On average it can take over a decade to discover, develop and get regulatory approval for prescription medicines in the United States. This time frame involves discovery, pre-clinical, safety assessment, clinical development and manufacturing. Along the way, there are regulatory insights into all phases of development as the product moves along the development and approval process. Regulatory input influences and changes the development by shaping the indication, patient population, the endpoints and ultimately the approved label. So regulatory input into the development of a medicine is essential for approval.
Once medicines are approved for sale in the US, manufacturers move to the next obstacle, reimbursement or formulary placement. It can take up to a year or longer for a new product to be accessible to a patient at a reasonable co-pay. Sometimes products are not included on formulary at all due to lack of appropriate evidence.
Lack of appropriate evidence at time of launch may be due to, unlike regulatory authorities, payers not being part of the development process. Early input into the development of the medicine is usually not designed to address specific payer needs or development of evidence that would improve the likelihood of optimal formulary placement so that patients in need can easily afford and have access to new innovative medicines.
It is important to note that approximately
of payers from a recent payer panel wanted to see pre-approval information 12 months prior to NDA submission and
wanted information at 12-18 months prior to approval.
of the payer participates in this same panel agreed that prior approval evidence provided data that enabled value-based decision making.1
Early payer input into drug development can be obtained by biopharmaceutical companies in several ways. Considering the development process may take a decade or longer it is imperative to get timely inputs along the way so that the development process can be optimized for reimbursement. For example, prior to end of phase II, the product profile and future competitive environment can be presented to a blinded payer panel to get valuable input into the phase III evidence plan. The payer panel should contain the relevant payers that the product will ultimately be contracted, such as specialty pharmacies for specialty products or Medicare plans for products that are mainly used by Medicare eligible patients.
Another check point with payers could occur at end of phase III so payer evidence gaps can be assessed and recommendations can be incorporated into a phase IIIb plan. In addition, as the pre-approval evidence presentation are being developed, these can be tested with payers in an unblinded manner so the impact of the data, and how the data are being presented can be critiqued. Changes to the presentation can be made prior to external roll out. Unblinding of the presentation to the payer panel can deliver a more in-depth assessment of the payer evidence.
Whatever path is chosen, manufacturers should be concise and strategic with any presentation to payers, keeping in mind not to overwhelm them with data. In addition, the aspirational value framework should be tested along with the evidence. For these presentations to be impactful, and to get access to the appropriate payers, manufacturers may want to so use external groups that have expertise not only in assessing evidence plans and value frameworks but also have relationships with appropriate payers that they can tap into to critique evidence plans and payer communications.
Innovative medicines that improve the health of patients can only work if patients have access to these medicines. Early payer inputs into pharmaceutical development plans can improve the likelihood of optimal formulary access and patient affordability.
1. Brixner D et al. J Manag Care Spec Pharm. 2019 Feb;25(2):164-173.
Please feel free to share your comments below on your experiences of successes, challenges, or questions in abilities to share HCEI in preapproval information exchange with payers.
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