Right to Try PRIA

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Annual Summary Reporting Requirements
Under the Right to Try Act
Docket No. FDA-2019-N-5553

Preliminary Regulatory Impact Analysis
Initial Regulatory Flexibility Analysis
Unfunded Mandates Reform Act Analysis

Economics Staff
Office of Economics and Analysis
Office of Policy, Legislation, and International Affairs
Office of the Commissioner

Table of Contents
I. Introduction and Summary .............................................................................................. 3 
A. Introduction ................................................................................................................ 3 
B. Summary of Costs and Benefits ................................................................................. 4 
II. Preliminary Economic Analysis of Impacts ................................................................... 6 
A. Background ................................................................................................................ 6 
B. The Need for Federal Regulatory Action ................................................................... 7 
C. Purpose of the Proposed Rule .................................................................................... 8 
D. Baseline Conditions ................................................................................................... 8 
E. Benefits of the Proposed Rule .................................................................................... 9 
F. Costs of the Proposed Rule ....................................................................................... 10 
G. Distributional Effects ............................................................................................... 13 
H. International Effects ................................................................................................. 13 
III. Initial Small Entity Analysis ....................................................................................... 13 
IV. References................................................................................................................... 14 

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I. Introduction and Summary
A. Introduction
We have examined the impacts of the proposed rule under Executive Order
12866, Executive Order 13563, Executive Order 13771, the Regulatory Flexibility Act (5
U.S.C. 601-612), and the Unfunded Mandates Reform Act of 1995 (Pub. L. 104-4).
Executive Orders 12866 and 13563 direct us to assess all costs and benefits of available
regulatory alternatives and, when regulation is necessary, to select regulatory approaches
that maximize net benefits (including potential economic, environmental, public health
and safety, and other advantages; distributive impacts; and equity). Executive Order
13771 requires that the costs associated with significant new regulations “shall, to the
extent permitted by law, be offset by the elimination of existing costs associated with at
least two prior regulations.” This proposed rule is not an economically significant
regulatory action as defined by Executive Order 12866.
The Regulatory Flexibility Act requires us to analyze regulatory options that
would minimize any significant impact of a rule on small entities. Because the effects are
low in cost and minimally dispersed, we propose to certify that the proposed rule will not
have a significant economic impact on a substantial number of small entities.
The Unfunded Mandates Reform Act of 1995 (section 202(a)) requires us to
prepare a written statement, which includes an assessment of anticipated costs and
benefits, before proposing “any rule that includes any Federal mandate that may result in
the expenditure by State, local, and tribal governments, in the aggregate, or by the private
sector, of $100,000,000 or more (adjusted annually for inflation) in any one year.” The
current threshold after adjustment for inflation is $154 million, using the most current
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(2018) Implicit Price Deflator for the Gross Domestic Product. This proposed rule would
not result in an expenditure in any year that meets or exceeds this amount.

B. Summary of Costs and Benefits
This proposed rule, if finalized, implements a statutory requirement in the Right
to Try Act that sponsors and manufacturers who provide an eligible investigational drug
under the Right to Try Act to eligible patients submit to the Food and Drug
Administration (FDA) an annual summary of such use. The Right to Try Act requires
FDA to specify by regulation the deadline, and requires that submissions include certain
information.
The proposed rule’s costs are summarized in Table 1; we are unable to quantify
benefits for this rule. This analysis estimates the incremental impacts of this proposed
rule, if finalized, for drug sponsors and these annual summary reports. Costs are
calculated as the time spent by firms to prepare and submit annual summary reports based
on participation in Right to Try Act requests from eligible patients for investigational
new treatments. The total estimated present value of this rule’s costs is $39,991 at a seven
percent discount rate and $49,345 at a three percent discount rate (in 2018 dollars). The
annualized cost of this rule over ten years is $5,694 at a seven percent discount rate and
$5,785 at a three percent discount rate.
The benefits of this rule consist of societal and public health outcomes that may
accrue from the disclosure of the use of investigational drugs and any known serious
adverse events provided in these annual summary reports. Without these reports, FDA
would not be made aware in a systematic manner of the use of eligible drugs under the
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Right to Try Act and any known serious adverse events. With these reports, there may be
increased awareness of investigational drugs, the diseases or conditions for which
patients are seeking access, and any known serious adverse events associated with such
use.
These reporting requirements instruct firms to collect all known serious adverse
events and submit them once per year to the FDA. In addition, based on the information
in these annual summaries, FDA intends to post an annual summary report in accordance
with section 561B(d)(2) of the FD&C Act. FDA’s posting of these reports may increase
awareness about the availability of investigational drugs. In some cases, access to such
drugs may help treat future patients.
Consistent with Executive Order 12866, Table 1 provides the costs and a
description of benefits for this proposed rule over a ten year period.

