Off-label drugs present many treatment opportunities.
They can be used to treat conditions or given in dosages that the FDA has not yet approved. This means that doctors can prescribe them but pharmaceutical companies are not allowed to market them to the public. To get these drugs then to patients to help treat their conditions, the role of the medical affairs team becomes paramount.
It’s the MA team and its medical science liaisons that are tasked with forming close relationships with physicians and key opinion leaders to educate them about a drug’s off-label benefits. Indeed, researchers at McKinsey & Company call the medical affairs team “the natural owners of scientific knowledge and data within the organization and across the lifecycle.”
In this post, we explore the benefits of prescribing off-label drugs and how MA teams can help facilitate the process of getting those drugs to patients in need.
Oncologists frequently prescribe off-label drugs. For example, a chemotherapy drug could be used off-label because the FDA approved it for treatment of a certain type of tumor only.
The premise of such a treatment approach is that existing drugs could treat patients, negating the need for new drugs to be developed. This is the theory behind the Drug Rediscovery Protocol, an adaptive, precision-oncology trial in the Netherlands, senior study author Emile Voest, M.D., Ph.D., tells science writer Laura Elizabeth Lansdowne.
The Drug Rediscovery Protocol explores the efficacy of anti-cancer drugs to treat advanced cancers outside of their approved label. Patient eligibility was based on a genetic or molecular variant that was matched to an off-label drug. Voest says through a program of large-scale sequencing his team has discovered that 13 percent of patients have a molecular target they have a drug to match.
Cancer patients and their doctors tend to be more willing to try off-label drugs, writes biochemist Chelsea Weidman Burke. And it’s not just cancer-specific drugs that are used off-label. “The anti-anxiety drug Ativan (lorazepam) is often used off-label as an anti-nausea drug and in an off-label route (under the tongue rather than the approved oral, intravenous, or intramuscular routes) during cancer treatment,” Weidman Burke explains.
FDA-approved beta-blockers for high blood pressure treatment have been used frequently to treat heart failure. Indeed, this has become so commonplace, some beta-blockers now have FDA approval to treat heart conditions. Similarly, tricyclic antidepressants treat chronic pain, while antipsychotics can be used to treat ADHD and other psychological disorders such as OCD, insomnia and anxiety, adds Weidman Burke.
The nonprofit organization Cures Within Reach, which repurposes medical research for new treatment applications, has funded 10 drug repurposing projects, says science journalist Anna Azvolinsky. The public charity is also testing current drugs for novel indications.
Thalidomide, for example, was approved in Europe in the 1950s as a sedative; 38 years later in the U.S., it was used to treat leprosy. It was also one of the first compounds researchers suggested for repurposing, Azvolinksy explains. Cures Within Reach supported the phase II trial necessary for the compound to be approved for the treatment of multiple myeloma. Because it had already been tested to treat leprosy, phase l safety and dosing trials were not required, saving researchers years of work. By 2012, the FDA had approved thalidomide for the treatment of multiple myeloma.
Cures Within Reach founder Dr. Bruce Bloom says the financial savings were also significant, as FDA approval in this case cost around $40–$80 million. Compare that with the average cost of drug development (from inception to approval) which runs anywhere from $1-$2 billion, writes Azvolinksy.
The job of a medical science liaison is based on relationship building. Providing hard science to doctors and key opinion leaders, the MSL informs, educates and engages the healthcare community on matters related to disease and treatments. That includes discussing the value of off-label drugs, explains clinical pharmacist and medical liaison Nabeel Qureshi.
The truth is doctors don’t have time to stay updated with the latest clinical trials and off-label uses, note consultants at management consulting firm L.E.K. This makes the job of medical science liaisons crucial in that they can provide healthcare professionals with this vital knowledge.
MSLs are uniquely placed in terms of their scientific knowledge, understanding of product data and ability to communicate what is most relevant to their medical audience. When this data is packaged in a manner that is accessible, understanding off-label drug use becomes far easier for doctors and other healthcare providers to appreciate.
The role of the medical science liaison is also indispensable to the commercial success of pharmaceutical organizations. Because there are regulatory limits on what a pharma marketing and sales team can do, the MSL sits apart as a reliable, evidence-based path into current research, explains Hillel Kuttler at the American Academy of Physician Assistants.
Indeed, the Medical Science Liaison Society describes the MSL role as such: “They work throughout a product’s lifecycle, help to ensure that products are utilized effectively, serve as scientific peers and resources within the medical community, and are scientific experts to internal colleagues at companies.” Their primary purpose is “to establish and maintain peer-peer relationships with leading physicians … at major academic institutions and clinics.”
The role is growing in popularity and relevance too. Research from the Medical Science Liaison Society shows the MSL role could grow by 20 percent in the next year or two, with an even bigger growth predicted in Asia and South America, writes business coach Porschia Parker. MSLs are working in biotech, pharma, and medical device manufacturing organizations.
Key opinion leaders are highly trained and professionally successful, very often leading physicians at major academic institutions and clinics. They want knowledge and valuable insights into current medical research. They also want mutually beneficial relationships with the clinical research and development communities, writes Felix Jansson, at cloud-based stakeholder platform Monocl.
So a medical science liaison reaching out to a key opinion leader should aim to highlight the opportunities that current clinical research presents such as investigator-initiated studies, speaking engagements and teaching opportunities. Jansson encourages MSLs to engage key opinion leaders through valued incentives. To do this, the liaison needs to understand what the opinion leader wants, values and hopes for in their field of interest.
Social media influencers are well established. And they’re getting in on the action when it comes to medicines too. The influencers are usually patients, notes biotech reporter Kate Sheridan. Their following is small but committed and the relationship between influencer and follower is based on trust. There are even organizations, such as Wego Health, that connect influencers with pharma.
Given the nascency of this approach, regulators are understandably questioning the method. “There’s a myriad of ethical issues around this,” says Lisa Gualtieri, assistant professor at Tufts University School of Medicine, who has studied how social media is being used to promote off-label drugs. “What’s the responsibility of various stakeholders to educate people that this is potentially dangerous and harmful?”
Currently, the situation is such that the pharma company has to trust the influencer to stay within the ambit of FDA rules. However, for a medical affairs team, building the right type of relationships with influencers — based on scientific information — could be a powerful means of informing patients of off-label drug uses.
Still, the FDA rules are clear: pharma companies cannot market off-label drugs. This is understandable but also frustrating as these drugs can be used to treat more conditions than their approval scope allows. So doctors become a patient’s best hope of being given access to these treatments. It’s up to the MA teams and their MSLs to connect with the relevant doctors, opinion leaders and possibly even patient influencers to educate the medical community about the efficacy of off-label drugs.
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