Republic of the Philippines
HOUSE OF REPRESENTATIVES
Quezon City

FIFTEENTH CONGRESS
First Regular Session

oT 4613

House Bill No.

Introduced by Representatives
DIOSDADO MACAPAGAL ARROYO and GLORIA MACAPAGAL ARROYO

AN ACT
TO HELP PERSONS AFFLICTED WITH RARE DISEASES BY CREATING AN
OFFICE OF RARE DISEASES IN THE DEPARTMENT OF HEALTH,
ENCOURAGING THE CONDUCT OF RESEARCH & DEVELOPMENT
ACTIVITIES ON RARE DISEASES, AND PROVIDING FOR FISCAL AND
REGULATORY INCENTIVES FOR THE MANUFACTURE OR IMPORTATION
OF HEALTHCARE PRODUCTS FOR USE BY SUCH PERSONS

EXPLANATORY NOTE

This proposed legislation seeks to establish a system that will help ensure
the early diagnosis and treatment of rare diseases in the Philippines through the
establishment of an Office of Rare Diseases in the Department of Health. The
Office of Rare Diseases will supervise the implementation of a research program
on rare diseases, ensure the provision of earty and sustainable care for patients
suffering from rare diseases, and conduct public educational and information
campaigns on rare diseases. The Bill also seeks to provide fiscal and regulatory
incentives to developers, manufacturers, and importers of medicines, medical
devices, diagnostic kits, and other pharmaceutical and nutritional products
needed by patients suffering frorn zare diseases.

A “rare disease”, otherwise called an “orphan disorder” is any health
condition resulting from genetic defects that afflict no more than 1 of every
20,000 individuals in the country. Examples are Gaucher Disease, Maple Syrup
Urine Disease, Pompe Disease, Galactosemia, Phynelketonuria, Methylmalonic
Acidemia, Urea Cycle Defects, Hurler Syndrome, Hunter Syndrome, Prader-Willi


Syndrome, and the so-called Lubag, which is found only in the island of Panay.
In the Philippines, it is estimated that less than 1000 individuals suffer from a rare
disease. Many of these cases are due to genetic defects, with many patients
exhibiting the symptoms during early childhood. Unfortunately, there is minimal
interest among research institutions in leaning more about these diseases
because they affect only a small segment of the population. Moreover, drugs
and healthcare products for these disorders have been called “orphan drugs” or
“orphan products” because of minimal attention paid by pharmaceutical
companies to them due to the high cost of production resulting from the absence
of commercially significant demand. Thus, patients die during infancy or, if they
manage to survive, suffer from chronic, debilitating illnesses that greatly diminish
their quality of fife, not to mention impose severe strain on their families.

This Bill will help provide patients with rare diseases better access to
adequate medical care, health information, and healthcare products needed to
treat their condition. It establishes a system to coordinate a sustainable research
& development initiatives and resource generation efforts among relevant
agencies of government and the private sector toward improving the quality of life
of patients with rare diseases and their families.

We are therefore enjoining our colleagues to ensure the expeditious
approval of this bill.

DIOSDADO MACAPAGAL ARROYO GLORIAMACAPAGAL ARROYO
Second District, Camarines Sur Secohd District, Pampanga


Republic of the Philippines
HOUSE OF REPRESENTATIVES
Quezon City

FIFTEENTH CONGRESS
Firat Regular Session

4613

House Bilt No,

Introduced by Representatives
DIOSDADO MACAPAGAL ARROYO and GLORIA MACAPAGAL ARROYO

AN ACT
TO HELP PERSONS AFFLICTED WITH RARE DISEASES BY CREATING AN
OFFICE OF RARE DISEASES IN THE DEPARTMENT OF HEALTH,
ENCOURAGING THE CONDUCT OF RESEARCH & DEVELOPMENT
ACTIVITIES ON RARE DISEASES, AND PROVIDING FOR FISCAL AND
REGULATORY INCENTIVES FOR THE MANUFACTURE OR {MPORTATION
OF HEALTHCARE PRODUCTS FOR USE BY SUCH PERSONS

Be it enacted by the Senate and the House of Representatives of the
Philippines in Congress assembled:

ARTICLE |
GENERAL PROVISIONS

SECTION 1. Short Title. — This Act shall be known as the “Philippine Rare
Diseases Act of 2011”.