Table 1: Summary of Benefits and Costs in 2018 Dollars Over a Ten-Year Time Horizon
Primary
Estimate

Category
Annualized
Monetized
$/year
Annualized
Quantified

Low
Estimate

High
Estimate

Units
Year
Dollars
2018

Discount
Rate
7%

Period
Covered
10

2018

3%

10

7%
3%
Disclosure of
serious
adverse events
and outcomes
related
to
investigational
new
drug
treatments.

Benefits
Qualitative

Costs

Annualized
Monetized
$/year
Annualized
Quantified

Notes

$5,694

2018

7%

10

$5,785

2018

3%

10

7%
3%

5

Qualitative

Transfers

Effects

Federal
Annualized
Monetized
$/year
From/ To
From:
Other
Annualized
Monetized
$/year
From/To
From:
State, Local or Tribal Government:
Small Business:
Wages:
Growth:

7%
3%
To:
7%
3%
To:

In line with Executive Order 13771, in Table 2, we estimate present and
annualized values of costs continuing over an infinite time horizon.
Table 2 – EO 13771 Summary Table (in 2016 dollars, over a perpetual time horizon)
Primary (7%)

Primary (3%)

Present Value of Costs

$63,120

$176,799

Present Value of Cost
Savings
Present Value of Net Costs

$63,120

$176,799

Annualized Costs

$4,418

$5,304

$4,418

$5,304

Annualized Cost Savings
Annualized Net Costs

II. Preliminary Economic Analysis of Impacts
A. Background
The Right to Try Act was signed into law in May 2018, creating section 561B of
the Federal Food, Drug, and Cosmetic (FD&C) Act. This new law amends the FD&C Act
to establish an option for patients who meet certain criteria to request access to certain

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unapproved medical products, and for sponsors and manufacturers who agree to provide
certain unapproved medical products other than through FDA’s expanded access
program. The law establishes a new pathway for patients to request, and manufacturers or
sponsors to choose to provide, access to certain unapproved, investigational treatments
for patients diagnosed with life-threatening diseases or conditions who have exhausted
approved treatment options and who are unable to participate in a clinical trial involving
the investigational drug. The Right to Try Act is designed to facilitate patients’ access to
certain investigational drugs from manufacturers and sponsors who may choose to
provide such drugs – a process in which FDA is not involved.
Manufacturers or sponsors who provide their investigational drug under the Right
to Try Act are required to submit to FDA an annual summary of drugs supplied to
eligible patients. Specifically, this annual summary must include the name of the
investigational drug, the number of doses supplied, the number of patients treated, the
uses for which the drug was made available, and any known serious adverse events. FDA
is required to specify the deadline for such reporting submissions. This proposed rule, if
finalized, will provide information on the necessary contents of the annual summary and
the deadline for its submission.

B. The Need for Federal Regulatory Action
The Right to Try Act requires FDA to specify by regulation the deadline of
submission of an annual summary of an eligible investigational drug supplied by
manufacturers or sponsors to eligible patients. This proposed rule, if finalized, would
implement this provision of the Right to Try Act. This regulation would allow, if
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finalized, FDA to receive information about Right to Try Act access and activity,
including any known serious adverse events. Requests for access under the Right to Try
Act are not overseen by FDA. This rule would provide a mechanism for FDA to receive
information about the use of drugs and adverse event data associated with the Right to
Try Act in a systematic manner.

