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Nkechi Azie, MD, MBA
Sr. Medical Director
Astellas Pharma US Inc.,
Sukirti Bagal MD, MPH
Director, Rare Disease Specialist Medical Affairs and Clinical Development
New York, NY
Global Genes | RARE Project
Dana Point, CaliforniaJonathan Bui, MD, PhD
University of California San Diego
La Jolla, CA
Stephen Eck, MD, PhD
Vice President & Global Head
Oncology Medical Sciences
Astellas Pharma Global Development, Inc.
Phyllis Frosst, PhD
Director of Collaborative Operations
NIH Center for Translational Therapeutics, NIH
Richard Haas, MD
Professor of Neurosciences and Pediatrics
University of California San Diego Medical Center
La Jolla, CA
Marlene E. Haffner, MD, MPH
Health Policy and Global Drug Development Consultant
Joanna C. Jen, MD, PhD
Associate Professor of Neurology
David Geffen School of Medicine at UCLA
Los Angeles, CA
Cathleen Lutz, PhD
Director, Mouse Resources
Rare and Orphan Disease Center – The Jackson Laboratory
Bar Harbor, ME
David Lynch, MD, PhD
Professor, Neurology and Pediatrics
The Childrenâ€™s Hospital of Philadelphia
Anne Pariser, MD
Reed E. Pyeritz, MD, PhD
Vice-Chair for Academic Affairs, Dept. of Med.
Director, Center for the Integration of Genetic Healthcare Technologies
Prof. of Medicine & Genetics, Raymond and Ruth Perelman School of Med at the Univ of Penn
Margaret Ragni, MD
Professor of Medicine, Division of Hematology/Oncology
Director, Hemophilia Center of Western PA
Professor Aly Rashid, MD
Medical Director LLR Cluster NHS, Leicester City
Leicester, Northamptonshire, United Kingdom
Peter L. Saltonstall
President and CEO
National Organization for Rare Disorders
Dhruv Sareen, Ph.D
Regenerative Medicine Institute
Director, Induced Pluripotent Stem Cell (iPSC) Core Facility
Research Scientist I-Faculty, Department of Biomedical Sciences
Cedars-Sinai Medical Center
Los Angeles, CA
Sharon F. Terry, MA
President and CEO
Barbara Wuebbels, RN, MS
Associate Director Patient Advocacy and Investigator Relations
BioMarin Pharmaceutical Inc.
WHY DOES USE OF EXPEDITED APPROVAL METHODS VARY SO
GREATLY? AN ANALYSIS OF ORPHAN DRUGS APPROVED IN 2014
John Ansell, MA, and Glenn Tillotson, PhD
TranScrip Partners, Reading, United Kingdom
The US Food and Drug AdministraTIon (FDA) now provides 4 different methods for expediting approval of new drugs. This article examines how companies employed these options to win FDA approval last year for 17 orphan drugs and examines the diverse issues encountered. For the first time, in 2014, the Priority Review option was used by all companies whose orphan drugs were successfully registered. This represented a sharp increase in its usage, given that just under half of all companies gaining approval for orphan products had used this opTIon in both 2012 and 2013. In contrast, since 2013 there has been little change in the extent to which the other 3 methods for expedited approval have been used for orphan products. Other than the universal use of the Priority Review option, we find remarkable variation in the options taken for expedited approval for orphan drugs in 2014: no less than 7 different combinations of the 4 methods were taken. Consequently, we believe this indicates that the options are neither fully understood by orphan drug companies or are not being used optimally by them—with this marked variation relating less to a correct analysis of likelihood of qualification than to companies’ internal attitudes toward the ease of proceeding via each method. It is also unclear why companies do not avail themselves more of the four options. Limited resources or inexperience with the different methods could well be playing a role. We suggest that there is now a case for granting automatic Priority Review status for all drugs granted orphan status and the scope for rationalizing, unifying, and thereby simplifying the 4 methods. This would benefit developers of all types of drugs, but particularly of orphans, as well as reduce the impact of increasing usage of FDA resources.
