Patient advocacy talk 1.28.18

Engagement with Independent
Patient Advocacy Groups
Synchrogenix
Darshan Kulkarni PharmD, MS, Esq
Vice President, Regulatory Strategy (US)

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UpdatesRecommendationsMistakesLegal Enforcement
Is patient
engagement
Important?

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Text Hererequired FDA “to develop and implement strategies to solicit the
views of patients during the medical product development process
and consider the perspectives of patients during regulatory
discussions” – FDASIA Section 1137 (2012)

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21st Century Cures Act
Requires FDA to issue new
guidance regarding methods
and approaches to be used
in capturing and measuring
patients’ experiences and
perspectives. The Section
requires that:
FDA develop a plan
Issue “one or more guidance
documents over a period of 5
years,”
Address a series of topics
outlined in the statute that
will be of interest to
stakeholders wishing to
participate in or engage in
patient focused drug
development.

© Copyright 2015 Certara, L.P. All rights reserved.
This is the Plan

Patient-Focused Drug Development: 2013-2017
1. Chronic Fatigue Syndrome and Myalgic Encephalomyelitis:
2. Lung Cancer:
3. Human Immunodeficiency Virus (HIV)
4. Narcolepsy
5. Sickle Cell Disease
6. Fibromyalgia:
7. Pulmonary Arterial Hypertension
8. Neurological Manifestations of Inborn Errors of Metabolism
9. Hemophilia A, Hemophilia B, von Willebrand disease, and Other Heritable Bleeding Disorders:
10. Idiopathic Pulmonary Fibrosis
11. Female Sexual Dysfunction
12. Breast Cancer
13. Chagas Disease
14. Functional GI Disorders
15. Parkinson’s Disease and Huntington’s Disease
16. Alpha-1 Antitrypsin Deficiency
17. Non-tuberculous mycobacterial infections
18. Psoriasis
19. Neuropathic pain associated with peripheral neuropathy
20. Patients who have received an organ transplant
21. Sarcopenia
22. Autism
23. Alopecia areata
24. Patient-Focused Drug Development for Hereditary Angioedema:

Other Initiatives
• A Voice of the Patient:A Series of Reports from FDA's
Patient-Focused Drug Development Initiative
• Public Workshop on Patient-Focused Drug Development:
Guidance 1 – Collecting Comprehensive and Representative
Input
• Patient Engagement Advisory Committee
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Other Opinions
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UpdatesRecommendationsMistakesLaws
Is Patient
Engagement
Important?

Legal & Regulatory Enforcement
Kulkarni Law Firm, PC
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HIPAA
Anti-Kickback
Law
False Claims
Act

HIPAA
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“a person who offers or transfers to a Medicare or Medicaid beneficiary any
remuneration that the person knows or should know is likely to influence the
beneficiary’s selection of a particular provider, practitioner, or supplier of
Medicare or Medicaid payable items or services may be liable for civil money
penalties (CMPs) of up to $10,000 for each wrongful act.”
section 1128A(a)(5) of the Social Security Act, enacted
as part of Health Insurance Portability and
Accountability Act of 1996 (HIPAA).

1.Bars the offering of remuneration to Medicare or
Medicaid beneficiaries where the person offering the
remuneration knows or should know that the
remuneration is likely to influence the beneficiary to order
or receive items or services from a particular provider.
Remuneration: Remuneration” includes, without limitation,
waivers of copayments and deductible amounts (or any part
thereof) and transfers of items or services for free or for other
than fair market value
“Should know”: The “should know” standard is met if a
provider acts with deliberate ignorance or reckless disregard.
No proof of specific intent is required
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HIPAA
Anti-Kickback
Law
False Claims
Act

What Matters – Anti-Kickback Law
How is it different from the FCA?
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Anti Kickback Law
Whoever knowingly and willfully solicits or receives any
remuneration (including any kickback, bribe, or rebate)
directly or indirectly, overtly or covertly, in cash or in kind—
(A) in return for referring an individual to a person for the
furnishing or arranging for the furnishing of any item or service
for which payment may be made in whole or in part under a
Federal health care program, or
(B) in return for purchasing, leasing, ordering, or arranging for
or recommending purchasing, leasing, or ordering any good,
facility, service, or item for which payment may be made in
whole or in part under a Federal health care
42 USC § 1320A–7B
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…solicits or
receives any
remuneration..
…for furnishing or
arranging.. Any item
or service for which
payment was made..

