The Pulse of Rare Disease Support

Industry-sponsored patient support programs have become an essential “lubricant” for coordinating care of patients with complex medical needs. By bringing together the drug developer with the healthcare providers (HCPs) and the payer network, therapies can get to patients sooner, with better overall results for all. Especially with complex specialty pharmaceuticals. And most especially for the fast-growing field of rare or orphan diseases.

That being said, patient support programs, or hubs, remain an underutilized part of healthcare. A 2020 survey from Accenture found that only 18% of patients are aware of the programs being offered—a number essentially unchanged from 2015, when an earlier survey was performed. This is despite indications that industry is funneling more funding into the programs.

There are two basic problems that account for this lack of uptake, according to Anthony Romito, life sciences lead at Accenture. One is organizational issues inside pharma companies that limit how well the organizations promote their services; another is the lack of communication to HCPs of the value of patient support.

On the organizational side, “Most organizations’ hubs are either outsourced and operated by a third party, or they sit in med affairs or market access” departments, he says. “For brand teams, these organizations are not necessarily knitted into the DNA of their go-to-market strategies; there are big gaps despite increased investment.”

Equally problematic, “Pharma companies have generally done a poor job of measuring the clinical effectiveness of patient-support programs, and so HCPs have been reticent to recommend them to their patients.” However, Romito sees some progress here: demonstrating values like reducing time to first dose, or improvement in patients staying on therapy, enable the pharma companies to demonstrate value to the HCPs.

Romito cites the variety of support programs sponsored by AbbVie for its blockbusting Humira treatment (currently the most valuable drug in the world, with sales of $20.7 billion) as an industry pacesetter. “AbbVie has been diligent in demonstrating longer-term patient retention and better outcomes through its support programs,” he says. The equation is simple: provide the service, measure the outcome, and communicate to HCPs.

Even so, industry appears to be moving in the opposite direction when it comes to close coordination of patient-support services and product management. Helio Health Group, a consulting firm, has surveyed the field for five years running; its 2021 survey, published in December, showed that, when a patient-support program exists inside a pharma company, it was placed within the brand/commercial team at 37% of pharma companies in 2017—but only 8% were so situated in 2021. (Conversely, there was growth in placement in managed markets teams, and dedicated patient-service teams.) A major reason for this decline is the difficulty in maintaining a compliant program, avoiding such federal restrictions on patient-data access, or providing improper financial inducements to patients.

Beyond prescriber paperwork

The ”classic” (circa five to 10 years ago) hub program was focused on moving patients through the obstacle course of paperwork for getting a prescription and getting it reimbursed. The process often starts with a benefit verification (BV) process to determine insurers’ policy on a drug or therapy. Payers’ use of prior authorization (PA) procedures require physicians to complete sometimes complex diagnostic evaluations, and symptom descriptions. Payers can also require so-called step edits that call for trying a variety of other treatments (usually, the less expensive ones) before the physicians’ preferred choice is permitted.

All these complexities are magnified for many rare diseases which, as Jan Nielsen, division president for patient support solutions at AssistRx, says, can take upwards of six to nine years to correctly diagnose. Testing that might originate with a patient’s primary care physician can itself require a fight with an insurance program’s benefit structure.

For the most part, patient-support providers have built information technology (IT) tools and databases to handle the paperwork, so now there are eBV and ePA resources to be used either by physicians or by the hub providers to assist physicians’ offices. Similar programs are in place for copay analysis and availability, and in some cases, patient assistance programs sponsored by manufacturers. (These have their own requirements for financial disclosures, and might have only limited durations during the patient journey.)

So-called “hub-light” programs pretty much start and stop with these digital services (perhaps with some in-person call- center activity included). But for complex pharmaceuticals, and especially for the newly establishing field of cell and gene therapies (CGTs), many more services are wrapped around the prescription. “There are between 5,700 to 6,000 preclinical and clinical assets that are being developed globally in terms of CAR-T and inclusive of gene therapies,” says Maria Kirsch, GM and head of patient services at Eversana. “This will be a massive wave of the most powerful therapeutic modality in the history of our industry that will address some of the most severe unmet needs for cancer patients, rare disease patients, and other patients who are, unfortunately, on their last hope of treatment, as they have failed other treatments.

“Taking that a step further,” she adds, “We estimate approximately 900 assets are in clinical-stage development. There is a higher likelihood of clinical trial success, for example, with Phase III, CAR-T programs, so hundreds of new CAR-Ts or cell therapies coming into the market will help patients’ lives, but make no mistake, there will be major implications and a significant amount of overhaul to our healthcare systems globally, to be able to absorb the clinical impact, operational impact, and financial impact of these therapies.”

Eversana supports seven exclusive rare disease products, according to Kirsch, “all with patient-service offerings being a critical component.” For companies like Eversana and others, patient support in rare diseases often involves coordination with family caregivers, in addition to the interactions with providers and payers. An estimated 60% of patients with rare diseases are under the age of 18, and a high proportion of such diseases involve chronic care. “We are often working with parents or caregivers to coordinate therapy, schedule adherence plans, and coordinate life coaching for major life events like starting school, going to college, or starting a family,” says Kirsch. “Through education and close connections with patients, we are able to anticipate challenges they may have, such as dietary restrictions, and provide them with the skills to overcome them.”

