PIE is Good for You: Drive Patient Access by Embracing FDA's Final Guidance


R&D organizations can leverage FDA’s final guidance on pre-approval information exchange, otherwise known as PIE, to engage with payers prior to regulatory approval and commercial launch.

Market dynamics are changing rapidly. Cost pressures are driving pharmaceutical manufacturers to generate evidence that demonstrates product value. While we’re seeing more drugs approved than ever before, R&D organizations continue to struggle with designing evidence generation strategies that improve patient access to medications. In the age of value and affordability, R&D organizations can leverage FDA’s final guidance on pre-approval information exchange, otherwise known as PIE, to engage with payers prior to regulatory approval and commercial launch. PIE can be a useful tool; however, payers and pharma companies have been slow to fully embrace it. Organizations can leverage PIE to drive patient access by assembling the right team, incorporating payer feedback and building a solid Real World Evidence (RWE) foundation.

The final guidance addresses scientific and health economic information exchange between pharmaceutical manufacturers and payers (which includes formulary committees and similar entities) 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. Pharmaceutical manufacturers, on the other hand, 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 lack of legislative safe harbor rules in the wake of increased  scrutiny by policymakers. The Academy of Managed Care Pharmacy (AMCP) made PIE a focal point during their 2019 Annual Meeting after making severalefforts to encourage bidirectional communication between manufacturers and payers. While many pharmaceutical manufacturers have collaborated with the AMCP to drive these efforts, pharma has made few strides in driving proactive scientific and economic discourse since PIE was issued in June 2018.

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 2 clinical trials) with the overarching goal of favorable coverage and reimbursement decisions. There are three critical steps to achieving this:

• 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 (Novartis’ Zolgensma at $2.125 million and Bluebird Bio’s Zynteglo at $1.8 million) has underscored the need for better value-based models to finance innovative medicines.

• 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 2-3 trial design.  As a result, these organizations pass the baton to medical affairs, health economics and outcomes research organizations, to design phase 3b-4, 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. Pharmaceutical manufacturers can incorporate payer feedback (e.g. the inclusion of a new secondary endpoint) in early clinical trial design, but this will require a careful consideration of patient burden, costs and complexity as well as foresight into the value of the data generated after the product is approved.

• Lay the foundation for RWE partnerships. FDA has embarked on the journey to include the use of RWE as part of the regulatory paradigm. FDA 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, pharmaceutical 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. The onus remains on the industry to ensure that we continue making strides toward data harmonization and standardization. In turn, Real World Data (RWD) can be fully harnessed to sustain partnerships between pharmaceutical manufacturers and payers.

At the current pace of change, organizations face an imperative to improve patient access. It’s understandable that the current regulatory environment might leave some nervous about adopting a framework like PIE; however, with the right strategy and approach, embracing FDA’s final guidance is a key to adapting to the age of value and affordability.

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 
Strategy Insights & Planning Associate Consultant, all at ZS.

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