
- Pharmaceutical Executive-08-01-2019
- Volume 39
- Issue 8
Using FDA’s ‘PIE’ Recipe to Enhance Patient Access
How R&D organizations can leverage FDA’s final guidance on pre-approval information exchange (PIE) to engage with payers prior to approval and launch.
Market dynamics are changing rapidly. Cost pressures are driving pharma manufacturers to generate evidence that demonstrates product value. While we’re seeing
To that end, in the age of value and affordability, pharma R&D arms can leverage FDA’s final
The final guidance addresses scientific and health economic information exchange (HCEI) between manufacturers and payers regarding their unapproved products and unapproved uses of approved products (i.e., PIE). This information could help payers plan for, budget for, and ultimately drive future coverage or reimbursement decisions on a population health basis prior to FDA approval. And drugmakers have an opportunity to enhance their evidence generation strategy through increased dialogue with payers.
Achieving these benefits, however, isn’t as easy as pie. Risk-averse payers and pharma companies remain uncertain about compliance and the
Instead of nibbling around the edges of PIE, R&D organizations can embrace FDA’s final guidance by working hand in hand with payers earlier in the development process (ideally prior to a drug’s Phase II clinical trials), with the overarching goal of favorable coverage and reimbursement decisions. There are three critical steps to achieving this:
1. Assemble the right team to engage. Payers are a sophisticated market force, and with industry consolidation on the rise, they possess stronger controls on patient access than ever before. Early interactions prior to product approval are high-stakes and high-reward, with the potential to reap downstream benefits for patients. The right mix of expertise from development, medical and commercial organizations can drive powerful, data-driven dialogue to ensure that payers make informed formulary decisions and nurture policies that allow patients access to high-value medicines. The sticker shock that followed the most recently approved gene therapies has underscored the need for better value-based models to finance innovative medicines.
2. Leverage clinical trial and value frameworks to incorporate payer feedback. The disparity between regulatory approval and successful reimbursement is widening as the R&D and value and access organizations are unable to integrate payer needs into Phase II-III trial design. As a result, these organizations pass the baton to medical affairs, health economics, and outcomes research groups, to design Phase IIIb-IV, investigator-initiated trials (IITs), and RWE strategies, including patient registries. The risk-adjusted net present value can be leveraged to find the appropriate balance of trial designs that go beyond achieving regulatory milestones alone.
3. Lay the foundation for RWE partnerships. FDA has embarked on the journey to include the use of RWE as part of the regulatory paradigm. The agency leverages advancements in analytics as well as the explosion of disparate data sets that span electronic health records, laboratory tests, wearable devices, and insurance claims data via its new RWE Framework. At the same time, value-based partnerships, such as risk-sharing agreements and data-sharing partnerships, are on the rise but fail to demonstrate tangible results to policymakers. By engaging earlier with payer organizations, pharma manufacturers can set the foundation for a streamlined negotiation and implementation process. They can also explore the use of novel clinical endpoints and integrate data sets earlier in the development process.
Dean Hakanson is a Principal in the R&D Excellence practice; Jon Gonzales is a Manager in the Medical Affairs practice; and Tara Alire is a â¨Strategy Insights & Planning Associate Consultant, all at
Articles in this issue
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A Look Back & Forward on Innovationabout 6 years ago
Routes to Exclusivity in Cell-Based Therapy Developmentabout 6 years ago
Gene Therapy: Managing the Clinical Trial Minefieldsabout 6 years ago
The Call to Cure: Geoff MacKay, AVROBIOabout 6 years ago
Four Takeaways from the Zolgensma Pricing Stormabout 6 years ago
FDA Faces ‘Explosion’ in Growth of Gene Therapiesabout 6 years ago
Mapping Out the Future of Cell and Gene Therapyabout 6 years ago
High-Stakes Manufacturing: Mitigating the Risksabout 6 years ago
Gene Therapy: How ‘Advanced’ is Europe?about 6 years ago
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