What you need to know about pre-approval information exchange: lessons learned

Written by Nicole Lodowski (Senior Director and US Practice Lead at PRMA Consulting) and Nathan White (US Head of Client Partnerships at Fishawack Health Policy, Access, Value, and Evidence)

• Although the field of health economics has evolved rapidly over the past several decades, manufacturers historically have been limited in what information they were permitted to share with payers and similar entities prior to FDA approval of a drug.
• Recent legislation and FDA guidance has created useful communication pathways and safe harbor for manufacturers to share much-needed preapproval product information with payers.
• Payers now have the ability to gain valuable insights about new products and therapy areas well in advance of FDA approval and make earlier and more informed coverage and reimbursement decisions.

History and context of HCEI

In 2016, FDAMA first defined healthcare economic information (HCEI) in an amendment of the FD&C Act (21 USC 352[s] section 502[a]), the 2016 enactment of the 21st Century Cures Act and subsequent guidance released by FDA further defined HCEI, who can use it and in what context, and how to ensure that HCEI communications are truthful and non-misleading. The guidance also addressed common questions related to dissemination to payers of information about medical products that are not yet approved, also known as pre-approval information exchange (PIE).

HCEI is defined as “any analysis that identifies, measures, or describes the economic consequences, including clinical data, inputs, clinical or other assumptions, methods, results, and other components underlying or comprising the analysis .” HCEI may be based on the separate or aggregated clinical consequences of the represented health outcomes. The analysis may be comparative to use of another drug, another healthcare intervention, or no intervention at all. The FDA guidance adds that HCEI can be presented in an evidence dossier, publication reprint, budget impact model, modeling software, slide presentation, or payer brochure.

The guidance also stipulates recipients of HCEI should have significant knowledge and expertise in healthcare economic analysis – this includes payers, formulary committees, drug information centers, technology assessment panels, pharmacy benefit administrators, and other multidisciplinary entities. Healthcare practitioners and consumers are not considered to be covered entities in this guidance, so it’s important for manufacturers to ensure that the appropriate audience is receiving the HCEI. 

HCEI analyses should relate to the disease/condition, manifestation of the disease/condition, or symptoms associated with the disease/condition. They should also be based on competent and reliable scientific evidence, based on good research practices developed by authoritative bodies (e.g., the International Society for Pharmacoeconomic and Outcomes Research [ISPOR] or the Patient-Centered Outcomes Research Institute [PCORI]). To provide a balanced and complete presentation, HCEI analyses should include components such as study design, methodology, generalizability, limitations, and sensitivity analysis.

Recent developments in PIE

FDA guidance released in 2018 (Drug and Device Manufacturer Communications With Payors, Formulary Committees, and Similar Entities / Questions and Answers / Guidance for Industry and Review Staff) allows for discussion of evidence – including clinical trials – prior to product and/or indication approval within the boundaries of a communication about HCEI to payers and healthcare decision-makers (HCDM). Examples of allowed information includes:

• product information
• information about the indication being researched
• factual presentations of clinical studies and trials  (including in-progress studies)
• anticipated development timelines
• product pricing information
• information to help payers and HCDMs estimate eligible member population
• patient utilization research
• product-related programs and services.

For all of the above, it’s important that the information presented is factual and non-promotional – in other words, not including product claims or value messages.

However, because of subsequent lack of clarity in the 2018 FDA guidance, several legislative initiatives are underway to codify the safe harbor for proactive PIE communications. The Pre-Approval Information Exchange Act (HR 7008) was introduced on March 9, 2022 by Rep. Brett Guthrie (R-KY-02) and, as of July 2022, is still in committee. Upon receiving and incorporating technical assistance from the FDA, the PIE Act of 2022 was included in HR 7667, which passed the full House of Representatives on June 8, 2022.

Key considerations for manufacturers

PIE is extremely important to payers and HCDM. Most recipients of HCEI via a PIE discussion want to see this information up to 12 months prior to FDA approval to help guide early decision-making around formulary and benefit design, as well as budget impact modeling.

Pre-approval HCEI can be communicated through a variety of formats, such as in-person meetings, individual web-based meetings, AMCP pre-approval dossiers, medical information requests, closed payer/HCDM platforms, email exchanges, and webinars. Recipients can also ask questions, creating a bi-directional conversation; this can be extremely useful to manufacturers and can lead to deeper understanding of payer evidence requirements prior to launch.

Because FDA does not consider PIE promotional and does not specify who can present PIE to HCDMs, cross-functional manufacturer teams (e.g., medical science liaisons, health outcomes liaisons, national account directors) can work together to construct a PIE communication that meets HCDM needs (keeping in mind that data in PIE communications needs to align with FDA guidance on what is appropriate to share).

That said, the team assembled by the manufacturer must be educated on the studies and be able to communicate the information scientifically, rather than promotionally. The manufacturer team involved in the PIE engagement typically comprises multiple stakeholders, including scientific and pricing experts. This is not a requirement, as well-trained individuals can likely speak to all aspects, but often enables a more robust discussion overall.  

