21st Century Cures Act: The 21st Century Cures Act was signed into law on December of 2016. The law authorized $6.3 billion in funding, primarily to the National Institutes of Health (NH) and the Food and Drug Administration (FDA). The Cures Act was designed to encourage development of medical products and devices, and to streamline the process of getting them into market.
Accelerated Approval: The Food and Drug Administration (FDA) initiated the Accelerated Approval Program in 1992 to allow faster approval of drugs for serious conditions that fill an unmet medical need. Drugs with accelerated approval can initially be tested in clinical trials that use a surrogate endpoint, or something that is thought to predict clinical benefit. Surrogate endpoints typically require less time.
Accelerating Kids’ Access to Care Act: The Accelerating Kids’ Access to Care Act would make it easier for kids with Medicaid to access providers in other state Medicaid programs for children under the age of 21. The law requires that state Medicaid programs establish a federal process permitting out-of-state providers to participate without undergoing additional screening requirements. Participating providers would enroll for 5 years and be continuously renewed if in good standing. This legislation is pending in Congress.
Access to Genetic Counselor Services Act: The Access to Genetic Counselor Services Act would improve Medicare beneficiaries’ access to genetic counselors. This legislation would update current Medicare law to provide beneficiaries with direct access to genetic counselors and enables genetic counselors to bill Medicare directly with a reimbursement rate of 85% of physician fee schedule amount. This legislation is pending in Congress.
Affordable Care Act: The Affordable Care Act (ACA or Obamacare) was signed into law in March of 2010. It was designed to extend health coverage to millions of uninsured Americans by legally requiring them to buy health insurance. The Act had three primary goals.
- Make affordable health insurance available to more people regardless of their preexisting conditions by providing consumers with subsidies or “premium tax credits” that lower costs for households with incomes between 100% and 400% of the federal poverty level and by establishing marketplaces for people to shop for private health insurance plans that meet minimum coverage standards.
- Expand Medicaid to cover all adults with incomes below 138% of the federal poverty level.
- Support innovative medical care delivery models and payment policies designed to improve the quality of care and identify ways to lower the cost of health care.
Creating Hope Act: The Creating Hope Act establishes an incentive for drugs to be developed for children with rare diseases. The incentive is a priority review voucher that can be earned by a company that develops a rare pediatric disease drug.
Federal Advisory Committee Act (FACA): The Federal Advisory Committee Act became law in 1972 and is the legal foundation defining how federal advisory committees operate. The law has special emphasis on open meetings, chartering, public involvement, and reporting.
Food and Drug Administration Safety and Innovation Act (FDASIA): The Food and Drug Administration Safety and Innovation Act, signed into law in 2021, expands the FDA’s authorities and strengthens the agency’s ability to safeguard and advance public health by:
- Giving the authority to collect user feeds from industry to fund reviews for innovator; drugs, medical devices, generic drugs and biosimilar biological products;
- Promoting innovation to speed patient access to safe and effective products;
- Increasing stakeholder involvement in FDA processes; and
- Enhancing the safety of the drug supply chain.
Guidance Document: Non-binding advice given by an administrative agency such as FDA to the public regarding how best to comply with a law or regulation. Guidance is often used to explain the objective or interpretation of a vague or nonspecific law or requirement.
HELP Copays Act: The HELP Co-Pays Act requires health plans to count the value of co-pay assistance toward patient cost-sharing requirements. The bill also closes a loophole in the Affordable Care Act that allows employer health plans to deem certain categories of prescription drugs as non-essential and thus not count any cost-sharing towards the patient’s deductible or out-of-pocket maximum.
ICD Code: The International Classification of Diseases (ICD) is the classification used to code and classify morbidity data from the inpatient and outpatient records, physician offices and mortality records for death certificates.
Inflation Reduction Act (IRA): The Inflation Reduction Act addressed a range of policy issues and was signed into law on August 16, 2022. Parts of the IRA that focus on healthcare primarily affect people on Medicare and those who get their health insurance through the Affordable Care Act marketplace. The law extended the availability of substantial subsidies for individuals to purchase insurance on the marketplace and made several changes to lower the out-of-pocket costs that people on Medicare pay to access their prescription drugs. The IRA also created the Medicare Drug Price Negotiation Program.