By contrast, the appeal of many of the CGTs—commercial or in clinical-stage—involve what amounts to a “one-and-done” procedure (at least, this is the promise of many of them). Once a patient’s cells have been collected, manipulated at a cellular or genetic level, and reinfused, the potential for a near-complete cure of a cancer or birth defect is high. Getting the patient to that point, however, can require travel accommodations (since many of these therapies are available only at selected medical centers), multiple trips, and—most significantly—long-term follow-up to ensure that the therapy is permanent.

“Give credit to Novartis for getting an OIG (Office of Inspector General of HHS) ruling that allowed low-income Kymriah patients to have their travel and related services legally reimbursed,” says AssistRx’s Nielsen. This occurred in early 2021; Kymriah, one of the first commercial CGTs, nominally costs $475,000 per patient. For support programs like these, AssistRx enables patients to be quickly reimbursed via e-banking services like Zelle or PayPal.

Mention of the HHS OIG highlights the line patient support providers must constantly adhere to, because poorly monitored practices can lead to serious HHS violations. Helio Health, which runs the aforementioned survey, notes that manufacturers have been increasing their oversight of nurse educator practices and awareness of patient privacy requirements, while reducing their support of some patient assistance programs (which provide direct financial support). Caution is the watchword.

Clinical to commercial

It’s hard to say whether the pharma industry’s emphasis on CGTs is influencing patient-support providers to get into clinical trial engagement, or whether those providers’ growing expertise is being better appreciated by clinical trial managers, but the upshot is that there is substantial value in providing patient support during trials, and business is growing for those providers. This trend has also been boosted by the pandemic, which drove many clinical trials into a decentralized, remote-services version.

“Over the past 18 months, we’ve become engaged in about a half-dozen programs,” says Nielsen. The main driver is trial patient retention. “Especially for some rare diseases with limited trial populations, the need to keep patients in the trial is critical,” she adds. “Service providers like us provide adherence nurse educators, helping remote patients with complex diagnostics and drug-delivery devices, or complicated drug regimens. We’ve become the 24/7 hotline for trial patients.”

In a February announcement, Eversana debuted a data-driven service for clinical researchers by partnering with Thread, a provider of decentralized clinical trial management and clinical outcome assessments (eCOA). The company will combine its commercial capabilities in linking real-world data and electronic health record (EHR) data with Thread’s, thus enabling “continuous active, passive, retrospective, and prospective data collection with patient consent and identity security as central tenets,” according to Eversana. Another Eversana partner, Datavant, provides patient privacy and security.

“In the evolving world of real-world data, registries, and clinical trials, it has become increasingly likely that a patient could end up participating in all three through the course of their care journey,” says Eversana’s Kirsch.

The clinical-to-commercial spectrum could have important benefits for winning over HCPs by the time a drug is approved. Accenture’s Romito says that the ideal entry point for getting patient-support programs engaged is in Phase II of a drug trial. “Today we have long lag between measuring the clinical value of the drug and the impact of the patient support service,” she notes. “There are untapped opportunities to have collaboration between the clinical development team and the commercial development team. We’re seeing more of this in the CGT space, with less walls between two organizations.”

Put it all in the cloud

There are unplumbed opportunities in bringing advanced digital technologies, some of them cloud-based and utilizing artificial intelligence, to better serve patients and the clinical research organizations driving for drug approvals or for payer acceptance. Accenture has rolled prior work in digitizing patient services into a broader, cloud-based platform it calls Intient. Eversana has a “next-generation” patient-services platform called Actis Patient Relationship Management, which promises to use predictive analytics and behavioral science to improve the patient experience.

Another approach is from AiCure, which offers expertise in what it calls “digital biomarkers” of patient condition. It started out being able to confirm a patient taking a clinical trial drug dose by having the patient do a video recording of the act with the patient’s smartphone. More recently, it has developed methods to observe patient condition for diseases such as Parkinson’s to assess whether in-person intervention is needed. While the technology has been used mostly during clinical trials, “We are in discussions with patient support-groups to engage with them,” says Rich Christie, MD, PhD, chief medical officer of AiCure. Most recently, it has partnered with Science 37, a clinical trial IT provider, to monitor patient condition for those suffering from depression.

Yet another approach is being championed by BrightInsight, which offers a compliant, cloud-based platform with which to combine apps, devices, companion diagnostics, and other resources for remote patient support. A showcase example is a partnership with CSL Behring for the latter’s Hizentra (immune globulin subcutaneous) drug, used for the rare diseases primary immune deficiency or chronic inflammatory demyelinating polyneuropathy. These serious conditions require patients to self-administer Hizentra multiple times daily. The BrightInsight platform enables CSL Behring to provide a smartphone app to manage infusions, get reminders, and record and share their infusion journal with their doctor.