Key decision makers throughout the pharmaceutical industry now have a better sense of just how difficult the past two years were for all Americans who contended with the challenges of the global COVID-19 pandemic. But the amplified struggles of the rare disease community are even more pronounced for specialty drug manufacturers amid growing optimism that the year ahead will bring numerous FDA-approved specialty drugs and cell and gene therapies to the marketplace. Identifying some of the key market trends helps all stakeholders to better gauge the impact of products and services, initiate changes and respond to market changes in order to achieve better quality of life for patients with rare disease or complex conditions.
Ensure Optimal Medication Access
As the marketplace places a greater focus on patient-centric care, patient care coordinators are likely to play an expanded role, streamlining communications between prescriber, patient and specialty pharmacy which increases patient engagement, decreases time to fill, and optimizes outcomes. They will also provide broad specialty therapeutic expertise needed to rapidly identify copay assistance and other financial resources, alleviating financial barriers, helping to ensure patients start and stay on therapy as prescribed.
Greater Focus on Rare Disease Patients
While the patient community has contributed to the commercial success of treatments for orphan and rare diseases – despite the exorbitant cost of drugs – the private sector is spurring investment, informing research and influencing policymakers to continue their support for research and development that encourages development of specialty drugs, including cell and gene therapies. Affordability challenges are currently being addressed by Specialty Pharmacy (SP) solution vendors with cell and gene therapy expertise and innovative programs to enhance access by introducing new financial assistance models that offset high-prices as well as providing distribution channels that are expected to unclog the issues surrounding supply chain.
Carving Out Prescription Medical and/or Pharmacy Benefits and Contracting
Increasingly, self-funded employers, Plan Sponsors and other payers will carve out specialty prescription drugs and pharmacy benefits from their medical plan by contracting directly with an SP provider. As costs for specialty medications continue to rise, rethinking contractual partners enables these payers to gain better controls over SP costs and greater transparency into their benefit claims, with enhanced negotiating power to get better deals and ensure that clinical and financial programs performed as promised. Further fueling this trend is the potential to achieve an average savings of 5-30% through plan language and benefit design. As this transition to specialty drug carve-outs builds, SP service providers will be in even greater demand to help payers implement unique Financial Assistance programs for the numerous high-cost gene/cell therapies and other expensive specialty medications that are being launched to treat rare and orphan diseases and complex conditions.
Unique and Expanded Financial Solutions Emphasizing Value-based Contracting
Going forward, expect to see a seismic shift toward value-based (VB) or outcomes-based contracts that can promote greater patient access to new biopharmaceutical treatments by linking reimbursement, coverage or payment to a treatment’s real-world performance and patient outcomes. For VB payment models to achieve their full potential, the support of SP solutions vendors that have deep expertise and experience with this contracting model will be required. Select SPs are pioneering innovative technology-based suites of unique financial solutions, including loan-based programs for cell/gene therapies to help offset the high cost of curative medications and copay advisory services to monitor and track manufacturer copay funds, providing full transparency into copay transactions, ensuring that allotted funds are utilized to help offset costs of expensive therapies for patients.
Soaring Costs of Cell and Gene Therapies (CGT)
There is every expectation that the costs of CGTs will continue to rise, putting pressures on employers, state Medicaid programs, commercial payers and providers throughout the health ecosystem to seek innovative solutions for absorbing these costs. The average gene therapy is likely to cost at least $1.5 million, according to Tufts Medical Center's NEWDIGS Initiative. Multi-million dollar price tags are becoming more common, as the FDA recently approved Skysona for a rare neurological disorder called cerebral adrenoleukodystrophy at a list price of $3 million. Another concern is the longevity of response to the product, since it may be too early to tell how long the effects of the treatments will last – a lifetime vs. a specific time period. To address these issues, progressive SP service vendors help track outcomes of novel treatments which may be tied to reimbursement.
Early Commercial Planning for Product Launch Success
Meeting launch milestones – from start to finish – will require meticulous planning and disciplined execution to ensure long-term market success throughout the product lifecycle. This process should get underway well in advance of real-time market entry, usually 12-24 months prior to launch. Informed go-to-market strategies will be more important in the year ahead, supporting not only manufacturer business goals, but also the special needs of patients with orphan, ultra-orphan and rare diseases. Increasingly, specialty drug companies will require an experienced partner like AscellaHealth and its subsidiaries, to provide end-to-end solutions that clear the barriers to successful market access: optimizing pricing from day one, ensuring patient access to therapy, fostering patient adherence to treatment, improving patient outcomes and building brand loyalty.
The transition to serve rare disease patients worldwide with access to specialty drugs, cell and gene therapies will intensify the need for global expertise to bring these products to market globally. SP solutions vendors with international reach are positioned to assist pharmaceutical companies in these efforts, offering innovative pharmaceutical financial solutions such as loan-based assistance programs that help payers and hospitals to fund these expensive products.