Advancing Rare Disease Therapies: The Role of Healthcare Systems and Industry Partnerships with Insights from David Mitchell, PharmD, MBA

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How Health Systems and Pharmaceutical Manufacturers Are Building Trust and Improving Access to Rare Disease Therapies

David Mitchell, PharmD, MBA, senior pharmacist manager and assistant clinical professor, UC Davis, discusses how health systems and pharmaceutical manufacturers are working together earlier and more proactively to bring innovative rare disease therapies to patients in an effort to build trust, improve access to medications, and ensure better outcomes for those with rare diseases.

american journal of Managed Care ( AJMC ): How has the relationship between industry and health systems changed over time?

Mitchell: The relationship has shifted from being very transactional to a true strategic partnership. We’re seeing the evolution of a collaborative effort focused on the patient, working to bring these innovative therapies to market, ensure access for appropriate patients, and achieve the best possible outcomes.

It’s really about working together, anticipating challenges, and strategically developing solutions.

AJMC: What is driving this change?

Mitchell: Several factors are at play. Ther’s a deeper understanding now that working collaboratively is the moast effective path to reach patients with rare diseases.The complexity of these therapies-the unique handling requirements, the need for specialty pharmacies, and the ofen limited number of treatment centers-necessitates a concerted effort from all stakeholders.

Historically, pharmaceutical manufacturers would launch a product and then address hurdles related to access, reimbursement, and patient identification. Now, we’re seeing increased engagement much earlier in the development cycle. Health systems are now involved in conversations about clinical trial design, evidence generation, and market access strategies before a drug is even submitted for approval.

AJMC: What are some of the specific ways health systems and manufacturers are collaborating?

Mitchell: We’re seeing several innovative approaches. one example is joint education programs for clinicians. Rare diseases are, by definition, rare. Many physicians may have limited experience diagnosing and treating these conditions. Manufacturers and health systems are partnering to provide comprehensive training on disease awareness, appropriate patient selection, and safe medication governance.

another area is the development of proactive identification strategies.Manufacturers can provide data and analytics to help health systems identify potential patients who may benefit from a therapy. This is particularly crucial in rare diseases where diagnosis is often delayed or missed.

We’re also seeing collaboration in the development of patient support programs and financial assistance resources. These programs can help patients navigate the complexities of insurance coverage, manage treatment costs, and adhere to their therapy.

AJMC: What role does trust play in these partnerships, and how is it fostered?

Mitchell: Trust is absolutely paramount. Building trust requires transparency, open communication, and a shared commitment to patient well-being. Health systems need to feel confident that manufacturers are acting in the best interests of patients, not just their bottom line.

Manufacturers, in turn, need to trust that health systems will use these therapies appropriately and ethically. This means adhering to established clinical guidelines, monitoring patient outcomes, and reporting any adverse events.

To foster trust, we’re emphasizing more frequent and candid dialogue. It’s not just about formal meetings; it’s about ongoing communication and problem-solving.

AJMC: What are the benefits of this more collaborative approach?

Mitchell: The benefits are significant. First and foremost, patients gain access to perhaps life-changing therapies faster and more efficiently. Second, it leads to better clinical outcomes. When clinicians are well-trained and patients have the support they need, they are more likely to adhere to their treatment plan and experience positive results.

Third, it helps to reduce healthcare costs by preventing unneeded hospitalizations and emergency room visits. it strengthens the overall healthcare system by fostering innovation and collaboration.

How can healthcare systems and pharmaceutical companies collaborate to ensure value-based pricing for rare disease therapies, considering the severity of the condition and patient impact?

The Challenge of Rare Disease Drug Progress

Developing therapies for rare diseases – frequently enough defined as conditions affecting fewer than 200,000 people in the United States – presents unique hurdles. These include limited patient populations, making clinical trials challenging and expensive, a lack of natural history data, and the complex regulatory pathways involved. The economic realities also play a notable role; the orphan drug market, while growing, often doesn’t offer the same return on investment as treatments for more prevalent conditions. This necessitates innovative approaches involving collaboration between healthcare systems, pharmaceutical companies, and patient advocacy groups.

