By Dana Edwards, director and lead for Patient Services in Life Sciences and Casey Horton, director in Global Life Sciences Governance, Risk Management and Compliance practice, Guidehouse
Thanks to the breakthroughs in cell and gene therapies, more pharmaceutical and biotechnology companies began ramping up investments in research and development of treatment options for rare diseases. Now, hundreds of potential therapies — far more than ever before — are in the approval process with many set to launch in the near term. While this is an exciting time for potential patients and the industry, without carefully plotted paths to access, the patients who need these novel therapies may encounter many roadblocks, and manufacturers may be putting their innovation at risk.
Manufacturers will need to adjust their patient services — with consideration to reimbursement support, adherence, financial assistance, patient assistance, data aggregation, and other services — to become even higher touch to help patients navigate the terrain, or to establish a safety net to catch patients who fall through the cracks of the healthcare system. Furthermore, because the population sizes for these diseases are relatively small, manufacturers may have more insights about individual patients and their lives, which means robust compliance programs must also be instituted along with the fine-tuned services.
For reference, in the United States, a rare disease is defined as a condition that affects fewer than 200,000 people. In the European Union, a condition is considered rare when it affects fewer than one in 2,000 people. An estimated 25-30 million Americans live with a rare disease. There are thought to be up to 7,000 types of rare diseases, but only some categories are reliably tracked — infectious diseases, birth defects, and cancers.
Traditionally, manufacturers focused their efforts largely on selling products in volume to large patient populations. With the passage of the Orphan Drug Act of 1983, which incentivized drug and biotech companies to conduct research and development for novel therapies for rare diseases, more attention than before was paid to identifying novel treatments. Even so, while more drugs were being developed and approved to treat rare diseases, a significant gap remained. Life sciences companies largely continued to focus on developing therapies to treat conditions affecting larger patient populations, until the recent breakthroughs in cell and gene therapy exponentially advanced the possibilities.
As more and more new therapies for rare diseases come to market, manufacturers, providers and payers are all trying to do right for the patient and their own respective businesses. However, “everybody” comprises different stakeholders coming from different angles: payers from coverage, specialty pharmacies and distributors from access, providers from care, etc. It is up to the manufacturer to ensure the entire experience is orchestrated to make it as easy as possible for the patient to access and use these therapies, and as efficient and effective to help them do so for all involved.
In the case of novel therapies for rare diseases, these services become ever-more important and complicated for several reasons, including they generally:
- Cost more to manufacturer and to procure;
- Are produced in limited quantities, and sometimes require patient biology;
- Can treat only select conditions;
- Only select patients with specific medical characteristics will qualify for treatment;
- Require a significant investment in time and resource for physicians to learn how to prescribe and administer treatment;
- Have heightened utilization controls;
- Have limited, usually closed, distribution networks through specialty pharmacy.
In addition, depending on the timing of the launch, manufacturers will need to consider how treatment for the novel coronavirus (COVID-19) has impacted healthcare operations and that may require further program design considerations.
Patients and their caregivers will be best served by manufacturers connecting the services dots to help physicians manage patients and payers manage the treatment options.
Mapping the Product Journey
Because rare disease treatments carry inherent complexity and intricacy related to the product journey, manufacturers need to design their patient services programs with consideration to coordination between payer-provided ancillary services and manufacturer-sponsored patient services, including case management, patient adherence reminders, disease education, and financial assistance options.
A smart starting point is to map the patient journey. Highlight where the patient services from providers, payers, distributor/specialty pharmacy and manufacturer intersect, then design a program that delineates service roles, while empowering the patient at each stage of the product journey. This will also help patients, providers and payers understand who is going to contact the patient when and for what purpose, as well as streamline the process and associated costs for all parties.
Affording Novel Therapies
With out-of-pocket expenses already on the rise for standard therapies, the high cost of novel therapies for rare diseases adds yet another service dimension that manufacturers will need to consider. This is especially considering that costs are likely to continue rising for providers, payers and patients alike.
A recent Guidehouse survey of pharmacy and medical directors across key managed Medicaid and private health plans found that 93% of participants consider rare disease management a priority today, with current priorities most frequently cited to be ensuring guidelines-based prescribing, tracking utilization, and reducing total cost of care. Meanwhile, 32% of payers said they anticipate the spending on rare diseases will increase to account for up to 40% of their budget over the next five years.
