The 21st Century Cures Act (Cures Act), which was passed by Congress, includes mandates to provide the U.S. Food and Drug Administration (FDA) with resources to modernize the Agency’s scientific and regulatory programs. Patient-focused drug development (PFDD) is one of the strategies. The Cures Act creates a window of opportunity for reliance on data developed in less traditional forms, most notably real-world evidence (RWE) like patient reported outcomes (PROs).

Professionals in commercial roles have an unprecedented opportunity to proactively and efficiently help lead their companies along the PFDD continuum. This requires partnering with legal, regulatory, and compliance colleagues to stay ahead of risk and leverage business opportunities, including the use of digital technology to collect data.

Guidance Expected From the FDA

To guide the industry in incorporating relevant patient experience data into drug development, the FDA is expected to release a Guidance this year.

Patient experience data is defined as “data collected by any person (including patients, family members, and caregivers of patients; patient advocacy organizations; disease research foundations; researchers; and drug manufacturers) that are intended to provide information about patients’ experiences with a disease or condition. The term specifically includes data regarding (a) the impact of the disease or condition, or a related therapy, on patients’ lives; and (b) patient preferences with respect to treatment of the disease or condition.”

“Barriers to finding patients who can add significant value to drug development have eroded dramatically,” said David Goldsmith, Chief Strategy Officer, WEGO Health, which works with companies to leverage patient experts. “Companies today can readily find patients who are deeply knowledgeable, highly engaged and open to sharing experiences and data to advance product development.”

Companies and Patient Groups Must Partner

When developing patient-relevant products, companies must partner with patient communities and identify patient key opinion leaders as early as possible in development. These audiences can be consulted on unmet needs at the pre-clinical stage. Aimovig, launched May 2018 through a Novartis and Amgen Neuroscience Partnership, exemplifies a product development process incorporating the patient’s voice at the beginning of the trial. As a result, PROs are included in the labeling for this new migraine treatment.

“Interacting with patients identifies their needs and network of influencers, including patient support groups, infotech companies, healthcare systems, specialty pharma, and academia,” states Dr. Francoise Simon, Professor at Columbia and the Mount Sinai School of Medicine, author of Managing Biotechnology: From Science to Market in the Digital Age (Wiley 2017).

When interacting with patient groups, companies must continue to define compliance guardrails, states Howard Dorfman, a legal, compliance and regulatory expert in life sciences. “Over the past several years, government agencies such as the Department of Justice and Office of Inspector General have examined relationships between manufacturers and patient support group programs for possible violations of healthcare compliance statutes,” explains Dorfman.

“These include company-sponsored Patient Assistance Programs, Patient Advocacy Programs, and utilization of Specialty Pharmacy distribution systems to obtain unfair or illegal commercial advantages.” Dorfman adds that patient privacy and restrictions imposed by federal (HIPAA) and state laws have given rise to civil liability arising from public and private litigation.

“I advise clients to interact with the FDA early and often because ultimately the issue lies with regulatory acceptance of technologies that capture real-world data and use of surrogate endpoints,” says Kerry Rothschild, President of KGR Pharma Consulting. “For example, can companies make an efficacy claim if a digital device confirms that an arthritis patient has moved more during the day? Does that mean the therapy works better for pain?”

Grace Castillo-Soyao, CEO of Self Care Catalysts (SCC), says that PRO collection platforms must be flexible enough to adapt to the compliance needs of diverse companies, including by not limited to their interpretation of data privacy laws such as HIPAA and General Data Protection Regulation (GDPR) legislation. SCC has developed a SaaS-based PRO collection tool that is used during clinical trials as well as in the commercial stage.

Value-Based Purchasing

By 2018, the Center for Medicare and Medicaid Services (CMS) wants 50% of Medicare fee-for-service payments tied to quality or value through alternative payment models. Manufacturers will need evidence of positive outcomes to support value-based payments. So, PFDD clearly becomes an important lever for enabling manufacturers to prove that use of their drug will reduce risk of more expensive treatments, while preventing payers from searching for less expensive alternatives. By focusing on patient needs, insights, and behavioral drivers, there is a greater likelihood that outcomes will be improved.

Meanwhile, under a value-based purchasing arrangement, formulary placement, co-payment tiers, and pricing may depend on clinical outcomes or the impact of drug therapies on clinical costs. Companies agree to share in the risk associated with a drug’s “success” or “failure” to help mitigate market access restrictions or justify higher price points. For example, if actual patient outcomes are not as successful as outcomes reported in clinical trials, a company might offer enhanced rebates on drug costs, or reimburse for cost of treatment complications.

“Companies need to understand the whole cost of treatment and recovery to determine which costs may be reduced by successful therapies. This provides a value-based justification for the pharmaceutical cost component of treatment,” says Michael S. Varadian, JD, MBA, and former director of a national behavioral health managed care company.

Insurance companies can also calculate avoided medical costs through individual or combined efforts of providers, manufacturers, and payers. “Companies need to watch overlaps between the three entities to avoid sharing costs and prices, which could violate antitrust regulations,” cautions Varadian. “In some instances, treatment gains may be achieved by off-label use of pharmaceuticals. Industry needs to be careful to avoid these circumstances, which risk regulatory and legal reprisals, particularly when problems arise in treatment or recovery from unapproved usage of medications.”

Concludes Dyan Bryson, President of Inspired Health Strategies: “Ultimately, the PFDD guidance will change how industry thinks about the value of patient insights. What has been a “nice to do” will become a “must do” and, most importantly, can impact patients’ lives in a very real way.”

  • Ilyssa Levins

    Ilyssa Levins is President and Founder of Center for Communication Compliance (CCC). Ilyssa helps manufacturers and their promotional agencies save time, money and manage risk, when promoting drugs and devices. She combines her regulatory compliance, marketing communications, and change management expertise to help clients achieve their goals.


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