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Philippine Model Congress July 27-28, 2013 Philippine Senate Building Pasay City, Philippines Rare Diseases Act

of 2013

Explanatory Note This bill seeks to create an Office of the Rare Diseases (ORD) at the Department of Health. The ORD aims to supervise and oversee the research studies and inventions on the cure of rare genetic diseases, also known as orphan diseases. It also aims to provide financial incentives to developers, manufacturers, and importers of medicines; and financial assistance to the patients in need of medical devices, diagnostic kits, and other pharmaceutical and nutritional products. Its main goal is to coordinate all research institutions related to the nature of the office and collaborate to early diagnose and treat these very rare genetic diseases by strengthening new born screening and other means that help detect orphan diseases. The funding and operational expenses of this office will be appropriated from the annual budget of the Department of Health, specifically from the one percent (1%) excise tax collected from Republic Act 10351 or also known as Restructured Sin Tax Law. This bill will also serve as a supplemental law to Republic Act 9288 or also known as New Born Screening Act of 2004 to provide further assistance, most especially to the parents of the babies diagnosed with rare diseases. This will also help the Philippine Genome Center to utilize genomic research to develop more precise and accurate diagnostic kits for these kinds of diseases with the help of the soon to be established Rare Disease Registry. Rare Diseases or orphan diseases affect a small percentage of a population. Most of these cases are caused by genetic defects in the human genome that affects no more than one (1) of every twenty thousand (20,000) individuals in a country. These diseases are as follows: Gaucher Disease, Maple Syrup Urine Disease, Pompe Disease, Galactosemia, Phynelketonuria, Methylmalonic Acidemia, Urea Cycle Defects, Hurler Syndrome, Hunter Syndrome, PraderWilli Syndrome, and the so-called Lubag, which is found only in the island of Panay. Based on statistics, the Philippines has an estimated less than one thousand (1000) individuals suffering from a rare disease. These diseases mostly manifest symptoms during the early childhood stage. Research institutions are having a hard time conducting such research studies for there are too few individuals who can actually consider their studies significant. In addition, private pharmaceutical companies argued that it is not marketable to conduct studies on these diseases due to the high cost of research and production, as well as the absence of commercially significant demand. Consequently, orphan drugs or orphan products were not widely available since most research institutions have given minimal attention to cure. This bill will support patients with rare diseases and help them have better access to adequate medical care, health information, and healthcare products needed for treatment. It plans to establish a system that will organize sustainable research and development initiatives, as well as resource generation efforts among relevant agencies of government including the private

sector toward improving the quality of life of patients with rare diseases and their families. This bill will also reinforce the responsibility of the government as mandated in our State Policies in Article II Section 15 of the constitution which states that the State shall protect and promote the right to health of the people and instill health consciousness among them. The authors believe that persons suffering from rare diseases should have access to timely health information and adequate medical care.

Preamble The Philippine government resolves to provide access to health information, proper and adequate treatment specifically for people with rare genetic diseases; as well as to establish a database system registry for research and development of orphan diseases. The Congress will act to establish an Office for Rare Diseases that will coordinate and develop medical treatments for persons with rare diseases. Be it enacted by the Senate and the House of Representatives of the Philippines assembled.

Definition of Terms Healthcare practitioner - shall mean any doctor of medicine, dentist, nurse, midwife, allied health professionals and other health care professionals duly licensed by the Professional Regulatory Commission. Healthcare institution - shall mean hospitals, health infirmaries, health centers, lying-in centers, whether public or private. Medical cure - shall mean any method used by a health care practitioner to prevent, diagnose, and remove the symptoms and cause of a disease. Orphan Drug shall mean any drug or medicine used to treat 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 shall mean 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. Rare Disease shall refer 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.

Rare Disease Registry shall mean 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.

Content 1. Title. This Act shall be known as Rare Diseases Act of 2013. 2. Establishment of Office of the Rare Diseases (ORD). An office which shall be named as Office of the Rare Diseases which will be established within the Department of Health. Its functions are as follows: a. oversee research & development activities on rare diseases; b. design and maintain a rare diseases registry which shall include data on rare diseases in the Philippines, patients afflicted with rare diseases, and orphan drugs and products; c. conduct public education programs to identify persons afflicted with rare diseases and help the public understand the special needs of such persons; d. provide regulatory and fiscal incentives to support research and development activities on rare diseases, import or manufacture of affordable orphan drugs or orphan products, and 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. 3. Organization Structure of Office of the Rare Disease. The Office of Rare Diseases shall be comprised of: i. Assistant Secretary of the Department of Health (Chairman) ii. Assistant Secretary of the Department of Science and Technology iii. one representative from the Food and Drugs Administration iv. one representative from National Institutes of Health v. one representative from the Philippine Genome Center vi. one representative from a national patient support group

4. Designation of Rare Disease, Orphan Drug and Permit for Restricted use of Orphan Product. The Department of Health, upon recommendation and consultation of the National Institutes of Health, shall have the authority to: a. designate any disease that afflicts no more than one (1) in twenty thousand (20,000) persons in the Philippines as a rare disease; b. designate any drug or medicine indicated for use by patients afflicted with any of the rare diseases as an orphan product. 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. c. 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 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 Food and Drugs Administration and Certifications from the Office of Rare Diseases with consultation and recommendation with the National Institutes of Health. 5. Establishment of a Rare Diseases Registry. 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. All healthcare practitioners and health institutions shall be required to report on the status of the patients. Provided that such reports shall be subject to guidelines issued by the National Institutes of Health to protect the privacy of the patients. Health practitioners and health institutions shall inform patients afflicted with rare diseases of relevant orphan drugs and orphan products in the Rare Disease Registry. 6. Funding of the Office of Rare Diseases. The funding of the Office of Rare Diseases shall be automatically appropriated from the annual budget of the Department of Health specifically from the one percent (1%) excise tax collected from the R.A. 10351 or also known as the restructured Sin Tax Law. 7. Tax Exemption. 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 research studies 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; b. 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. c. For individuals, organizations, and companies who will donate orphan drugs and orphan products solely to patients afflicted with rare diseases as certified by the National Institutes of Health, the Department of Health in cooperation with the Bureau of Internal Revenue will provide the specificity of the duration of the tax exemptions for the sponsors of the donated orphan drugs and orphan products depending on their category.

8. Implementing Rules and Regulations. Department of Health, in consultation with the National Institutes of Health, shall issue the implementing rules and regulations to this Act. Proposed By: Joshua Israel Sumague Los Baos, Laguna John Daniel Ang Las Pias City Margarita Atienza Sta. Rosa City, Laguna Aaron Marc Dimaano Legazpi City, Albay Marlon Cantal Calamba City, Laguna Justin Paolo Interno Lucban, Quezon John Paulo Quitoriano Caoayan, Ilocos Sur Kathryn Legaspi - Bacoor, Cavite

References:

Angara, Eduardo. 2009. An Act to Help Persons Afflicted with Rare Diseases by Creating an Office of Rare Disease in the Department of Health, Encouraging The Conduct of Research and Development Activities on Rare Diseases and Providing For Fiscal and Regulatory Incentives For The Manufacture or Importation of Healthcare Product Use for Use by Such Person. Philippine Senate. Pasay City, Philippines Angara, Juan Edgardo. 2010. An Act to Help Persons Afflicted with Rare Diseases by Creating an Office of Rare Disease in the Department of Health, Encouraging The Conduct of Research and Development Activities on Rare Diseases and Providing For Fiscal and Regulatory Incentives For The Manufacture or Importation of Healthcare Product Use for Use by Such Person. House of Representatives. Quezon City, Philippines