The Critical Role of Healthcare systems

Healthcare systems are pivotal in accelerating rare disease diagnosis and treatment access. Their contributions span several key areas:

* Enhanced Diagnostic Capabilities: Investing in genomic sequencing, specialized diagnostic centers, and training for clinicians to recognize subtle symptoms are crucial.Early and accurate diagnosis significantly improves patient outcomes.

* Real-World Evidence (RWE) generation: Healthcare systems possess vast amounts of patient data. Leveraging this data to generate RWE can supplement clinical trial findings, providing valuable insights into treatment effectiveness and safety in diverse populations. This is particularly vital for rare genetic diseases where traditional trials are challenging.

* Streamlined Access to Therapies: Navigating insurance coverage and reimbursement for expensive orphan drugs can be a major barrier for patients. Healthcare systems can advocate for favorable coverage policies and establish patient assistance programs.

* Center of Excellence Networks: Creating specialized centers focused on specific rare diseases allows for concentrated expertise, improved care coordination, and the development of best practices.

Industry Partnerships: A Collaborative Imperative

Pharmaceutical and biotechnology companies are increasingly recognizing the need for collaborative partnerships to overcome the challenges of rare disease drug discovery. These partnerships take various forms:

* Joint Research Ventures: Combining the research expertise of pharmaceutical companies with the academic rigor of research institutions can accelerate the identification of potential drug targets and the development of novel therapies.

* Data Sharing Initiatives: Securely sharing anonymized patient data between industry and healthcare systems can facilitate research and improve understanding of disease progression.

* Patient Advocacy Group Collaboration: Organizations like the Patient Access Network (PAN) Foundation, and those championed by david Mitchell, pharmd, MBA, play a vital role in representing patient needs, providing funding for treatment, and advocating for policy changes. Mitchell’s work emphasizes the importance of patient-centricity in drug development and access.

* Innovative Funding Models: Exploring choice funding mechanisms, such as venture philanthropy and public-private partnerships, can help de-risk rare disease research and attract investment.

David Mitchell’s Perspective: Patient-focused Drug Access

David Mitchell, founder of Patients For Affordable Drugs, consistently advocates for policies that lower drug prices and improve patient access to essential medications, including those for rare conditions. His insights highlight the following:

* The Importance of Value-Based Pricing: Pricing should reflect the clinical value a drug provides, considering the severity of the disease and the impact on patients’ lives.

* Transparency in Drug Pricing: Greater transparency in the pharmaceutical supply chain is needed to understand the factors driving drug costs.

* Addressing Patent Thickets: Strategies to prevent companies from extending monopolies through incremental patent modifications are crucial to fostering competition and lowering prices.

* Patient Assistance Programs: While helpful, these programs are not a sustainable solution and should not be used as a substitute for affordable drug prices.

Case Study: Spinal Muscular atrophy (SMA) Treatment access

The development and subsequent access challenges surrounding gene therapies for Spinal Muscular Atrophy (SMA) exemplify the complexities of rare disease treatment. Zolgensma, a groundbreaking gene therapy, offers a potential cure for SMA, but its extremely high price tag initially limited access. Collaboration between pharmaceutical company Novartis,healthcare systems,and patient advocacy groups,including those actively supported by mitchell’s advocacy,was essential in negotiating payment plans and securing insurance coverage for eligible patients. This case demonstrates the power of collective action in overcoming financial barriers to life-changing therapies.

Navigating Regulatory Hurdles for Rare Disease Therapies

The FDA’s Orphan Drug Act of 1983 has been instrumental in incentivizing the development of drugs for rare diseases. However, navigating the regulatory process still presents challenges.

* Accelerated Approval Pathways: Utilizing accelerated approval pathways, based on surrogate endpoints