Advancing Rare and Gene Therapies: Specialized Support for Manufacturers

Mark Jacob, Product Management Senior Director
Mark Jacob
Product Management Senior Director
Feb 21, 2024
my care connect infographic

Accredo’s My Care ConnectSM service model connects the dots for rare and gene therapy manufacturers.

There are challenges when you are transforming lives.

Rare and gene therapy manufacturers are at the forefront of transforming the lives of individuals grappling with rare, ultra-rare, chronic, and incurable health conditions. The journey to develop these groundbreaking medications is marked by formidable challenges, including large investments in research and development for such a limited population. Recent estimates put the costs of developing a new drug at staggering figures, ranging anywhere from $314 million to $2.8 billion.

To maximize the impact of these critical therapies and ensure widespread accessibility, manufacturers must rely on strategic partnerships with a specialty pharmacy to bridge the gap between innovation and the patients who need them. This article explores what we consider the gold standard approach.

The role of a specialty pharmacy partner

A good specialty pharmacy partner plays a crucial role in connecting patients with gene therapies while supplying comprehensive clinical and non-clinical supportive services. Manufacturers depend on the expertise of a specialty pharmacy to navigate the complexities of the market and establish successful connections with payers, prescribers, and patients.

Accredo is an industry leader in specialty pharmacy partnerships, particularly for exclusive distribution, limited distribution, and rare and gene therapy products. With over 50% of Accredo’s exclusive therapies dedicated to rare diseases and fourteen programs in the limited specialty pharmacy network space, it is clear we are committed to advancing care in the most challenging therapeutic areas.

My Care ConnectSM: A service model tailored to your therapy’s needs.

Accredo goes beyond the role of a typical specialty pharmacy to support gene therapy manufacturers to help ensure optimal patient access. To achieve this, Accredo created My Care Connect, a customizable service model built on these key principles:

Rare Disease Pharmacy Center of Excellence

Our Memphis-based Rare Disease Therapeutic Resource Center® (TRC) operates as a URAC-certified Rare Disease Pharmacy Center of Excellence for patients across the U.S and U.S. territories. It leverages the resources of our larger parent organization, while a small core team maintains a hyper-focused approach to rare or gene therapies. This certification was developed by URAC in collaboration with rare disease experts including the National Organization for Rare Disorders (NORD)to affirm an accredited URAC Specialty Pharmacy has quality programs that address the unique challenges experienced by patients with a rare disease.

Patient Access

Accredo collaborates with manufacturers to create programs that maximize patient access to therapy, ensuring best clinical outcomes for patients with the rarest of conditions. Recognizing the unique requirements of these patients and therapies, we provide expertise in benefits investigation, prior authorization, and connections with copay assistance programs and foundations. The goal is to simplify the journey for patients and caregivers by providing unimpeded access to necessary therapies and care.

Accredo created a team solely dedicated to patient access within the gene therapy space. This team is called GeneAXS™ (pronounced “gene access”), and it involves not only the clinical ability housed within our Rare TRC, but also a resolute team focused on non-clinical aspects. The team encompasses payer contracting, clearance, fulfillment, patient services, and clinical consultation, collectively forming a comprehensive and forward-thinking model for navigating the complexities of gene therapies.

Storage and distribution management

Complex storage and distribution management is a core function that cannot be emphasized more when managing the fulfillment and distribution of rare, ultra-rare or gene therapies. Accredo demands a highly systematic and coordinated approach from its logistics teams using a proprietary IceGen algorithm and cold chain technology by reviewing weather patterns from the point of dispense to the point of delivery – ensuring the product arrives as required. Our proven expertise is the linchpin in ensuring therapy success and optimal patient outcomes.

Data insights and performance

Accredo’s Pharma Portal offers a unique platform to the industry, offering customizable data insights and real-time performance tracking – all vital for a rare, an ultra-rare or a gene therapy’s success. For manufacturers to have this capability offered by your specialty pharmacy partner translates into a more agile and informed strategy, optimizing the overall performance of therapies in the market.

A collaborative partnership equals success.

With Accredo as your specialty pharmacy partner, you benefit from dedicated program management and the Rare Disease TRC Center of Excellence teams. This collaboration, paired with My Care Connect, will help ensure in-market success for your gene therapy products and the well-being of patients.

Together we can co-create a service model that works for you and harnesses innovation of gene therapies. Watch the video below to learn more.

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