Intent - AKL
“a person need not have actual
knowledge of this section or specific intent to
commit a violation of this section.”
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Anti-Kickback Law - Penalties
“… shall be guilty of a felony and upon conviction thereof, shall
be fined not more than $25,000 or imprisoned for not more than
five years, or both.”
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$25000
Imprisonment
= 5 years
Penalties

What Matters – Anti-Kickback Law
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FCA
Anti-
Kickback

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HIPAA
Anti-Kickback
Law
False Claims
Act

What Matters - FCA
What is it?
o Actions brought by the Department of Justice
o Whistleblower or qui tam provisions allow
individuals to bring actions in the name of the
government, and can share in any recovery
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No specific
intent to
defraud must
be proved

What Matters -FCA
“any person who—
(A) knowingly presents, or causes to be presented, a false or
fraudulent claim for payment or approval;
(B) knowingly makes, uses, or causes to be made or used, a
false record or statement material to a false or fraudulent claim;
… “
31 USC § 3729
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“presents, or
causes to be
presented a
false.. claim”
“makes, uses,
or causes to be
made or used, a
false record…”

Penalty & Damages - FCA
… is liable to the United States Government for a civil penalty
of not less than $5,500 and not more than $11,000, …, plus 3
times the amount of damages which the Government sustains
because of the act of that person.”
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5500/
11000
3x Govt.
damages
Civil
Penalty

Concerns Raised
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Donor pressure to take policy positions that
are best for the donors – Stat News, Jan 2017
Doubted their own level of
independence
- Stat News, Jan 2017
Disguised promotion channeled through a
seemingly neutral third party – Public Citizen
April 2011
Confusion between patients' and sponsors'
interests in policy of patient groups
- Citizen.org April 2011

Why Do We Care?

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UpdatesRecommendationsScenariosLegal EnforcementScenarios

Primary Forms of Assistance
http://pharmaceuticalcommerce.com/brand-marketing-communications/pharma-struggles-to-manage-the-complexity-
of-its-patient-assistance-programs/

What do you think?
Assume for all of the following (unless stated otherwise) that the
patient is obtaining drugs via a Medicare Part D plan.
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Patient is single and makes $80,000. She does
is considering a trip to Aruba next year. The
PAP in question says that she makes too much
money and will hence deny her request for PAP
assistance.
It is not ok to base PAP accessibility only on
financial need. Financial need is one of many
conditions to consider.
CEO John’s mom died from Fabry disease. His
company FabryCo makes the newest treatment
for the rare disease and is expected to have a
98% treatment rate. Unfortunately, he expects
the drug to cost $200,000 a year. He knows that
some patients will be charged upto $10,000 in
co-pays per year. He wants treatment for the
product to reach as many patients as possible
and hence offers to have his company cover the
cost of any co-pay for patients using his drug.
This would likely be considered a pharma
company steering patients towards a specific
drug, and the PAP would likely be considered to
be a sham charity looking to offset drug costs
incurred by Part D beneficiaries.
CEO John hears about Sister Mary’s Patient
Assistance Program from his wife Vanessa. His
wife does not (and never has) worked for
FabryCure. She merely serves as a board
member at St. Mary’s and knows that they can
help the patients and help commemorate John’s
mom.
Neither the pharmaceutical manufacturer nor
any affiliate of the manufacturer (including,
without limitation, any employee, agent, officer,
shareholder, or contractor (including, without
limitation, any wholesaler, distributor, or
pharmacy benefits manager)) exerts any direct
or indirect influence or control over the charity
or the subsidy program
Vanessa retires from Sister Mary’s PAP. CEO
John wants to be sure that Sister Mary’s PAP is
using the money appropriately. They hence
send John a monthly message with the list of
how the money was used. No confidential
information is shared. Only aggregated
information is provided.
The pharmaceutical manufacturer does not
solicit or receive data from the charity that
would facilitate the manufacturer in correlating
the amount or frequency of its donations with
the number of subsidized prescriptions for its
products. So this may be ok.
Sister Marys has recently had to deal with some
fake patients. So, they require that the patients
have a doctors note confirming that they have a
SCr >3, neuropathy, and tinnitus. Additionally, to
optimize use of resources, the payments were
restricted to patients who have to pay for
expensive intravenous drugs.
Charities may not artificially define their disease
categories so narrowly that the earmarking
effectively results in the subsidization of one (or
a very few) of donor’s particular products.
Sister Mary’s wants to be compliant with CMS
and OIG expectations. Accordingly, it decides to
only cover drugs for their on label indications.Refusal to cover drugs for off-label indications
may not be appropriate.
Sister Marys is very happy with the generous
donation by FabryCure. They hence put up
banners talking about FabryCure and the
wonderdrug they have.
This arrangement likely destroys the idea that
FabryCure is not connected to Sister Marys.
Additionally, it likely constitutes advertising and
may be subject to FDA requirements.

