doses supplied, the number of patients treated, the uses for which the drug was made available, and any known serious adverse events. Most likely, there will be an increased expectation to provide terminally ill patients with access to investigational drugs.
Pharma companies must be ready to handle these new expectations and public reporting requirements. Many of the systems currently in place to deliver investigational drugs to clinical trial participants and compassionate use patients can be leveraged, but the ecosphere needs to be upgraded to handle an undefined increase in demand and ethical considerations.
A Right-to-Try Framework
Let’s examine how the familiar but powerful framework of people, process and technology can be applied to meet the new demands of Right to Try.
- People: Pharma companies will need to establish an internal organization that can be truly impartial in making decisions on the patient’s right to try an investigational drug that has only completed Phase I testing. This organization should be shielded from the pressure of proving success in clinical trials at one end and commercial success at the other. Considering the complexity of rare and specialty diseases and the emotional state of patients and family members, this group needs to be staffed with people who possess a strong medical and science background and have the maturity to make fair decisions.
It’s equally important to provide visibility on the group’s role and ensure there is an organizational mandate for leveraging this group across all scenarios of unapproved use. Medical affairs professionals and scientific liaisons can initially staff this organization, but it’s important to maintain the distinct identify of this group. Think of it in the same way that grant management became a new functional group once the Sunshine Act was passed.
- Process: Most drug companies don’t have published policies on compassionate use, or at least they don’t make them easily accessible. A review of more than 100 companies developing multiple potential cancer treatments shows that fewer than 20 had compassionate use policies clearly posted on their websites. Over the past few years, there have been several examples of social media campaigns forcing a company to provide its drug for compassionate use, and these cases will most likely multiply in the future, with or without a new federal law. To ensure fairness and reduce the chance of legal complications, it’s extremely important for companies to establish a clear process for accepting or rejecting requests for compassionate use.
Just as companies began to define an appropriate Risk Evaluation and Mitigation Strategy (REMS) prior to commercialization in order to comply with the 2007 Food and Drug Administration Amendments Act (FDAAA), pharma should establish a similar approach: develop a Compassionate Use Risk Evaluation Strategy – what I’ve coined a CURES – for each investigational new drug as soon as results from Phase I safety testing in humans are available. Mechanisms for continuous training on CURES as new clinical trials start, either for new investigational drugs or for new indications for existing drugs, must also be developed.
- Technology: No one can be certain of the requirements that a finalized Right to Try law would include and how it would impact the volume of requests to pharma. Thus, it’s important to plan to leverage a technology platform that can help in the entire lifecycle of patient risk. Easy-to-use channels such as an external website or a mobile app should enable patients and caregivers to search for clinical trial information and allow physicians to submit and track Right to Try requests, securely and privately. Internally, the request should trigger a workflow system that facilitates data collection and decision-making by medical specialists. Pharma’s ability to face public, legal and regulatory scrutiny depends on having an internal system that:
- Captures all aspects of approving or rejecting requests to use an investigational drug outside of a clinical trial.
- Reports on the number of patients and doses per investigational new drug and indication.
- Sends real-time notifications to the FDA about new Right to Try patients and serious adverse events.
Preparing for Right to Try
A standardized framework of people, process and technology will create the ecosystem needed to get ahead of any potential Right to Try law. It will help pharma avoid the negative publicity and reactive decision-making that occurs when social media campaigns are launched by patients and their families feeling desperate and left in the dark. An independent, impartial organization supported by a transparent process and enabling technologies can go a long way toward ensuring that legal, moral and financial risks can be managed effectively.
The implications of a federal Right to Try law on the role of the FDA and payers are equally large and complex, so I’ll address these topics in upcoming blogs.