Pre-approval dossiers can be disseminated to payers and HCDMs prior to launch proactively, and while these dossiers have some restrictions and are not generally as robust or lengthy as post-approval dossiers, they can be used as a foundation for a post-approval dossier. Manufacturers are permitted to discuss in these dossiers and presentations, pricing information or estimated pricing (included in safe harbor), which can be very useful to HCDM who are attempting to predict actuarial impact on drug budgets. This can also be useful to manufacturers who want to have early contract discussions with HCDMs and payers.

Building a PIE strategy in 3 steps

Step 1: First ask the right questions. As building a PIE communication is a lot of work, can involve multiple consultants and vendors, and is an investment, it’s imperative for manufacturers to understand:

• Is PIE the best approach? Can market research solve the issue or question?
• Would a PIE bring value to payers?
• Is this a new MOA, a new class, or a rare therapeutic area that payers may need to better understand?
• Is there a potential for a large budget impact that payers and HCDMs should be aware of prior to launch for planning purposes?
• What information do payers want to see in a PIE?
• How do we create value in the PIE interaction with payers and not overload with too much data or too many touch points?
• What accounts are best suited to receive the PIE?

Step 2: Figure out the resources needed to support the PIE activity. Whether it’s creating a segmented target list of accounts, internal documents that provide context and legal guidance, FAQs, outreach tools such as emails and trackers, and trainings, all support resources for your PIE initiative should be carefully vetted and included in your tactical plan.

Step 3. Create and deliver a tailored PIE communication. While payers may be your primary audience, other HCDMs, such as integrated delivery networks, group purchasing organizations, healthcare policymaking organizations, and healthcare ecosystem organizations, ma y have an interest in your PIE data. It’s important to tailor your medical and commercial content to the specific needs and interests of your audience; this can include topics such as commercial models, market and disease landscape, pricing strategy, launch timing, cost burden studies, and pivotal trials. Where possible, it might also be beneficial to include information specific to the member or patient population relevant the payer or HCDM.

Typically, a PIE presentation is between 45 and 60 minutes in length, so it’s important to be highly specific about what you want to communicate and seek to present the material in a simple and compelling manner. Also make sure to include a list of discussion questions, as this is a prime opportunity to gather feedback about your data or model; prioritize your questions by order of importance, and weave them through your presentation.

Ultimately, gauge the interest level of your audience throughout the presentation and hone in on topics of high importance – these will be the questions and feedback that will have the most impact on your follow-up/through strategies and help to guide development of your post-approval medical and commercial communications plans.

Real-world example: a PIE program with US payers to support preliminary access for a new immunology product

Client situation

Our client was launching a new immunology product with a new MOA in a crowded US market. In order to support payers in their upcoming formulary planning and to better understand payer dynamics within the disease area, there was an identified need to develop a pre-approval communication strategy for US payers.

Our approach

Review of evidence

To begin, we reviewed available client evidence to determine what data was available – and where evidence gaps would need to be addressed through a targeted literature review and/or generation of additional data.

Strategic planning workshop

Following our review of available data, we held a planning workshop with a cross-functional client team to determine the goals of the pre-approval communications as well as key messages that were important to include in these scientific communications. Within this workshop, we also brainstormed materials that may support these communications, and generated a strategy for communications, including a target account list.

Development of PIE deck

Our team next generated a detailed outline of key components within the presentation, which was ratified through cross-functional team. Once aligned, we developed the scientific PIE deck, and supported internal client approvals to begin scheduling PIE meetings. Key topics included the market landscape (disease burden, competitive landscape), product launch timing, clinical evidence, and pricing information.

Additional deliverables

In addition to the PIE deck, our team also helped with the generation of other ancillary PIE support materials, including:

• pre-approved discussion questions and responses to support presenter preparation
• training presentation to support account directors and anyone else who would be involved in PIE presentations
• approved outreach emails to support communications.

Client value

These PIE materials were used in over 30 payer regional and national accounts across the US to support pre-approval account meetings. The presentations provided clarity to our client around potential access and clinical uptake barriers and allowed time prior to launch to address some of these concerns. Feedback provided also demonstrated that some payer accounts were extremely appreciative of the proactive communication, which allowed them ample time to prepare for entry of an additional high-cost product in a crowded market that would likely have preferred status due to clinical benefit. Overall, this PIE initiative was regarded as highly successful within our client’s organization and became a gold standard for high-visibility or high-cost products moving forward within the organization.

Conclusion

As payers’ evidence needs continue to evolve and formulary and budgetary decision-making becomes more complex, manufacturers have a prime opportunity to create added value by engaging with payer customers earlier and more often through PIE programs.

With a safe harbor now in place, manufacturers can feel confident their product information can be shared with payers without significant legal and compliance risks. It’s a win/win scenario for both sides: payers have the information they need to accurately predict budget impact and plan for formulary changes, and manufacturers have earlier opportunities for education and communications touchpoints to gain valuable insights and continue building relationships with key customers.