Rare Disease Pharmacy Insights

Rare diseases affect over 300 million people globally, yet treatments remain elusive due to challenges in research funding and patient trials. Orphan drugs, developed for such rare conditions, offer hope, having received a boost from governmental incentives like India's National Policy for Rare Diseases 2021. This policy supports financial accessibility, enabling breakthrough therapies like Zolgensma to transform patient outcomes.

Despite progress, barriers persist, including high costs, stringent regulatory processes, and limited drug availability across countries. Innovations such as gene therapy and biologics signal potential for future one-time cures, though affordability and access remain critical issues. In balancing innovation and affordability, pharmaceutical companies are pivotal in making these life-altering treatments broadly accessible, underscoring the importance of global collaboration in rare disease treatment advancements.

Lumicera Health Services has entered a significant purchase agreement for a biosimilar of Stelara, a prominent yet costly drug treating various inflammatory conditions. With this agreement, Lumicera aims to alleviate the financial burden on plan sponsors by reducing costs between $112,000 and $336,000 per patient annually and generating $120 million in client savings. This approach offers immediate affordability through their cost-plus model, bypassing traditional rebate delays.

Lumicera, a subsidiary of Navitus Health Solutions, uniquely focuses on maintaining medication cost transparency and prioritising specialty medication accessibility. As Navitus plans to remove Stelara from its formulary by July 2025, this transition promises minimal disruption due to Lumicera's dedicated patient support. Their efforts, combining strategic negotiation and personalised patient care, reinforce their commitment to making essential treatments attainable for all.

Highmark has transitioned from exclusive relationships with specialty pharmacies to a more patient-centric approach, connecting with Free Market Health. By focusing on individual patient needs and specific conditions, it aims to improve outcomes and reduce medical costs. This new relationship now adapts to diverse pharmacies, from health system and community settings to those specialising in niche disease states.

This strategy allows Highmark members to benefit from tailor-made healthcare experiences. Even though the decision burden is heightened, the potential to create value within an open network model is substantial. Highmark hopes to demonstrate that aligning pharmacy services more closely with patient requirements will enhance care effectiveness and cost efficiency.

As AI integrates more deeply into specialty pharmacies, significant shifts are on the horizon. According to a survey published in the Journal of Managed Care & Specialty Pharmacy, 62% of participants expect a surge in drug shortages by over 25% in the next five years, affecting patient access to essential medications. This paints a challenging landscape where AI's role is both transformative and tumultuous, like rolling a set of magical dice where the stakes are high.

Additionally, 92% anticipate AI's integration into over half of prior authorisation processes, potentially streamlining operations but also hinting at growing complexities. Drug costs remain a pressing concern, with 54% forecasting that numerous states will create drug-affordability boards. Furthermore, there's a predicted 25% increase in GLP-1 therapies. As the pharmacological world edges into this new era, the balance between innovation and accessibility will be crucial, requiring an agile response from healthcare stakeholders.

The No One Waits (NOW) study innovatively approached hepatitis C treatment by integrating a pharmacy team with direct involvement in delivering care. Participants, primarily from socially marginalised groups in San Francisco, began treatment instantly after diagnosis, thanks to a two-week starter pack. This practical model flouted usual delays tied to insurance formalities, showcasing a refreshing immediacy in healthcare. The partnership with a specialty pharmacy enabled 90% of the participants to transition to insurance-covered medication rapidly, addressing systemic barriers.

Structured to tackle hurdles like prior authorisations and medication delivery, the NOW model offered a glimpse into a more accessible health framework. By employing a streamlined testing and treatment initiation in non-clinical settings, this method links the dots between diagnosis and treatment, illustrating a viable pathway for similar populations nationwide. The model not only adhered to WHO treatment guidelines but also proved effective in a real-world scenario, hinting at broader applicability.