Priority review is the mechanism used by the United States Food and Drug Administration (FDA) to speed up the review process for medicines that are expected to have a major impact on the treatment of diseases. The priority review voucher program is a program that provides vouchers for priority review use to drug developers as an incentive to develop treatments for drugs that may not be profitable to develop due to smaller pools of patients in need of care.
A review of current priorities is obtained for drugs that treat neglected tropical diseases, rare pediatric diseases, and "medical countertercations" for terrorism. These vouchers can be used for future medicines that can have wider indications to use, but companies are required to pay a fee (about $ 2.8 million) for using vouchers.
When seeking approval for drugs, manufacturers may apply to the FDA for priority review. This is given when medications are meant to treat serious conditions and will "provide a significant increase in safety or effectiveness" over the currently available treatments. Priority review vouchers can be used when drugs do not comply with these requirements, but companies want to speed up the review process.
In 2007, Title XI of the Food and Drug Administration Amendment Act of 2007 created a vaccine priority review program for neglected tropical diseases. This was extended in 2012 by the Food and Drug Administration's Security and Innovation Act to include rare pediatric illnesses. Actions that are built on the tropical disease system and make amendments include shorter notices to the FDA before running the voucher, the appointment system so that at the beginning of the drug development cycle sponsorship may use the possibility of getting vouchers in their judgment of their company, the requirements of marketing plans and marketing reporting, and unlimited voucher redirects. By 2016, medical countermeasures are added into the program.
Video Priority review
Priority review
Prior to approval, any drug marketed in the United States must undergo a detailed FDA review process. In 1992, under the Prescription Drug Usage Fees Act (PDUFA), the FDA approved specific objectives to improve drug review time and created a system of double-review - standard review and priority review.
The priority review setting is given to medicines that offer great advances in treatment, or provide treatment where there is no adequate therapy. The 2002 amendment to the PDUFA stipulated the goal that standard drug application review was conducted within a period of ten months. The FDA's goal of completing a priority review is six months. The priority review status may apply both to drugs used to treat serious illnesses and drugs for less serious illnesses.
The difference between the priority and timing of the standard review is that additional FDA attention and resources will be directed to drugs that have the potential to provide significant advances in treatment. Such progress can be demonstrated by, for example: evidence of increased effectiveness in the treatment, prevention, or diagnosis of the disease; elimination or substantial reduction of drug-limiting drug reactions; documenting an improvement in the patient's willingness or ability to take medication according to the required schedule and dosage; or evidence of safety and effectiveness in new subpopulations, such as children.
Requests for Priority Reviews must be made by drug companies. It does not affect the duration of the clinical trial period. The FDA determines within 45 days of the drug company's request whether the priority or standard review indicates will be set. The designation of drugs as "priorities" does not alter the scientific/medical standards for approval or the quality of evidence required. Safety requirements for priority review are the same as standard reviews.
The amendment can be found on page 150 of the Food and Drug Administration Amendment Act of 2007.
Maps Priority review
Priority review voucher program
The law authorizes the FDA to provide priority review vouchers to approved new drug or biological sponsors who target neglected tropical diseases or rare pediatric diseases. Terms apply to New Drug Applications (NDA), Application of Biological Licenses (BLA) and 505 (b) (2) applications. Vouchers, which can be transferable and available for sale, entitle the carrier to get priority reviews for other products.
According to the Prescription Drug Current Drugs Act, the FDA aims to resolve and act on priority drug reviews within six months instead of the standard ten-month review period. However, the actual FDA review schedule may be longer than the target PDUFA review period, especially for new products that have not previously been approved for any indication. Economists at Duke University, who published this concept in 2006, estimate that priority reviews could cut the FDA assessment process from an average of 18 months down to six months, shortening as much as a full year of time required for the company's drug to reach the market.
The unreal benefit of the voucher is the value made to the company if a faster review gives them the "first mover advantage," which allows the product of the coupon holder to be introduced before a similar and competitive product. By taking advantage of existing market forces, patients in developing countries can have faster access to rescue products that may not be developed. And sponsors of abandoned illness medicine can be rewarded for their innovation
Sponsors must notify the FDA of their intention to use a priority review 90 day voucher prior to shipment. Before Adding Ebola to the FDA Priority FDA Voucher Act Act of 2014, this requirement is 365 days, which is a barrier to the rapid review process, since companies typically do not specify when the drug will be delivered until results from safety studies are available.
