Regulatory Requirements for Orphan Drugs

Regulatory Requirements for
Orphan Drugs Delivery
Dr. Basavaraj K. Nanjwade M.Pharm., Ph.D
Professor of Pharmaceutics
KLE University, Belgaum, India
E-MAIL: bknanjwade@yahoo.co.in
Cell No: 00919742431000

What is an Orphan Drug?
 A drug to test a rare disease or condition
affecting <200,000 people in the U.S.
 A vaccine, a preventive drug, or a
diagnostic drug to be administered to
<200,000 persons per year
 A drug with no expected profitability
within years even if prevalence >
200,000 people in the U.S.
02/03/2010 2BIOBIO-2010, Hyderabad

Orphan Drug Act Incentives
 Marketing Exclusivity-7 years
 Tax Credits-50% of Clinical
Investigations Expenses
 Exemption from PDUFA Application Fees
 Protocol Assistance in Drug
Development Process

Orphan Drug Act Incentives
 Grant Support-Medical Foods and
Medical Services also
 Open Protocols to increase drug
availability for patients
 Establish DHHS orphan products board

Orphan Drugs
 Among orphan drugs, 15-20% are for
diseases only affecting children, 55% for
diseases affecting both adult and
children
 Orphan drugs receive 10 years of
market exclusivity in EU
 Orphan drugs must submit a Pediatric
investigation plan
 Orphan drugs can get 2 additional years
of exclusivity when they comply with PIP

Paediatric Investigation Plan (PIP)
 Paediatric investigation plan proposed
by company
 By end of phase 1 for new products
 Plan is discussed, modified, and
agreed/refused by paediatric
committee
 Followed by EMEA decision
 Binding on company

Paediatric Investigation Plans
 Define necessary data on Quality, Safety
and Efficacy for use in the paediatric
population (0 – 18 years)
 No explicit link with adult indication
 Specify development timelines, including
deferral of studies
 Define age-appropriate formulation
 Results according to agreed plan serves
as basis for approval

PIP Waivers/Deferrals
On request from applicant or initiative of
Paediatric Committee, for all or part of
the paediatric population:
 Waiver of development for ‘classes’ of
indications, or for a specific product
 Deferral of initiation of studies and/or
completion
Development is most often a combination of a
plan with deferrals and waivers (population
subset)

Life cycle of a Paediatric
Investigation Plan
1
Phase 1 Phase 2 Phase 3 Post Approval
Modifications
MAPIP
Compliance
Paediatric Committee
Non-clin

Life cycle of a Paediatric
Investigation Plan
1
Non-clin Phase 1 Phase 2 Phase 3
ComplianceModifications
MAPIP With deferral
Paediatric Committee
Post Approval

Applications and Indications
 Each PIP corresponds to an active
substance (or a combination) from a
company
 Each PIP usually covers more than one
indication (e.g. high grade glioma and
neuroblastoma)

PIP Therapeutic areas (%)
- CNS 6
- Cardiovascular 10
- Oncology 14
- Anti-infectives 10
- Endocrinology/metabolism 21
- Immunology/vaccines 11
- Other 28

Tips for Applying for Orphan
Product Designation
1. The required information to be included
in the application is found under 21 CFR
316.20(b), shown as (9) items.
- Number the items in your application 1
though 9 and respond to the nine items
as described.
- Creative numbering is not helpful

Product Designation
2. While all nine items will be reviewed,
the application will be reviewed most
critically in two areas: scientific rationale
(Item 4) and population prevalence
(Item 8).
- Do not confuse prevalence with
incidence
- They are different entities and can’t be
substituted for one another

Product Designation
3. Information provided by the sponsor
relating to Item 7 is often incomplete
- Provide the IND or NDA/BLA numbers if
they are available to you

Product Designation
4. Format your application so that it is user
friendly
- After addressing the nine required
items, provide a bibliography displayed
in a related fashion as shown in the text
of your document.
- Usually references within the application
are cited numerically with superscripts

Product Designation
5. Submit the original and one photocopy
of the application in separate binders or
report covers to the office of Orphan
Products Development
- The photocopy needs to be an exact
duplicate of the original application.

Product Designation
- If there is a cover letter with the
original, there needs to be a copy of the
cover letter with the duplicate
- No correspondence should arrive loose
or out of the binder or report cover
- They are inexpensive and readily
available, plus they fit in standard filing
cabinets nicely

Product Designation
6. Submit the application (one original and
one photocopy) for orphan designation
to:
Director,
Office of Orphan Products Development
Food & Drug Administration, HF-35
5600 Fishers Lane, Room 6A55
Rockville, MD 20857

How to Apply for Designation as
an Orphan Product
(a)A sponsor that submits a request for
orphan drug designation of a drug for a
specified rare disease or condition shall
submit each request in the form and
containing the information required in
paragraph (b)
(b)A sponsor shall submit two copies of a
completed, dated, and signed request or
condition that contains

an Orphan Product
1. A statement that the sponsor requests
orphan drug designation for a rare
disease or condition, which shall be
identified with specificity

an Orphan Product
2. The name and address of the sponsor;
the name of the sponsor’s primary
contact person and/or resident agent
including title, address, and telephone
number; generic and trade name

an Orphan Product
3. A description of the rare disease or
condition for which the drug is being or
will be investigated, the proposed
indication or indications for use of the
drug, and the reasons why such therapy
is needed

an Orphan Product
4. A description of the drug and a discussion
of the scientific rationale for the use of
the drug for the rare disease or condition
- Copies of pertinent unpublished and
published papers are also required

an Orphan Product
5. Where the sponsor of a drug that is
otherwise the same drug as an already
approved orphan drug seeks orphan
drug designation for the subsequent
drug for the same rare disease

an Orphan Product
6. Where a drug is under development for
only a subset of persons with a
particular disease or condition, a
demonstration that the subset is
medically plausible

an Orphan Product
7. A summary of the regulatory status and
marketing history of the drug in the US
and in foreign countries, e.g., IND and
marketing application status and
dispositions

How to Apply for Designation
as an Orphan Product
8. Documentation, with appended
authoritative references, to demonstrate

An orphan disease has an incidence fewer than 5
patients per 10,000 population (EU) or <200,000
patients (US).

THANK YOU

Regulatory Requirements for Orphan Drugs

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Regulatory Requirements for Orphan Drugs