Developing new drugs is very expensive and time consuming. Out of the thousands of compounds that are developed only one makes it through the FDA approval process. It is because of this that it costs the drug companies so much to get a drug on the market. The amount of time that it takes for a drug to be marketed can be discouraging to many companies from entering the drug development field. It still takes between 9 years and 13 years for a drug to be able to hit the market and this is with the view that the FDA has accelerated the process of reviewing the drug with the introduction of the breakthrough therapy designation. This can cut the development by several years by allowing the FDA to introduce promising drugs to the market based on promising phase 2 clinical trial results.
With all these challenges the rate of new drugs approved in 2013 was less than those approved in 2012. But the new drugs approved in the first quarter of 2014 shows that the FDA is committed to expedite approving innovative drugs.
With the technological advancements, it seems that specialty pharmaceuticals are leading the drug pipeline, even though the number of patients benefiting from these drugs remains a small percentage of the population. In 2013 60% of the drugs approved by the FDA were specialty drugs and it is estimated that by the end of the decade more than 50% of the drugs covered under the pharmacy benefit will be specialty drugs.
With the focus on the role of genetics and the identifications of bio markers, targeted drugs which use biotechnology in their development are being used to treat patients and this would justify the their use even with the high cost of these drugs. The cost of these specialty drugs is proving to be a challenge even with the growing competition in the specialty drug field. Though biosimilars are expected to reduce the price of these specialty drugs by about 20%, they are yet to be approved by the FDA and it is estimated that it will take another year or two before the first biosimilar drugs are approved by the FDA.
It is estimated that by 2018 the overall spending on specialty drugs will far exceed the total spending on traditional drugs even though the specialty drugs are used by a very small percentage of the total patients. Traditional medicine drugs are though surprisingly being developed in a large number for many clinical categories.
Orphan drugs are those that are used to treat rare diseases and though there are over 25 million people in this country suffering from rare diseases, one of these drugs only benefits around 200,000 people. The FDA has used its priority review to expedite its approval of these drugs so that patients could avail of their benefits. The growth of these orphan drugs is more than 20% annually and even though spending on these drugs is a total of 6% more than 30% of these drugs are considered to be Blockbusters and can cost thousands of dollars monthly.
As the competition in the specialty drugs grows so are the options for these drugs and the methods of delivering the drugs. The number of specialty drugs in the FDA pipeline is booming with thousands of clinical trials being conducted for new therapies and new mechanisms of actions targeting many different disease states which include many new therapies for rare diseases. The trend of delivering these specialty drugs is moving for them to be taken orally or subcutaneously. This helps to save time for the provider of these drugs and may help in reducing costs. On the other hand by putting the patient in charge of taking the medication can lead to non adherence of timely delivery.
The specialty drug categories in 2014 include inflammatory conditions, MS, cancer, HIV, Aids, and hepatitis C virus infections. These specialty drug categories also include a new class of drugs for the treatment of diabetes, lipid disorders and also drugs for improving the metabolism for patients who are obese or have heart conditions.
The list of specialty drugs being approved or which are in the pipeline are being developed to treat conditions such as Pulmonary arterial hypertension, inflammatory disorders such as arthritis, MS as well as infectious diseases, cystic fibrosis and cancer. The growth in these specialty drugs is going to continue as some of the new therapies are reaching new successes in treatment of these diseases.
Looking at the current drug pipeline it is safe to assume that treatment of cancer and diabetes will remain the top diseases that would be targeted for the development of the specialty drugs. Though these new drugs help patients in the treatment of their diseases there are new challenges that will have to be overcome. The most important being how the patients are going to pay for these expensive medications.
Another challenge for the established pharmaceutical companies is the growing competition from small biotechnological companies who are entering the specialty drug development arena. In the face of this competition innovation seems to be the key for success in the specialty drug pipeline.
Dr. Trydzenskaya has 5+ years of Pharma and Life Sciences experience: Clinical, Medical Affairs, Medical Information, Medical Communication, Publication, Program Management. She has received multiple Young Investigator Awards for the highest ranked clinical research in Immunology. She has led multidisciplinary clinical studies in the following areas: Transplant, Nephrology, Oncology, Immunology, Hematopoietic Stem Cell Transplantation.