SECTION 2. Declaration of Policy. — It is the policy of the State to protect
and promote the tight to health of the peaple, including the right of persons
suffering from rare diseases, to have access to timely health information and
adequate medical care. In pursuit of such policy, the State recognizes the leading
role of the Department of Health in overseeing research & development activities
on tare diseases and, while working with the private sector and nongovernmental
organizations, in designing and implementing programs for the benefit of those
afflicted with them. The State further recognizes that an effective public


education program is vital in helping ensure the early diagnosis and treatment of
rare diseases and in preventing those afflicted with them from being the subject
of discrimination.

SECTION 3. Objectives. — The objectives of this Act are as follows:

1) Ensure that every patient diagnosed to have a rare disease has access
to timely health information and adequate medical care,’ including drugs
and other healthcare products to treat or otherwise help them cope with
their condition;

2) Establish an office within the Department of Health: (i) to oversee
research & development activities on rare diseases; (ii) to design and
maintain a rare disease registry which shall include data on rare diseases
in the Philippines, patients afflicted with rare diseases, and orphan drugs
and products; and (iii) to conduct public education programs to identify
persons afflicted with rare diseases and help the public understand the
special needs of such persons.

(3) Provide regulatory and fiscal incentives to support research and
development activities on rare diseases and the import or manufacture
affordable osphan drugs or orphan products; and

{4) Institutionalize a financial incentive system for agencies involved in
clinical researches, patient care, medical information management, and
other similar activities for the benefit of persons afflicted with a rare
disease.

ARTICLE I
DEFINITION OF TERMS.

SECTION 4. Definitions. — Under this Act, the following terms shall have
the meanings respectively given to them below:

(1) Healthcare practitioner means any doctor of medicine, dentist, nurse,
midwife, allied health professionals and other health care professionals
duly licensed by the Professional Regulatory Commission.

(2) Healthcare institution means hospitals, health infimaries, health
centers, lying-in centers or puericutture centers, whether public or private.

(3) Medical cure means any method used by a health care practitioner to
prevent, diagnose, and remove the symptoms and cause of a disease.


(4) Orphan Drug means any drug or medicine used to treat or alleviate the
symptoms of persons afflicted with a rare disease and declared as such
by the Department of Health upon recommendation of the National
Institutes of Health.

(5) Orphan Product means any healthcare or nutritional product, other
than a drug or medicine, including, but not limited to, diagnostic kits,
medical devices and biological products, used to prevent, diagnose, or
treat rare diseases and declared as such by the Department of Health
upon recommendation of the National Institutes of Health.

(6) Rare Disease means Gaucher Disease, Maple Syrup Urine Disease,
Pompe Disease, Gatactosemia, Phynelketonuria,  Methyimalonic
Acidemia, Urea Cycle Defects, Hurler Syndrome, Hunter Syndrome,
Prader-Willi Syndrome, Lubag, and other diseases affecting no more than
‘one (1) in twenty thousand (20,000) individuals in the country and
recognized as such by the Department of Health upon recommendation of
the National Institutes of Health, For the avoidance of doubt, it does not
include catastrophic (i.e., life threatening, seriously debilitating, or serious
and chronic) forms of more frequently occurring diseases.

7) Rare Disease Registry means the health information system maintained
by the Department of Health, including the electronic database system,
relating to data on rare diseases, persons afflicted with rare diseases, and
orphan drugs and orphan products.

ARTICLE IIt
DESIGNATION OF RARE DISEASE, ORPHAN DRUG, AND ORPHAN
PRODUCT STATUS

SECTION 5. Designation of Rare Disease. — Gaucher Disease, Maple
Syrup Urine Disease, Pompe Disease, Galactosemia, Phynelketonuria,
Methyimatonic Acidemia, Urea Cycle Defects, Hurler Syndrome, Hunter
Syndrome, Prader-Willi Syndrome and Lubug are hereby designated as rare
diseases. The Department of Health, upon recommendation of the National
Institutes of Health, shall have the authority to designate any disease that afflicts

no more than one (1) in twenty thousand (20,000) persons in the Philippines as a
tare disease.

