The ORPHAN Cures Act is bipartisan legislation that would maintain existing incentives and boost research into new treatments for the 30 million Americans currently suffering from one of more than 7,000 rare diseases. The bill would achieve this by amending the Inflation Reduction Act’s harmful orphan drug exclusion and incentivizing critical follow-on investment into rare disease drug development.

The Inflation Reduction Act (IRA) of 2022 requires biopharmaceutical companies to pay rebates to the government if the price for drugs in Medicare Part B or Part D increase faster than the rate of inflation.

Maximum Fair Price: A manufacturer of a drug selected for negotiation will be required to
offer a “maximum fair price” for the selected drug with respect to Medicare beneficiaries.

Timeline: The first set of drugs will be selected for negotiation in September 2023, with the
negotiation process starting a month later. The first set of negotiated prices taking effect in
“initial price applicability year” 2026.

OVERVIEW MEDICARE PART D BENEFIT RESTRUCTURE

Currently, millions of Medicare beneficiaries reach the catastrophic phase in January or February, which exacerbates the financial hardship of the high prices of OOP costs. The Inflation Reduction Act (IRA) will allow Medicare beneficiaries to spread their out-of-pocket cost over the course of the plan year.

Congress created the 340B Drug Discount Program to help uninsured and vulnerable patients gain access to affordable prescription drugs and services. Over the years this program has expanded well past the original intent of Congress and now patients are not seeing the benefits they deserve.

How DSH Hospitals Abuse the 340B Drug Pricing Program

DSH Hospital Abuse the 340B Drug Pricing Program 

The details of the 340B Drug Pricing Program. 

The bioscience industry and its supporting ecosystem drive bold innovations to address unmet human needs in the U.S. and around the world, including developing new breakthrough therapies that treat deadly diseases. The collective efforts of the industry and our public and private stakeholders have met the challenges of the COVID pandemic head-on, while lessening the impacts of climate change and nourishing a growing global population. 

There is a misguided perception that NIH funding, not private market investment, is largely responsible for the creation and approval of new therapies. A new study by Vital Transformation¹ tests that hypothesis by tracking more than 20,000 patents linked to NIH grant over a 20 year period, identifying those associated with clinical trials and approved medicines, and quantifying the public and private investments made for those investigational and approved medicines.

Leveraging V3 for Digitally Derived Endpoints Framework - V3 as Applied to DHTTs Used in Various Drug Development Scenarios

Learn about clinical trials and get answers to your questions.

Patient Experience Data is the systematic collection of meaningful data related to the experiences, perspectives, needs, and priorities of patients. Below you will find a graphic that outlines types of patient experience data, how that data might be used in different phases of drug development and review.

Private entities take foundational knowledge from public research and invent practical applications that result in real-life treatments and cures for patients.

In the debate over drug costs, there is a lot of discussion about so-called “evergreening.” The truth is that the U.S. PTO applies the same legal standards to pharmaceutical patents as they do to other technologies.

Plant and animal breeding are continually improving as people learn more about biology. Gene editing is really just the newest tool in a very long history of biological advancement.

Genome editing is based on a naturally occurring system that directs a molecular scissor, called a nuclease, to a target region of DNA.

The goal of expedited approval pathways is to provide safe and effective therapies for serious, unmet medical needs as quickly as possible.

Learn how patient advocacy groups can get involved in the federal rulemaking process. 

Resources for emerging growth biotech companies would be better used for science than compliance with Section 404(b), economists find.

Learn about the Hatch-Waxman Act and its role in balancing innovation and drug competition.

PDUFA provides greater consistency, certainty and predictability in the FDA's drug review programs.

Learn about how the Bayh-Dole Act benefits the U.S. economy, consumers & patients.

Over the last five years, there has been a dramatic increase in the global development of transformative gene therapies. Given this increase and the global development, there is a clear need for harmonization across global regulatory authorities to ensure that these therapies can be delivered to the patients that need them as quickly as possible. Such harmonization efforts may include regulatory terminology and definitions, and nonclinical, quality, and clinical considerations.

