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Insights from AMCP Nexus: Navigating pre-approval information engagement in oncology and rare diseases

At AMCP Nexus 2024, Tasmina Hydery discussed her research on optimizing pre-approval information exchange (PIE) for oncology products, products for rare diseases, and cell and gene therapies. The study revealed that the majority of healthcare decision-makers believe PIE significantly impacts patient access, preferring information three to six months before approval. One of the most interesting findings was that the type of pre-approval information most valued by HCDMs was the specific product indication being sought. Future research will further explore market trends and improve PIE engagement to enhance patient access.

5 questions with Tasmina Hydery

At AMCP Nexus 2024, Cencora team members presented research posters on a variety of topics across the managed care space. We took the opportunity to chat with them about their work and its potential impact. Here, Tasmina Hydery, PharmD, MBA, BCGP, Associate Director, Scientific for Cencora answers questions about the poster, “Optimizing the timing of pre-approval information exchange engagement for oncology products, products for rare diseases, and cell and gene therapies.” Ben Penley, PharmD, MS, and Charles M. Dragovich, BSPharm, served as co-authors.

Download the poster

What inspired this research?

Our consulting teams have been researching the demand and needs for pre-approval information exchange (PIE) among healthcare decision-makers (HCDMs) for the last five to six years.

In 2018, the FDA issued guidance for biopharma companies on sharing information with HCDMs about products that aren't yet approved. Still, at that time, biopharma companies were reluctant to share information on their pre-approval products because there wasn't formal legislation.

In December 2022, legislation was codified that put into effect a safe harbor for biopharma companies that are interested in engaging HCDMs with PIE. Looking at the robust drug pipeline, we realized that some of the different therapeutic areas and product classes might be of greater interest to HCDMs in the pre-approval period.

There are several reasons for this interest. The products might be more costly. The administration of the medication itself might be complex. It might be an infused medication or something that's administered directly to a site to modify the gene. It might be for a disease state that an HCDM is less familiar with. It might be a rare disease, or it could be a protected class of medications like oncology.

We wanted to conduct research to see what type of information an HCDM is looking for and the time frame in which they need that information. That set the stage for us to see what HCDMs’ preferences are for certain product categories.

This research started in the spring of 2024. We fielded a survey to the Cencora Managed Care network, a panel of healthcare executives including pharmacy directors and medical directors. The 41 HCDMs who responded collectively are responsible for an estimated 63 million covered lives.

What are the key takeaways from your research?

One of the main takeaways is that about 50 percent of the individuals who responded to the survey stated that PIE impacts the timing of patient access to certain product categories. This was especially the case for the three product categories in the title of the survey—oncology products; products for rare disease; and cell and gene therapies (CGT).

We also asked if there is a time when pre-approval information exchange is too late. Regardless of product category, about half of the HCDMs surveyed said that the latest that a biopharma company should share PIE is up to 6 or 3 months before anticipated approval. Biopharma companies shouldn’t wait until a couple of weeks before their product is approved. They need to create a plan of how they're going to engage in PIE well in advance.

Was there anything in the research that was surprising, that you didn't expect?

If biopharma companies are sharing pre-approval information a year or more out from approval, HCDMs most often want to know the specific indications the product is seeking and the anticipated approval timeline. Those are some of the basic pieces of information a biopharma company can provide when the product is further out from approval.

Other items, pricing or clinical trials results, are necessary for a deep-dive analysis, which may come later.

Is there any way that the FormularyDecisions platform can have impact with providing information to healthcare decision makers?

FormularyDecisions is a vehicle for biopharma companies to provide information on pre-approval products. They can share information via dossiers and PIE webinars, which we host exclusively on the platform in collaboration with AMCP. Biopharma companies can partake in a subscription where they have a Resource Center to post information on their pre-approval products. This can be approval timelines, clinical trial results and study information, and disease state awareness pieces. It would depend on the type of product that a biopharma company is bringing to the market to determine which information is best suited to post in a Resource Center.

But certainly, there are multiple vehicles that they can use on FormularyDecisions to share information before approval. The bidirectional flow of information through our digital platform is how we facilitate filling information gaps for HCDMs.

What are the next steps from this research?

We want to continue doing this research and look at topics year after year to see what changes there are in market trends and market perceptions. PIE is one of those things that we will continue evaluating so we can better understand what HCDMs’ perceptions are and understand their preferences for engaging in PIE.

We also want to continue identifying what the impact is on patient access. Ideally, if HCDMs are getting information earlier, they're better suited to make decisions so patients can access the medication more quickly. We're trying to underscore the value of PIE engagement and reduce the gap in what HCDMs are looking for and what's actually available from the biopharma companies.