HOUSE F REPRESENTATIVES

Republic of the Philippines RECENTD
HOUSE OF REPRESENTATIVE: 24 iis
Quezon City Dave g? AUS 2018

Time 6:40 pm

BY:
SEVENTEENTH CONGRESS ——

First Regular Session memaaaaaian

BILLS AND INC EX SERVICE

House Bill nesss

Introduced by Representative 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 (1) supervise the implementation of a research program on rare diseases; (2)
ensure the provision of early and sustainable care for patients suffering from rare
diseases; and (3) 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 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 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 product 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.

| am therefore enjoining my colleagues to ensure the expeditious approval of this bill

GLORIA MACAPAGAL ARROYO,
2" Disffict, Pampanga


Republic of the Philippines
HOUSE OF REPRESENTATIVES
Quezon City

SEVENTEENTH CONGRESS
First Regular Session

House Bill No. 28.99

Introduced by Representative 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

Be it enacted by the Senate and 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 2016."

SECTION 2. Declaration of Policy. - It is the policy of the State to protect and
promote the right of 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 affective 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 diseases 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 orphan
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 II
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 Professional Regulatory Commission.

(2) Healthcare institution means hospitals, health infirmaries, health centers,
lying-in centers or puericulture 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 Institute of Health

(6) 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 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 (ie., 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 Ill
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, Methylmalonic
Acidemi, 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 rare disease

SECTION 6. Designation of Orphan Drug. - The Department of Health, motu
propio 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, Methylmalonic Acidemia, Urea Cycle Defects, Hurler Syndrome,
Hunter Syndrome, Prader-Willi Syndrome, Lubug, and other rare 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, motu
propio 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 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 consultation 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 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

(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 annual 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 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

(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 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 the status of patients; Provided, That such reports shall be subject to
guidelines issued by the National Institutes of Health to protect to 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 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; (i)
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 (ii) 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 rare 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 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 V
INCENTIVES FOR RARE DISEASE FUNDING AND RESOURCE GENERATION

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 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


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

SECTION 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,