C. Purpose of the Proposed Rule
The purpose of this proposed rule is to implement section 561B(d)(1) of the
FD&C Act, as amended by the Right to Try Act, which requires sponsors and
manufacturers who provide an eligible investigational drug under section 561B of the
FD&C Act to submit to FDA an annual summary of such use and requires FDA to
specify by regulation the deadline of submission. The proposed rule, if finalized, would
provide information on the necessary contents of the annual summary along with the
deadline for its submission. Under this proposed rule, FDA would establish an email
address or electronic portal for these submissions.

D. Baseline Conditions
This proposed rule is part of the implementation of the Right to Try Act, and so
the baseline conditions refer to current conditions where the legislation has been enacted,
but the rule not yet promulgated; thus, patients and physicians are currently able to
determine eligibility, and drug sponsors are currently able to supply eligible
investigational drugs under the Right to Try Act if they are willing to do so.

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However, FDA has not yet specified by regulation the requirements for the
content and the deadline for submission of an annual summary report. This regulation
would affect any drug manufacturers or sponsors that provide an eligible investigational
drug to eligible patients under the Right to Try Act. We assume these drug sponsors,
when providing such eligible investigational new drugs to eligible patients, will have
information on the number of doses that they supplied, the life-threatening disease for
which the investigational drug was made available under the Right to Try Act, and
known serious adverse events. The incremental burden drug sponsors may encounter
from this regulation is the preparation and submission of the annual summary report
described in the proposed rule based on information that was likely already collected.

E. Benefits of the Proposed Rule
The benefits of this rule consist of societal and public health outcomes that may
accrue from the disclosure of the use of investigational drugs and any known serious
adverse events provided in these annual summary reports. Without these reports, FDA
would not be made aware in a systematic manner of the use of eligible drugs under the
Right to Try Act and any known serious adverse events. With these reports, there may be
increased awareness of investigational drugs, the diseases or conditions for which
patients are seeking access, and any known serious adverse events associated with such
use.
These reporting requirements instruct firms to collect all known serious adverse
events and submit them once per year to the FDA. In addition, based on the information
in these annual summaries, FDA intends to post online an annual summary report in
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accordance with section 561B(d)(2) of the FD&C Act. FDA’s posting of these reports
may increase awareness about the availability of investigational drugs.

F. Costs of the Proposed Rule
This proposed rule implements a statutory requirement: the date of a submission
of an annual summary report to be submitted by a drug manufacturer or sponsor
providing an eligible investigational new drug to an eligible patient and includes the
contents of the summary report. The incremental burden imposed by this proposed rule
will be in the form of costs associated with the drug sponsors’ compilation and
submission of these summary reports. This proposed rule is related only to the
submission of the annual summaries and not to the Right to Try Act requests made by
patients. The provisions of this proposed rule are not necessarily expected to lead to
additional Right to Try Act requests from patients. Thus, we do not expect that the
proposed rule would lead to additional summaries and incremental cost burdens.
The Right to Try Act specifies the content of these annual summary reports. The
drug sponsor’s annual summary report would be required to include (1) the name of the
investigational drug and applicable IND (investigational new drug) number, (2) number
of doses supplied, (3) number of patients treated, (4) uses or conditions for which the
drug was made available, and (5) any known serious adverse events or outcomes. The
proposed rule includes an example of a tabular summary that could be used for these data
fields and report submission to FDA.
The purpose and attributes, along with the intended preparer, of this annual
summary report are close to the information required in Form FDA 2252. Form FDA
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2252 is required to accompany all annual report submissions regarding new information
that might affect the safety, effectiveness, or labeling of a drug or biological product for
human use. The sponsor of the drug or biological product is responsible for collecting the
relevant information and submitting this form to the FDA. The time required to complete
this form has been previously estimated by the FDA to average five hours; this includes
time to review instructions, search and gather the existing data, and complete the
information collection1. Because the proposed rule’s tabular summary requires fewer
required data elements (such as Field 8: Reporting Period in Form FDA 2252) and less
summary information overall, we halve this time estimate to 2.5 hours. We request
comment and data on these assumptions and estimates.
We assume a medical director or regulatory affairs director will be responsible for
preparing and submitting this annual summary report. The mean hourly wage (in 2018)
for Medical and Health Services Managers in the Pharmaceutical and Medicine
Manufacturing industry was $106.39 [Ref. 1]. The total cost of labor is the fully-loaded
wage, which includes overhead and benefits. We assume that the cost of overhead and
benefits equals 100% of the wage, resulting in a fully-loaded total hourly wage of
$212.78. We estimate a cost of $531.95 (=$212.78 * 2.5 hours) to prepare and submit
each report.
Between passage of the Right to Try Act in May 2018 and January 2019, there
were two publicly reported instances of patients who have received access to