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UpdatesRecommendationsMistakes
Legal
Enforcement
Is Patient
Engagement
Important?

Typical Reviewing Strategy
Does the donation made to a
Patient Assistance Program (PAP):
• Induce the PAP to recommend or arrange
for the purchase of the donor’s federally
reimbursable items?
• Is the PAP’s grant of financial assistance
made to influence the patient to purchase
(or induce the patient’s physician to
prescribe) certain items?

Factors to Consider
Nature of arrangement
Structure of
relationshipSource of SponsorshipFunding provided
Likelihood to influence
a beneficiary’s
selection of a particular
provider, practitioner or
supplier?
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Types of PAPs for Part D
Pharmaceutical
Manufacturer
PAPs
Independent Charity PAPs
PAPs operating
outside Part D
Coalition Model PAPs
Bulk
Replacement
Models
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Provided directly by pharma
company
Third party provider
Typically ok
Multiple pharmaceutical
manufacturers would join together
to offer financially needy Part D
enrollees a card or similar vehicle
Pharmaceutical manufacturers (or
their affiliated PAPs) provide in-
kind donations in the form of free
drugs to pharmacies, health
centers, clinics, and other entities
that dispense drugs to qualifying
uninsured patients

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Updates
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ations
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Companies Being Investigated

• “…the CIA also requires that Aegerion implement controls
and monitoring designed to ensure true independence from
any charity patient assistance programs to which it donates in
the future.”
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Drug Maker Aegerion Agrees to Plead Guilty; Will Pay More Than
$35 Million to Resolve Criminal Charges and Civil False Claims
Allegations – September 2017

OIG Reversal

Basics.
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• OIG issued favorable opinion regarding the Charity’s
operation of a PAP to provide grants to defray medical
expenses
April 4, 2002
• Sent the charity a letter that highlighted concerns.
• Asked for a series of certifications;
• Issued a Supplemental Special Advisory Bulletin regarding
Independent Charity Patient Assistance Programs
May 21, 2014
• Released new advisory opinion
March 3, 2017

Lawsuit by PSI
• Filed Jan 8, 2018
• Concerns:
o “[PSI] does not, and will not, solicit suggestions from donors regarding
the identification or delineation of disease funds.
o “[n]o donor or affiliate of any donor (including, without limitation, any
employee, agent, officer, shareholder, or contractor (including without
limitation, any wholesaler, distributor, or pharmacy benefits manager))
directly or indirectly influences or will influence the identification or
delineation of any of [PSI’s] disease funds.”
o “[PSI] will not establish or modify funds for specific diseases at the
request or suggestion of donors or prospective donors (or affiliates of
donors or prospective donors) that manufacture drugs or devices for
the treatment of such diseases or that otherwise have a financial
interest in the establishment of modification of such funds.”
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Why?
First amendment: Government-
imposed censorship shuts off
essential avenues for PSI to
obtain information necessary to
create or modify a PAP and
thereby places an untenable
burden on PSI’s operations.
Wastage of resources

Ex rel Steve Greenfield v. Medco Health Solutions, Accredo
Health Group, Hemophilia Health Services
Steve Greenfield,
Fmr. area VP of Accredo
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Accredo Health Group, Inc.,
Specialty pharmacy
Facts Accredo made donations to charities, two of
which allegedly recommended Accredo as an
approved provider for hemophilia patients.

District Court
Greenfield must show that federally insured
patients were referred to Accredo as a result of
its donations to HSI/HANJ.
“Absent some evidence . . . that those patients
chose Accredo because of its donations to
HANJ/HSI,” the Court reasoned, Greenfield could
not carry his burden on his claim.
Greenfield appeals, arguing the District Court
erred in requiring him to prove a direct link
between the alleged kickback scheme and each
false claim
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3rd Circuit Court holding
There must be “proof the harm would not have occurred in the
absence of—that is, but for—the defendant’s conduct.”
For a False Claims Act violation, Greenfield must prove that at
least one of Accredo’s claims sought reimbursement for medical
care that was provided in violation of the Anti-Kickback Statute
(as a kickback renders a subsequent claim ineligible for payment
A kickback does not morph into a false claim unless a particular
patient is exposed to an illegal recommendation or referral and a
provider submits a claim for reimbursement pertaining to that patient
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Potential Methods of Engagement
Sunshine
Engagement
..Transparency
and disclosure
is important and
can be useful.
Tech Enabled
Engagement
..Rules based
engagement
..Just in time
responses
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Synchrogenix Patient Engagement
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Questions?Questions?

Patient advocacy talk 1.28.18

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