Advancements by the biopharmaceutical sector have radically altered the trajectory of HIV/AIDS.

Biotechnology is helping to transform a terrifying diagnosis into a healthier and longer life for patients.

As Baby Boomers age, the rate of Alzheimer's disease will reach epidemic proportions.

Priority review vouchers provide a powerful incentive to stimulate drug development for underserved diseases or conditions.

Provider choice means providers decide what immunization is best for their patients.

The creation of a vaccine involves scientists and medical experts from around the world, and it usually requires 10 to 15 years of research before the vaccine is made available to the general public.

Vaccination is often cited among the 10 greatest public health achievements of the 20th century. Only access to safe and clean water has had a larger effect on human health by preventing disease and extending lifespans.

Reasons human cells are used in vaccine research and development:

• To develop a vaccine that can be produced on a large scale, researchers must grow viruses or bacteria in large quantities and with great consistency.
• Bacteria can be grown in a laboratory, but viruses need living cells to infect so they can make copies of themselves.
• Viruses tend to grow better in cells from humans than those of other animals.
• Human cells can be maintained for long periods of time at low temperatures, enabling scientists to use the same cell lines from many decades ago.

To ensure people get safe and effective vaccines as quickly as possible, biotechnology companies have leveraged global partnerships and
innovative collaborations to reach patients all around the world. 

When compared to other emerging diseases, see why influenza poses an especially dangerous and unique threat to society.

Certain infectious disease products have limited or no commercial market. No one incentive will address all challenges.

Learn about how America has become the global leader in civilian biodefense.

BIO supports the Growing Climate Solutions Act (GCSA) (S. 1251/H.R. 2820), which will foster sustainability and economic resiliency in agriculture and preserve America’s rich environmental diversity.

In recent years, America’s position as the global leader in new technologies has come under attack. As countries race to secure access to these technologies, governments are working to weaken global rules and international organizations, instead of investing in innovation. BIO recommends several foreign policy actions that will restore our standing as a global leader on biotechnology, enhance economic growth and job creation, and lay the groundwork for leveraging science and American innovation to solve society’s biggest and most pressing challenges.

Transportation emits more greenhouse gases than any other sector. Thanks to biotechnology, however, we can decarbonize all modes of transportation—air, land and sea—using sustainable sources like plants and waste.

Our obsession with plastics is harming our planet—from pollution in our oceans to emissions from production. Biotechnology is creating plastic alternatives—often called bioplastics—that reduce environmental impact by using renewable chemicals made from plants and other sustainable sources.

Biotechnology enables consumers to choose everyday products for their home with natural, sustainable and renewable chemical ingredients.

BIO estimates a direct U.S. economic impact of $205 billion and 1.665 million jobs from biobased manufacturing.

Innovations in agricultural biotechnology, such as new gene-editing tools like CRISPR, are crucial to transforming agriculture and food systems. With biotechnology, America’s farmers can sustainability increase production and resilience in the face of climate change, enhance America’s food supply through more affordable and nutritious products for consumers, and boost economies by lowering the cost of production and creating jobs. 

Because of the integration of the North American economies, enabled by the North American Free Trade Agreement (NAFTA) and recently confirmed through the United States-Mexico-Canada Agreement (USMCA), the agricultural markets of U.S. and Mexico are inseparably linked. Unfortunately, Mexico’s treatment of innovative biotech products is undermining the development and deployment of technologies critical to sustainably feeding the world and addressing climate change.

Innovations in animal biotechnology can improve human and animal health, make our farming and food systems more sustainable, and boost our bioeconomy. But these breakthroughs are dependent on a clear, timely, and science based regulatory approval process that provides a viable path to market.

BIO supports the Growing Climate Solutions Act (GCSA), which will support America’s farmers, ranchers, and foresters who want to adopt innovative practices that combat climate change, while continuing to provide the world with food, feed, and fiber. 

Government and industry must do more to build an informed and trust-based dialogue about biotechnology innovation in food and agriculture and its potential to achieve greater outcomes for the planet, our food system and human well-being.