SECTION 6. Designation of Orphan Drug. - The Department of Health,
motu proprio of upon application by any interested person, may designate any
drug or medicine indicated for use by patients afflicted with any of the rare
diseases as an orphan drug; Provided, That there is no existing drug or medicine


in the Philippines that can provide the same or superior altemative therapy, as
‘certified by the National Institutes of Health.

The drugs or medicines for the treatment or for the alleviation of
symptoms of Gaucher Disease, Maple Syrup Urine Disease, Pomp Disease,
Galactosemia, Phynelketonuria, Methyimalonic Acidemia, Urea Cycle Defects,
Hurler Syndrome, Hunter Syndrome, Prader-Willi Syndrome, Lubag, and other
tare diseases designated as such by the Department of Health are hereby
deemed to be orphan drugs. Within one hundred twenty (120) days from the
effectivity of this Act, the Department of Health, in consultation with the National
Institutes of Health, shall publish a list of orphan drugs for these rare diseases.

SECTION 7. Designation of Orphan Product. - The Department of Health,
mofuproprio ot upon application by any interested person, may designate any
healthcare or nutritional product, other than a drug or medicine, including, but not
limited to, diagnostic kits, medical devices and biological products, used primarily
to prevent, diagnose, or alleviate the symptoms of rare diseases as an orphan
product; Provided, That there is no existing product in the Philippines that can
provide the same or superior results, as certified by the National Institutes of
Health.

Any healthcare or nutritional product, other than a drug or medicine, including,
but not limited to, diagnostic kits, medical devices and biological products, used
primarily to prevent, diagnose, or alleviate the symptoms of Gaucher Disease,
Maple Syrup Urine Disease, Pompe Disease, Galactosemia, Phynelketonuria,
Methylmalonic Acidemia, Urea Cycle Defects, Hurler Syndrome, Hunter
Syndrome, Prader-Willi Syndrome, Lubag, and other rare diseases designated
‘as such by the Department of Health, are hereby considered as orphan products.
Within one hundred twenty days from the effectivity of this Act, the Department of
Health, in consuttation with the National Institutes of Health, shall publish a list of
orphan products for these rare diseases.

SECTION 8 Permit for Restricted Use of an Orphan Drug/Orphan
Product. — Any person may import any orphan drug or orphan product without
need of obtaining a Certificate of Product Ragistration; Provided, That he first
secures a Permit for Use of an Orphan Drug/Orphan Product from the Bureau of
Food and Drugs. Within thirty (30) days from receipt of the following
requirements, the Bureau of Food and Drugs shall issue a Permit for Use of an
Orphan Drug/Orphan Product:

(1) Sworn application for the issuance of a Permit for Restricted Use of an
Orphan Drug/Orphan Product, containing the name and address of the
applicant and the estimated annua! volume requirement of the drug or
product;


(2) Certification from the Office of Rare Diseases that the drug or product
qualifies as an orphan drug or orphan product;

(3) In the case of a drug or medicine, medical device and diagnostic kit: (i)
the names and addresses of medical specialists qualified and authorized
to use them; (ii) a written commitment on the part of all the authorized
specialists to submit to the Bureau of Food and Drugs, with copies to the
National Institutes of Health and the Office of Rare Disorders, no later than
January 15 of each year, a Clinical Study Report for every patient
administered the drug or product describing the quantity administered or

(4) Certification that the drug or product is registered in the county of
origin; and

(5) An Affidavit stating that the applicant shall be responsible for any
death, injury or damage arising from the use of the orphan drug or orphan
product and holding the Bureau of Food and Drugs and its officials and
employees free and harmless therefrom.

The Permit for Use of an Orphan Drug/Orphan Product shall be effective
for a period of three years, renewable for periods of three years thereafter.

SECTION 9. Rare Disease Registry. - (a) The National Institutes of
Health, with the assistance of the Office of Rare Diseases, shall maintain a
national database of rare diseases, patients afflicted with rare diseases, orphan
drugs and orphan products which, except for the names of the patients afflicted
with rare diseases, shall be mad lable to any reputable research institution
working on rare diseases; Provided, That such access shall be subject to
guidelines issued by the National Institutes of Health to protect the privacy of
patients afflicted with rare diseases.