Companies can also sell vouchers to other drug companies. So far, priority review vouchers have sold for $ 50-350 million.
Cost
Companies that use vouchers will be required to pay additional user priority review fees to ensure that the FDA can cover costs incurred by the agency for faster review, in addition to fees for standard review of medicines. The cost of additional users also aims to ensure that the new program will not slow the progress of other products awaiting FDA review. Costs have dropped dramatically from over $ 5 million in 2012. For the fiscal year 2018, this fee is $ 2.8 million.
Use of the priority review program
By 2017, fourteen priority review vouchers have been awarded, four for tropical diseases, and ten for rare childhood illnesses. The first priority review voucher was given in 2009 to Novartis for Coartem approval. The next voucher is not awarded until 2012. Other vouchers have been awarded to Janssen's Sirturo, BioMarin Vimizim, miltefosine Knight Therapeutics, dinutuximab United Therapeutics, Asklepion Therapeutics, Xuriden Wellstat Therapeutics, Alexion's Strensiq, Alexion's Kanuma, PaxVax Bermuda Vaxchora, Sarepta Therapeutics's eteplirsen, Ionis Pharmaceuticals's Spinraza, Emflaza Marathon Pharmaceuticals, and BioMarin Brineura.
Vouchers have been used to speed up the approval process for guselkumab, alirocumab, and emtricitabine/rilpivirine/tenofovir alafenamide. The voucher is used by Novartis in the application for canakinumab, but the drug is not approved.
Extensions
Extensions for rare childhood illness
In 2012, President Obama signed the FDA Security Act and the Innovation Act that included Section 908, "The Voucher Award Program of Superior Skin Diseases of the Child". Section 529 extends a coupon program for rare childhood illness, but only on experiments. After the third voucher is awarded, the US Treasury will conduct a study on the effectiveness of the pediatric priority voucher program.
The pediatric voucher program includes changes to the voucher program. First, pediatric care developers can ask the FDA in advance to indicate whether the disease qualifies as a rare childhood illness.
The gift recipient must market the drug within 365 days of approval, or the voucher may be revoked. Within five years of approval, the manufacturer must submit a report containing information on the estimated population in the United States suffering from rare pediatric diseases, the estimated demand in the United States for such rare pediatric disease products, and the rare actual number of such pediatric disease products distributed in the United States.
The Advancing Hope Act of 2016 reauthorized the program until December 31, 2016 and instructed GAO to prepare a report on program effectiveness.
Extensions for ebola virus
In December 2014, the Senate approved a bill that would add the Ebola virus to the Priority Review Voucher List. The bill, S. 2917 - Adds Ebola to the FDA Priority Review Program Voucher , was introduced by Senator Tom Harkin on November 12, 2014. President Obama signed it on December 16, and it became Public 113- 233. Forty-five Senators sponsored the bill (26 Democrats and 19 Republicans). This action also eliminates the difference between tropical disease and pediatric disease vaccination, but allows both to be sold in unlimited quantities and used after a 90-day notice period to the FDA.
At the technical level, S. 2917 adds "Filoviruses" to the priority review list. Ebola virus is a type of Filovirus. According to the Congressional Budget Office, the enactment of the law has no effect on the federal budget.
Extension for medical precautions
The 2016 Senate Medical Initiative Bill's Initiative proposes adding new drug categories to the priority review voucher program. By 2016, it is certain that drug approval for medical countermeasures will be eligible for a priority review voucher. Medical countermeasures are drugs for "preventing or treating the dangers of biological, chemical, radiological or nuclear agents identified as material threats".
Proposed adoption by the European Medicines Agency
Writing in The Lancet, David Ridley and Alfonso Calles SÃÆ'¡nchez apply for a voucher extension to the European Union. The proposed EU voucher will provide a review of priority rules through the European Medicines Agency, as well as pricing and replacement decisions accelerated by EU member states.