1

This estimate for Form FDA 2252 has been reviewed and approved by the Office of Management and
Budget. It was approved under OMB control number 0910–0001. See, Form FDA 2252 available at:
https://www.fda.gov/media/73005/download;
https://www.reginfo.gov/public/do/PRAViewDocument?ref_nbr=201704-0910-006.

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investigational treatments via the Right to Try Act pathway, though it is possible that
there are other cases that have not been made public2. The annual summary report must
include data for the preceding calendar year. For manufacturers or sponsors that have
supplied eligible investigational drugs between the period of enactment and the date the
final rule becomes effective, the first annual summary would be submitted 60 days after
the rule becomes effective and would include all uses of eligible investigational drugs
May 30, 2018 through the effective date of the final rule. Based on the two instances
publicly reported of so far, we estimate (and potentially overestimate) that there may be
six summaries submitted by drug sponsors included in this initial group of reports.
With increasing awareness of the Right to Try Act pathway for access to investigational
drugs without being part of clinical trials, the number of individual patients seeking
access to such treatments may rise. There is uncertainty concerning the extent of this
increase, however. There may be some increase in the years following passage of the
Right to Try Act, which may at some point level off or even decline. Table 3 outlines our
assumptions and estimates for the expected number of Right to Try Act annual summary
reports submitted. The cost to prepare and submit each report, estimated above at
$531.95, is multiplied by the estimated number of annual reports we expect to receive
over a ten-year period (six in year one, eight in year two, twelve in each subsequent year)
to obtain the overall estimated costs of preparing and submitting these annual summary
reports. We request comment on our assumptions and the number of years stakeholders
expect the impacts of the proposed rule, if finalized, would continue.

2

https://med.nyu.edu/pophealth/divisions/medical-ethics/compassionate-use#Q21

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Table 3: Expected Increase and Estimated Number of Right to Try Act Annual
Summary Reports
Year After Passage of Right to
Try Act
1
2
3
4
5 - 10

Expected Percent Increase in
Patient Requests and Annual
Reports
30
40
50
0
0

Expected Number of Annual
Reports
6
8
12
12
12

G. Distributional Effects
We do not expect there to be any distributional effects of this rule. This proposed
rule outlines the contents and deadline for an annual summary report submitted to FDA
by drug sponsors and is not expected to generate any disproportionate impact on any
specific industry or population group.

H. International Effects
We do not expect there to be any significant international effects of this proposed
rule. Both domestic and international drug sponsors would be subject to this annual
summary reporting requirement.

III. Initial Small Entity Analysis

The Regulatory Flexibility Act requires Agencies to analyze regulatory options
that would minimize any significant impact of a rule on small entities. Because the
number of annual summary reports is anticipated to be relatively small (as a percentage
of all investigational new drugs) and widely dispersed, we propose to certify that the

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proposed rule will not have a significant economic impact on a substantial number of
small entities. This analysis, as well as other sections in this document, serves as the
Initial Regulatory Flexibility Analysis, as required under the Regulatory Flexibility Act.
The Small Business Administration defines an entity in the pharmaceutical
industry as small if it has fewer than 1,250 employees. Based on this definition, about 90
percent of the drug entities are small. The impact on each entity will vary depending on
its information collection capabilities when the rule is implemented, but all firms within
this sector are familiar with the data and categories that comprise this annual summary
report. The submission and data collection requirements are generally straight-forward
and appropriate to the investigational new drug process. We request detailed comments
and data on the number of small entities that would be affected by the proposed rule, as
well as data on the economic impact of the proposed rule on these small entities.

IV. References

[1]
U.S. Bureau of Labor Statistics, May 2018. National Occupational Employment
and Wage Estimates United States. 2018. Available from:
https://www.bls.gov/oes/current/naics4_325400.htm.

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