The Orphan Drug Act (ODA), which was passed in 1983 to incentivize drug development for rare diseases, has come under scrutiny in recent years. There are some within industry who claim that the program has been manipulated by drug manufacturers to maximize profits and protect niche markets for treatments for more common conditions. However, advocates in [...]
The FDA has recently initiated a pilot program which will help drug manufacturers use complex innovative trial designs (CID). This move comes after the agency committed to helping facilitate and advance the use of complex adaptive, Bayesian, and other novel clinical trial designs, as part of the sixth iteration of the Prescription Drug User Fee [...]
Late last month, the European Medicines Agency (EMA) released a survey to help assess pharmaceutical companies’ preparedness for Brexit. The purpose of this assessment is to identify and address any issues that could potentially disrupt medicines access, or negatively affect human and animal health, following the UKs departure from the European Union (EU). The survey [...]
President Donald Trump has recently released his budget plan, and many within the healthcare sector are disappointed to see that The Creating and Restoring Equal Access to Equivalent Samples Act, or CREATES Act for short, has been left out. This bill promises to increase competition and lower prescription drug prices, by eliminating abusive delay tactics [...]
Expanded Access Programs (EAPs), also referred to as Managed Access or Compassionate Use Programs, allow patients with no viable treatment options available to them to access investigational drugs before they are approved for market use. In the US, the FDA is charged with approving these programs before patients can receive drug, and many see this [...]
Over the past year, there has been a lot of talk around how to reduce prescription drug prices in the US. However, there has been little consensus on the issue, which has made passing actual drug pricing reform difficult. As a result, lawmakers have recently introduced legislation which could cut costs in a way that [...]
House Republicans recently released a tax reform bill which would eliminate the tax credits currently awarded to drug companies for developing drugs for rare diseases. These tax credits are part of a package of incentives awarded to companies under the Orphan Drug Act of 1983, a piece of legislation introduced to try and encourage the [...]
For years, Randomized Controlled Trials (RCTs) have been the gold standard for assessing the safety and efficacy of investigational new drugs. These trials are considered the most effective at overcoming bias and yielding high-quality evidence because patients are allocated at random to receive either the study drug or a comparison/control product. Patients are unaware of [...]
Last month, the European Medicines Agency (EMA) and European Union healthcare payers came together for the first time to discuss synergies for fostering timely and affordable access to medicines. The meeting took place at the EMA headquarters in London and marked an important first step in a bid to streamline drug availability for EU citizens. [...]
Immunotherapy has become a hot topic in the oncology space over the past decade. For many cancer patients, the traditional treatment options, like chemotherapy, radiation therapy, or targeted therapies like Gleevec or Herceptin, are ineffective in treating their disease. For these patients, immunotherapy, which uses the body’s own immune system to fight cancer, is offering [...]