Republic of the Philippines
HOUSE OF REPRESENTATIVES
Quezon City

FIFTEENTH CONGRESS
First Regular Session

H.B.No._392

Introduced by HONORABLE JUAN EDGARDO “SONNY” M. ANGARA

EXPLANATORY NOTE

This Bill 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
Disease in the Department of Health. The Office of Rare Disease will supervise the
implementation of a research program on rare diseases, ensure the provision of early 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
from rare 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 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 learning 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 life, 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.

Hence, we seek the early passage of this Bill.
DL.
ier
JUAN EDGARDO “SONNY” M, ANGARA

Representative
Lone District, Province of Aurora



FIFTEENTH CONGRESS OF THE )
REPUBLIC OF THE PHILIPPINES —)
First Regular Session )

H. B. No.

Introduced by Honorable JUAN EDGARDO “SONNY” M. ANGARA

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

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

ARTICLE 1
GENERAL PROVISIONS

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

SEC, 2. Declaration of Policy. It is the policy of the State to protect and promote the
right to health of the people, 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 rare 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.

SEC. 3. Objectives. The objectives of this Act are as follows
a) 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;

b) Establish an office within the Department of Health: (i) to oversee research &
development activities on rare diseases;

to design and maintain a rare disease
registry which shall include data on rare diseases in the Philippines, patients afflicted

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with rare diseases, and orphan drugs and products; and (ii) to conduct public education
programs to identify persons afflicted with rare diseases and help the public understand
the special needs of such persons.

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

d) 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 2
DEFINITION OF TERMS

SEC. 4. Definitions. Under this Act, the following terms shall have the meanings

respectively given to them below:

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

b) Healthcare institutions means hospitals, health infirmaries, health centers, lying-in
centers or puericulture centers, whether public or private.

©) Medical care 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.

) 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

f) Rare Disease means 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 diseases affecting no more than 1 in 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 (ie., life threatening, seriously debilitating, or serious and chronic)

forms of more frequently occurring diseases

9) 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 3

DESIGNATION OF RARE DISEASE, ORPHAN DRUG, AND
ORPHAN PRODUCT STATUS.

SEC. 5. Designation of Rare Disease. Gaucher Disease, Maple Syrup Urine Disease,
Pompe Disease, Galactosemia, Phynelketonuria, Methylmalonic Acidemia, Urea Cycle Defects,
Hurler Syndrome, Hunter Syndrome, Prader-Willi Syndrome and Lubag 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 afflict no more than 1 in 200,000
persons in the Philippines as a rare disease.

SEC. 6. Designation of Orphan Drug. The Department of Health, motu proprio or 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 alternative
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, Pompe Disease, Galactosemia, Phynelketonuria,
Methyimalonic Acidemia, Urea Cycle Defects, Hurler Syndrome, Hunter Syndrome, Prader-Willi
Syndrome, and Lubag 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
Institute of Health, shall publish a list of orphan drugs for these rare diseases.

SEC. 7. Designation of Orphan Product. The Department of Health, motu proprio or
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

3:


Disease, Galactosemia, Phynelketonuria, Methylmalonic Acidemia, Urea Cycle Defects, Hurler
Syndrome, Hunter Syndrome, Prader-Willi Syndrome, and Lubag are hereby considered as
orphan products. Within one hundred twenty 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
products for these rare diseases.

SEC. 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
Registration; 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

a) 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 annual volume requirement of the drug or product;

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

©) 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 used, the therapeutic or desired effect, and adverse reactions, if any;

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

e) 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 there
from.

The Permit for Use of an Orphan Drug/Orphan Product shall be effective for a period of three (3)
years, renewable for periods of three (3) years thereafter.

SEC. 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 made available 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
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 4

OFFICE OF RARE DISEASES

SEC. 10. (a) 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 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.

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

a) 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;

b) Promote coordination and cooperation among institutions involved in researches on
rare diseases, orphan drugs and orphan products;

c) 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;

) 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;

f) 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;


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

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

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

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

k) 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 shall 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 5

INCENTIVES FOR RARE DISEASE FUNDING AND RESOURCE
GENERATON
SEC. 11. Fiscal Incentives. The following shall be exempted from all taxes, whether
national or local:

a) Donations to the Office of Rare Diseases and the National Institutes of Health
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

b) 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 6
FINAL PROVISIONS

SEC. 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 Health, shall issue the implementing rules and regulations to this Act.

SEC. 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.

SEC. 14. Separability Clause. |f, 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.

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

Approved,