Secondary market
According to Forbes journalist Tim Worstall, a priority check voucher emerged as a "lucrative secondary market". In an article in the Wall Street Journal there were concerns about the sale of this voucher that "requires the FDA to shorten its decision deadline to six months from the standard 10 months - potentially giving the company an additional four months of sales vouchers do not guarantee the FDA will approve the drugs.
In July 2014, Regeneron and Sanofi announced that they had purchased a priority review voucher that BioMarin had won for a recent approval of a rare illness drug of $ 67.5 million; vouchers were cut four months from the time of regulatory review to alirocumab and were part of their strategy to defeat Amgen to market with the first approval of PCSK9 inhibitors.
In 2015, a voucher for pediatric cancer drugs developed by United Therapeutics sold for $ 350 million. With this fee, vouchers will be expected to offset the large investments and risks required for the discovery and development of new treatments for neglected diseases. If the time saved from getting priority review is much shorter, however, the voucher value will be much less.
Targeted diseases
Eligible tropical diseases include the following:
A rare disease in children is any disease that primarily affects people under the age of 18 and affects 200,000 or fewer people in the United States.
Medical countermeasures are drugs to be used "in public health emergencies stemming from terrorist attacks with biological, chemical, or radiological/nuclear materials, naturally occurring diseases, or natural disasters."
Limitations
Critics have claimed a number of issues with the priority review program. First, the priority review voucher may be too small or too large to encourage drug development. It may be too small because tropical diseases with tremendous loads can be assumed to get more resources. This may not be the case for rare diseases in children, some drugs developed for the use of children through the expansion of adult drug research for similar conditions. The priority review voucher may be too large, if it rewards research that should have been done, or low value research.
Priority review vouchers may levy an FDA resource tax. To mitigate this, the use of a priority review voucher includes additional costs paid by the manufacturer to the FDA and requires that the voucher holder grant the FDA 90 days notice before using the voucher.
Critics of the FDA have alleged that priority reviews may be unsafe. The priority review should not be confused with accelerated approval or quick path alignment. Priority review does not eliminate safety or efficacy studies or require approval within a certain time frame. It sets a target of 6 instead of 10 months for an FDA review. However, a study in 2008 suggested that new molecular entities approved within two months before the first examination deadline indicate a higher level of post-mortem security problems than drugs approved at other times. Nardinelli and colleagues (2008) of the FDA, however, wrote that they could not replicate findings and findings likely to be driven by HIV-AIDS therapy. After Nardinelli's piece, Carpenter acknowledged some errors in their data set and pointed out errors in FDA and Nardinelli data; Carpenter and colleagues report that the original association between last minute approval and security issues continues to occur.
There are also complaints that priority review vouchers encourage innovation, but do not pay for access to existing therapies. Funding from government or foundations may be needed to buy care for the poor. Aidan Hollis of the University of Calgary has commented that the proposal does not address "access issues, but helps increase incentives through creating distortions in markets in developed countries". This is entirely a separate issue of research promotion that is meant by the priority review program.
News and reactions
Menurut Bill Gates,
"Some of the highest-leveraged jobs the government can do are organize policies and disburse funds in ways that create market incentives for business activities that improve the lives of the poor.Based on a law signed by President Bush last year, every drug company developing new treatments for neglected diseases such as malaria or tuberculosis can get a priority review of the Food and Drug Administration for other products they have created.If you are developing a new drug for malaria, a cholesterol-lowering drug that you can get into the market a year earlier. could be worth hundreds of millions of dollars. "
See also
- FDA's Fast Track Development Program
- Breakthrough Therapy
References
External links
- Priority review voucher page published by one of the Duke's authors of the voucher
- Jack, Andrew (April 30, 2008). "FDA to help research tropical diseases". Financial Times . Ã,
- Hollis, Aidan (April 5, 2005). "Optional Prize for New Medicines for Developing Countries" (PDF) . Delivery: Commission on Intellectual Property Rights, Innovation and Public Health (CIPIH) . World Health Organization. < span> Ã, - a critique of the Priority Review Voucher
- the BVGH information page on the Priority Review Voucher
- SEC. 524. (21 USC Ã,§360n) Text of the amendment
- Duke University's video on vouchers
Source of the article : Wikipedia
0 Comments