In addition, most payers expressed some level of concern over growing out-of-pocket costs and anticipate changes to how the plan considers patient cost burden over the next three-to-five years. In fact, 73% of payers anticipate an increase in coinsurance, and 77% an increase in co-pay, with most payers citing expecting to see these increases in the next plan year. And, while over 60% of payers already offer ancillary services, including a range of diagnostic and support services, as part of rare disease management, respondents suggested use of these services will continue to increase in the future.
With these cost considerations, payers likely will seek to manage the use of these novel products more closely and implement new cost-sharing models. Thus, the path to reimbursement will become even more intricate and difficult to navigate for these novel therapies. At a basic level, patients will need more help understanding how to qualify for coverage, what ancillary services are covered, what their out-of-pocket costs will be, among other economic factors.
As a result, manufacturers need to design their patient service program with consideration to coordination between payer-provided ancillary services and manufacturer-sponsored patient services. This includes helping make sure providers are clear on policies and procedures, especially in relation to prescription, authorization and treatment, as well.
Building a Culture of Compliance
As in the case of any product, compliance should be considered and addressed throughout the product journey. However, products for rare diseases carry a different paradigm than those targeted at the general populace. Foremost, because the patient relationship will be closer and more intertwined with each of the stakeholders, there are more opportunities for (unintended) breaches.
No “one-size-fits-all” exists for compliance, so it will be critical to tailor the program to the novel therapy’s product journey. However, it is generally wise to begin by carefully reviewing each stage in the product journey for potential compliance risks, and then implementing strategies to mitigate them. For example:
- Controls should be implemented and monitored to help ensure any information captured does not violate Health Insurance Portability and Accountability Act (HIPAA) Patient Privacy rules, and that any adverse events (AEs) are recorded and reported properly.
- Robust safeguards need to be put in place to protect patient identity and help ensure protocols are followed, especially because in the rare diseases space manufacturers often have direct relationships with patients. To this end, all field employees should be required to take both HIPAA and AE training.
- To prevent breaches, specific guidelines should be created to outline what field team representatives are and are not allowed to say to patients and providers, what information will be captured about patients, and who has access to that information.
- Compliance should be involved in the design of the materials used for presentations. The information in the compliance-approved materials should be comprehensive and tailored to the individual stakeholders, including healthcare providers, patients, caregivers, and payers. The information should be fair and balanced, as well as include information on both the benefits and the associated risks the product carries. Only information related to the approved indication for the products is allowed to be provided for marketing purposes.
- All patient services should be carefully reviewed for compliance with the Anti-Kickback Statute, which makes it illegal for manufacturers to knowingly or willfully provide remuneration to providers. In this context, manufacturers must be careful not to give services to providers that providers would otherwise have to fund and manage themselves.
To develop and institutionalize a robust compliance regimen, the manufacturer’s compliance team should be given descriptions of all the field roles, as well as the goals of these roles, how each is compensated, and how targets will be determined. This will help them design a program specific to the therapy option.
Taking Hold of the Future
Today, as manufacturers prepare to bring hundreds of novel therapies for rare diseases into formulary management, they should also aim to meet a new standard for developing and implementing patient support services. The breakthroughs in biotechnology and pharmaceuticals are ushering a new era for the treatment of rare diseases in the United States and around the world. As more and more novel treatment options gain approval and come to market, the manufacturers that adjust their patient services models to keep pace with the advancements will be in the best position to help patients gain access to their products.
About the Authors
Dana Edwards is a director and lead for Patient Services in Life Sciences at Guidehouse. With more than 20 years of experience in the pharmaceutical and life science industry, she helps pharmaceutical and biotech manufacturers identify solutions to support patient access to therapy. She has led the design, development, and implementation of programs servicing both patients and providers to support patient access to and coverage for high-cost therapies. Her focus on quality assurance and process improvement has led to innovative work processes and system solutions to facilitate a portfolio of services addressing complex reimbursement issues across payer types.
Casey Horton is a director in Guidehouse’s Global Life Sciences Governance, Risk Management and Compliance practice, He brings over 20 years of experience leading risk, compliance and operational improvement projects in the U.S. and across the globe. Casey brings Guidehouse clients a unique perspective and understanding of the regulatory and operational challenges facing life sciences companies. His practice focuses on performing compliance assessments and investigations, reviewing and optimizing organizational processes and implementing effective and sustainable compliance solutions. Casey brings deep experience and knowledge of compliance program development, Corporate Integrity Agreements, compliance and enterprise risk assessment execution, global monitoring and investigations initiatives, anti-bribery / anti-corruption issues, third-party due diligence review programs, patient services and assistance program reviews, and financial and economic damage calculations.