(b) All healthcare practitioners and health institutions shall be required to
report to the National Institutes of Health newly-diagnosed cases of rare diseases.
and provide reports on the status of patients; Provided, That such reports shall
be subject to guidelines issued by the National institutes of Health to protect the
privacy of patients afflicted with rare diseases.

(c) Health practitioners and health institutions shall inform patients afflicted
with rare diseases of relevant orphan drugs and orphan products in the Rare
Disease Registry.



ARTICLE IV
OFFICE OF RARE DISEASES

SECTION 10. Creation of Office of Rare Diseases. - Within one hundred
twenty (120) days from the effectivity of this Act, the Department of Health shall
create an Office of Rare Diseases to: (i) to oversee research & development
activities on rare diseases; (ii) to design and maintain a rare disease registy
which shall include data on rare diseases in the Philippines, patients afflicted with
rare diseases, and orphan drugs and products; and (iil) to conduct public
education programs to identify persons afflicted with rare diseases and help the
public understand the special needs of such persons.

In particular, it shall perform inter alia the following specific functions:

(1) Recommend agenda for the conduct and support of research and
development initiatives on rare diseases, orphan drugs and orphan
products to identify relevant research opportunities;

(2) Promote coordination and cooperation among institutions involved in
researches on rate diseases, orphan drugs and orphan products;

(3) Promote sufficient allocation of resources for the conduct of research
on rare disease, orphan drugs and orphan products;

(4) Enter into agreements with and award research grants for centers for
researches doing studies on rare diseases, orphan drugs and products;

(5) Prepare annual reports describing the research and development
activities supported by the Office, and identifying projects that should be
conducted in the future on rare disease, orphan drugs and orphan
products;

(6) Evaluate the activities of the National Institutes of Health in the
management of information system and ensure that the Rare Diseases
Registry is current and useful;

(7) Ensure appropriate coordination among interested agencies,
manufacturers, and organizations representing patients afflicted with rare
diseases;

{8) Inform health practitioners and the public regarding the availability of
the orphan drugs and orphan products;

(9) Promote the development of updated treatment guidelines on the
approach to patients suspected or diagnosed with rare diseases;


(10) Establish a referral system to facilitate the provision of appropriate
medical care to patients with rare diseases

(11) Liaise with national and international patient, health and scientific
organizations working on rare diseases, orphan drugs and orphan
products to improve patient access.

The Office of Rare Diseases shall be comprised of an Assistant Secretary
of the Department of Health, who shail act as Chairman, and one representative
each from the Bureau of Food and Drugs, the National Institutes Health, a
national patient support group. and a private healthcare company, as members.

ARTICLE V
INCENTIVES FOR RARE DISEASE FUNDING AND
RESOURCE GENERATON

SECTION 11. Fiscal incentives. — The following shall be exempted from
all taxes, whether national or local:

(1) Donations to the Office of Rare Diseases and the National Institutes of
Heatth intended for researches on rare diseases, maintenance of the Rare
Disease Registry. or for purchase of orphan drugs or orphan products for
use solely by patients with rare diseases; and

(2) Procurement of orphan drugs and orphan products for use solely by
patients with rare diseases, as certified by the National Institutes of
Health;

In addition, orphan drugs and orphan products for donation solely to
patients afflicted with rare diseases or institutions, as certified by the National
Institutes of Health, shall be exempt from payments of all tariffs and duties.

ARTICLE Vi
FINAL PROVISIONS

SECTION 12. Implementing Rules and Regulations. — Within one hundred
twenty (120) days from effectivity of this Act, the Department of Health, in
consultation with the National Institutes of Heaith, shail issue the implementing
rules and regulations to this Act.


SECTIQN 13. Repealing Clause. — All general and special laws, decrees,
executive orders, proclamations and administrative regulations, or any parts

thereof, which are inconsistent with this Act are hereby repealed or modified
accordingly.

SECTION 14. Separability Clause. ~ If, for any reason or reasons, any
part of provisions of this Act shall be declared or held to be unconstitutional or

invalid, other provision or provisions hereof which are not affected thereby shall
continue to be in full force and effect.

SECTION 15. Effectivity. — This Act shall take effect fifteen (15) days after
its publication in at least two (2) newspapers of general circulation.

Approved,