The Lobbying Disclosure Act (LDA): The Lobbying Disclosure Act of 1995 is legislation aimed at bringing increased accountability to federal lobbying practices. This law applies to legislative and executive brand contacts. The LDA does not apply to state or local lobbying.
Medicare Drug Price Negotiation Programs: The Inflation Reduction Act passed by Congress and signed into law by President Biden he created the Medicare Drug Price Negotiation Program (MDPNP), administered by the Centers for Medicaid and Medicare Services (CMS), to negotiate the prices of certain prescription drugs as ordered by the Inflation Reduction Act (IRA). Beginning in 2026, 10 drugs covered under Medicare Part D will become eligible for negotiation, and each year, the number of drugs available for negotiation will increase. Drugs covered under Medicare Part B will also become available for negotiation beginning in 2028. The IRA exempted orphan drugs that are approved to treat only one rare disease or condition from being available for negotiation.
Medical Device User Fee Amendments (MDUFA): Device user fees were established in 2002 by the Medical Device User Fee and Modernization Act. Under the user fee system, medical device companies pay fees to the FDA when they register their establishments and list their devices with the agency, whenever they submit an application or a notification to market a new medical device. These fees are used to help the FDA increase the efficiency of regulatory processes with a goal of reducing the time it takes to bring safe and effective medical devices to the market.
MVP Act: The Medicaid VBPs for Patients (MVP) Act would expand access to treatments, such as gene therapy, by establishing a value-based purchasing structure. Affordability would be based on the treatment’s effectiveness, resulting in greater access for patients on Medicaid. This legislation is pending in Congress.
Newborn Screening Saves Lives Act: Congress passed the original Newborn Screening Saves Lives Act in 2008, which established national newborn screening guidelines and helped facilitate comprehensive newborn screening in every state. The Act was first reauthorized in 2014.
Orphan Drug Act (ODA): The Orphan Drug Act is a law that was passed in 1983 to facilitate development of orphan drugs. Orphan drugs are drugs that remain undeveloped or neglected because of limited potential for commercial gain.
Orphan Drug Tax Credit: The Orphan Drug Tax Credit (ODTC), established as part of the ODA, allows companies to claim a tax credit for a portion of their qualified research expenses. Originally set at 50% of qualified expenses, the credit was reduced to 25% in 2017.
Patient Experience Data: Patient experience data is defined in the 21st Century Cures Act as data that is collected with the intention to provide information about patients’ experiences with a disease or condition. Patient experience data can be interpreted as information that captures
experiences, perspectives, needs, and priorities.
Patient Focused Drug Development: A systematic approach to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into the development and evaluation of medical products throughout the medical product life cycle.
Prescription Drug User Fees Amendments (PDUFA): The Prescription Drug User Fees Act was created by Congress in 1992 and authorizes the FDA to collect feeds from companies that produce certain human drug and biological products. PDUFA must be reauthorized every five years. The last reauthorization, PDUFA VI, was passed in 2017 and included the following improvements:
- Formalized inclusion of Patient Experience Data into the drug development and review process, including use of patient reported outcomes
- Use of real-world evidence for regulatory decision-making;
- Dedicated process to improve use of biomarkers as surrogate endpoints in drug development
Priority Review Vouchers: A priority review vouchers are earned by pharmaceutical companies for the development and approval of drugs treating rare pediatric diseases. The vouchers grant priority review to a drug developer as an incentive to develop treatments for drugs that might otherwise not be profitable to develop because of a smaller pool of patients needing treatment.
Regulation: Federal regulations are specific details directives or requirements with the force of law enacted by the federal agencies necessary to enforce the legislative acts passed by Congress.
Safe Step Act: The Safe Step Act would require group health plans to provide exceptions for medications protocoled by step therapy. Often referred to as “fail first,” step therapy is a practice in which health plans order patients to first try medications preferred by their insurance instead of approving the original medication prescribed by the doctor. The Safe Step Act would eliminate the potential harm patients face due to delayed access to treatments. This legislation is pending in Congress.
SCHIP: SCHIP, State Children’s Health Insurance Program, is a partnership between the federal government and state governments enacted by Congress in 1997. Children whose family income is above Medicaid eligibility levels may qualify for health care coverage depending on their state’s upper income limits for eligibility.
