March 31, 2022

Accelerated Approval Pathway Reform

How it Could Work

The Accelerated Approval Integrity Act of 2022 (HR 6963), introduced by House Energy and Commerce Committee Chairman Pallone (D-NJ), aims to reform the FDA accelerated approval program to remove products from the market that do not demonstrate clinical effectiveness after a certain time period.

The FDA’s Accelerated Approval Program was created with safeguards to allow the FDA to withdraw products from market if they do not prove to be clinically beneficial; however, those safeguards are rarely used. The Accelerated Approval Integrity Act of 2022 will give the FDA additional authority that will allow the agency to remove products from market that do not prove to be clinically beneficial in a timely manner. The policy would require manufacturers to come to an agreement with the FDA on how post-accelerated approval studies will be conducted (i.e., enrollment targets, milestones, and study design) before accelerated approval is granted as well as requiring those studies to be underway at the time of approval. The accelerated approval status would also automatically expire one year after the post-approval studies were required to have been completed and no later than five years after the accelerated approval was granted. The two exceptions to that proposed timeline are if a post-marketing study has been completed and confirms the product to be clinically beneficial or if the HHS Secretary determines that “adequate progress” has been made. In addition, the bill would require a disclaimer that a product has been approved under the accelerated approval pathway on the product’s labeling.

What Experts Think

The CIDSA experts unanimously agreed that this policy would minimally reduce drug spending. Similarly, they all agreed that neither list nor net prices would be affected by this proposed legislation. The majority of experts (6) believed that this policy would moderately reduce access for rare disease patients, while the other two experts believed access would not be affected. One expert believed uninsured patients and large patient groups would have a moderate reduction in access, whereas the majority of the experts believed those groups would not be affected. Similarly, three experts opined that Medicare, Medicaid, and privately insured patients would see a moderate reduction in drug access, where as the remaining experts opined those groups would not be affected.

The CIDSA experts unanimously agreed that this bill would minimally advance drug spending policy landscape. The vast majority of the experts agreed that the ability for the policy to be implemented, the evidence base in support of this policy, and the precedent-setting value of this policy should all be considered strengths of the policy. The experts were split whether that the magnitude of the impact that this policy would have on drug spending should be considered as strength or a weakness. Finally, there was no consensus on whether the size of the affected population would be a strength or weakness.

*Only 8 experts were able to complete this survey.

How likely would this policy be to reduce drug spending?
Would Increase Drug Spending
Would Not Affect Drug Spending
Would Minimally Reduce Drug Spending
Would Moderately Reduce Drug Spending
Would Significantly Reduce Drug Spending
Would Substantially Reduce Drug Spending
How likely would this policy be to reduce drug prices?
Would Significantly Increase Drug Prices
Would Moderately Increase Drug Prices
Would Not Affect Drug Prices
Would Moderately Decrease Drug Prices
Would Significantly Decrease Drug Prices
List Prices
Net Prices
How likely would this policy be to increase patient drug access?
Would Significantly Reduce Drug Access
Would Moderately Reduce Drug Access
Would Not Affect Drug Access
Would Moderately Increase Drug Access
Would Significantly Increase Drug Access
Rare Disease
Large Patient Groups
How significant is this policy in the evolution of US drug spending policy?
Does Not Advance Drug Spending Policy
Minimally Advances Drug Spending Policy
Moderately Advances Drug Spending Policy
Significantly Advances Drug Spending Policy
Ground-Breaking Shift in Drug Spending Policy
What are strengths and weakness of this policy?
Size of Affected Population
Evidence Base in Support of Policy
Precedent-Setting Value
Magnitude of Drug Spending Impact
How important are the following in your analysis of the policy's impact?
Not Important
A Little Important
Somewhat Important
Very Important
The likelihood of HHS Secretary saying adequate progress has been met
Clarification on whether reduced access means reduced access to the drug or to drugs in general
Impact on biotech industry’s R&D investments
No specification of pricing policy in advance of final approval
Ability of manufacturers to recruit patients for trials in a timely manner
Unclear how drug manufacturers will respond to this set of incentives
Unclear how providers will respond
Uncertainty surrounding enforcement

Considerations for Policymakers

Experts highlighted many considerations for policymakers. The primary concerns that the experts brought up were the uncertainty surrounding the enforcement of the policy, the likelihood of the HHS Secretary declaring that adequate progress has been met, and that there is no specification of pricing policy in advance of final approval notice. Other considerations that the experts believed could be important were the uncertainties surrounding the possible impact on R&D investments, the providers’ response, and the manufacturers’ response to the set of incentives. Other minor concerns were the need for clarification on whether “reduced access” means reduced access to the drug or to drugs in general and a manufacturer’s ability to recruit patients for trials in a timely manner.