Payers Want Access To US FDA's Pre-Submission Meetings
Executive Summary
As sponsors push for more access to physicians, payers hope to be able to gather more pre-approval information about drugs – like they now can with devices – for budgeting and patient access.
Payers want to attend FDA pre-submission meetings with drug sponsors to help better gauge pricing and utilization post-approval. The change, which the agency's device center recently adopted, could be one way for FDA to deal with some industry concerns about off-label communications.
Humana Inc. VP-Federal Affairs Douglas Stoss and others asked during FDA’s two-day public hearing on off-label communications that payers be allowed more freedom to talk to drug developers about their pipelines in order to better predict premiums and spending.
Stoss said Nov. 9 that with "access to a more complete picture of a drug's current and future utilization profile and clinical effectiveness data, we can make better informed decisions about plan design, formulary placement and coverage criteria."
"FDA's Center for Devices and Radiological Health has recently taken a step in this direction by allowing device manufacturers to invite payers and technology assessors to their pre-submission meetings to provide feedback on evidence generation and clinical trial design," he said. "Similar interaction between the agency, manufacturers and payers on the drug and therapeutic side, should be considered."
More Coverage Of FDA Off-Label Communications Hearing
FDA prohibitions on off-label communications often hinder sponsor conversations with payers about new drugs before they are approved. The public hearing could be a precursor for some loosening of the rules. But a number of questions and concerns about more off-label communication were raised throughout the event. (Also see "Off-Label Communication Growth: Could FDA Afford To Police It?" - Pink Sheet, 10 Nov, 2016.)
FDA seemed interested in the idea of payers attending pre-submission meetings. Rachel Sherman, deputy commissioner for medical products and tobacco, asked Stoss when in the development process payers would like to see data. "Perhaps about at the time of submission? How early might you want to go?" she asked him.
Stoss said he would elaborate more on that idea, as well as what particular types of information that could be provided by manufacturers at that time, in written comments.
Is Device Program Adaptable To Drugs?
The CDRH program allowing payers to attend pre-submission meetings launched earlier this year, but has seen some problems. It stems from the center's FDA-CMS parallel review program. (Also see "FDA Plans For Private-Payer Meetings Ahead, But Roadblocks Remain" - Medtech Insight, 13 Jun, 2016.)
The program is intended to bridge the divide between device clearance and reimbursement. (Also see "Bringing Private Payers To The Regulatory Table: FDA Reaches Out To Insurers" - Medtech Insight, 23 Feb, 2016.)
A number of drug sponsors conduct pre-NDA meetings with the agency to familiarize the review team with the contents of the application, especially if it will include unconventional items.
At that point, clinical trial and other work needed for approval would be completed and efficacy and safety data available. Payers may find that information helpful. Their earlier involvement also could help sponsors avoid problems where data does not support reimbursement.
Industry has argued that in the wake of legal challenges, FDA should loosen its off-label communication policy. Allowing sponsors and payers to speak prior to a drug approval was raised as one of the easy adjustments that could be made. (Also see "Industry's Off-Label Hearing Priorities Taking Shape" - Pink Sheet, 30 Sep, 2016.)
Congress already has allowed more of those discussions, but a lack of FDA guidance has left many sponsors unwilling to engage. (Also see "FDA's Off-Label Communication Changes Should Start With Payers – PhRMA" - Pink Sheet, 20 Sep, 2016.)
The reforms hold promise and peril for all sides. For sponsors, the prospect of payers sitting in on their meetings with FDA is less than ideal; they’d much rather prepare their own presentations to update payers on the progress of pipeline candidates. For payers, the idea that sponsors could be talking to physicians about prescribing products off-label means that prescription drug spending might become harder to project.
Payers Need Information On Breakthrough Therapies
Allowing more payer access during drug development could improve launch planning for therapies FDA is expecting to be transformative.
Samuel Nussbaum, formerly Anthem Inc. chief medical officer and now with the USC Schaeffer Center for Health Policy and Economics, said expedited pathways like the breakthrough therapies program are pushing drugs out faster, which is affecting payer planning.
"With the introduction of FDA breakthrough therapies designations, significant drugs are moving more efficiently through the development pipeline [and] through the review and approval process, and that poses timing challenges for both plans and patient access," Nussbaum said.
Nussbaum also said the lack of information for payers can affect patient access to drugs approved through breakthrough and accelerated approval.
"As a result of breakthrough therapy and accelerated approval processes, the most promising new medications may be vulnerable to timing challenges, which could implicate patient accessibility," he said.
Breakthrough, which allows increased FDA involvement for therapies judged to potentially be substantial advances from existing care in the hopes of getting them to market faster, has been much more popular than expected when created in 2012. In the upcoming prescription drug user fee reauthorization, industry and FDA agreed to devote user fees to hiring more reviewers so the agency can better handle the breakthrough workload. (Also see "FDA's Breakthrough Workload Will Be Eased By Hiring Reviewers With PDUFA VI Funds" - Pink Sheet, 20 Jul, 2016.)
Off-Label Communication Could Affect Premiums
For payers, the increased communication could substantially impact budget formation and potentially premiums.
Stoss said estimating use without more data is extremely difficult. He said Humana's estimates for spending on the PD-1 drug class in its Medicare Advantage plans next year varies by $100m, about 160%, depending on which new indications are approved next year.
"We expect additional approved indications for PD-1 drugs in 2017 based on information obtained from clinicaltrials.gov, conference presentations and news outlets," Stoss said. "However, absent more specific data and information from manufacturers, it is difficult for payers to estimate the potential impact on utilization and spending."
In particular, Bristol-Myers Squibb Co.'s Opdivo (nivolumab), which was approved in 2014 for unresectable or metastatic melanoma, was approved for seven additional indications over the next 13 months.
A similar scenario occurred with Merck & Co. Inc.'s Keytruda (pembrolizumab), also approved for unresectable or metastatic melanoma. It received three new indications in three months.
Stoss said that substantially increased potential usage of the drug and Humana knew that the indications were under consideration, but was surprised by the timing of them.
He said payers suffer from a lack of "visibility into future product pipelines."
"While we knew there was potential for these additional indications, we did not anticipate that the approvals would come so quickly, often well before the clinical trial and end dates posted on clinicaltrials.gov," Stoss said.