Orphan drug status applies to drugs that are developed for a specific condition or disease (there are more than 7000 recognized rare diseases) affecting fewer than 200,000 Americans. In the United States, the Office of Orphan Products Development administers the major provisions of the Orphan Drug Act, which provide incentives for sponsors to develop products for rare diseases. The Orphan Drug Act has been very successful—more than 400 drugs and biological products for rare diseases have been brought to market since 1983. In contrast, the decade prior to 1983 saw fewer than 10 such products reach the market. This regulatory status has several benefits for the sponsor, including marketing the drug without competition for 7 years and possible qualification for clinical trial tax incentives. Awareness of orphan
conditions has increased significantly over the past decade for a number of reasons, not least of which is the response by the US Food and Drug Administration (FDA) to enhancing or simplifying the process for approval. The European Union (EU) has enacted similar legislation, Regulation (EC) No 141/2000, in which pharmaceuticals developed to treat rare diseases are referred to as “orphan medicinal products.” The EU’s definition of an orphan condition is broader than that of the FDA’s, in that it also covers some tropical diseases that are primarily found in developing nations. In Europe the medicine must treat, prevent, or diagnose a life‐threatening or chronically debilitating condition, and this condition must affect fewer than 5 in 10,000 people in the EU. Alternatively, if a condition affects more than 5 in 10,000 people in the EU, it may still be considered for orphan designation. Notably, for a treatment to qualify for orphan designation, there must be no existing approved treatments for the indicated condition, or if there are, the product in question must offer significant improvements over the other options. Orphan drug status granted by the European Commission gives marketing exclusivity in the EU for 10 years after approval. The EU’s legislation is administered by the Committee on Orphan Medicinal Products of the European Medicines Agency. In 2014, seventeen orphan drugs were approved for clinical use in Europe, a remarkable increase compared with previous periods.2
THE 4 METHODS FOR EXPEDITED APPROVAL IN THE UNITED STATES
Orphan drug status, in itself, provides various benefits to drug development companies that include measures to expedite approval. But, in addition, as for all drugs, orphans can qualify for any of the FDA’s 4 methods of expedited approval. This article examines how companies with orphan drugs approved in 2014 are availing themselves of these options. The box shows the 4 different methods of expedited approval available to drug developers. The methods have much in common; in particular, all 4 methods target therapies intended to treat a serious aspect of a condition or a serious condition. Also, all require that a new compound address an unmet medical need.3
ANALYSIS OF EXPEDITED APPROVAL METHODS FOR ORPHAN DRUGS APPROVED IN 2014
In 2014 the FDA approved 41 new drug applications, of which 17 were orphans, all from different companies.4 Details are shown in the table. The 17 orphan drugs dominated successful usage of the 4 different methods for expedited approval that have evolved: Variety of Options Taken for Orphan Products All 17 orphan drugs approved in 2014 had been granted Priority Review status (compared with only a third of non ‐orphan products that were granted this status). This is a recent development: in both 2012 and 2013, just under half of all orphan drugs approved (6/13 and 4/9, respectively) were granted this status. There was also considerable, though lesser, use of the other 3 options for orphan drugs, as shown in Figure 1:10 received Fast Track status, 8 had Accelerated status, and 7 were designated as Breakthrough. For each of these methods, the proportions of orphan drugs approved in 2014 were similar to those in the 2013 cohort. The extent to which companies used different options varied enormously. Figure 2 depicts the distribution of use for each of the 4 methods. Only 2 companies used all 4 methods, 7 used 3 methods, and 5 used 2 methods. Three companies used just 1 method.
Underlining the degree of variety of approaches used was the remarkable diversity in combinations of methods used. These are shown in Figure 3. As mentioned previously, all 4 options were used by just
2 of the companies whose orphan drugs, Opdivo (nivolumab) and Zydelig (idelalisib), were approved in 2014. As Figure 3 shows, no less than 7 combinations of the 4 possible expedited approval methods were used. All 3 possible combinations of the 4 options were used, and 2 types of combinations of just 2 of them. However, the only method used alone was Priority Review. Thus, there was a very wide variety of patterns of usage of the 4 methods. Although the most popular of the 7 different combinations was Priority + Accelerated reviews, this was still only used for 4 of the 17 orphan products. It is interesting that whereas the Breakthrough process is envisaged by the FDA as only to be granted to a subset of those products eligible for Fast Track status that especially merit it, 3 products in 2014—Blincyto (blinatumomab), Keytruda (pembrolizumab), and Zykadia (ceritinib)—gained the former status without being granted the laƩer. Perhaps the companies concerned just did not apply for it. We suggest that some companies might have considered that Breakthrough status superseded or gave them no advantage over Fast Track status. Indeed, the FDA has even expressly stated that new drugs receiving Breakthrough therapy designaTIon are eligible for all of the features of the Fast Track designaTIon.2 On the other hand, 4 orphan drugs approved in 2014 did have Fast Track as well as Breakthrough status. These products were Opdivo, Zydelig, Ofev (nintedanib), and Esbriet (pirfenidone).
Current expedited approval methods employed by companies with successfully registered orphan drugs vary greatly. It is not clear why such a degree of difference exists. Also, it appears to us, on examining the data presented, that some companies might have benefited if they had applied for more of the 4 options. Kepplinger3 and Jae5 have thoroughly reviewed this perspective. We conclude that it is unlikely that the 4 different methods are being used by companies optimally. A further factor could conceivably be contributing to this diverse usage. The summary criteria for the 4 expedited approval methods have much in common; however, it could be that the FDA is applying different degrees of stringency to similar approval criteria when it addresses eligibility for the different methods.6 The areas of overlap in assessing eligibility among the 4 methods would appear to offer the FDA opportunities to rationalize, unify, and thereby simplify these expedited approval systems—not just for orphans but for all types of drugs. It has already been proposed recently that the Breakthrough Therapy and Accelerated Approval processes be merged,4, 5 and we believe this could be a sensible first step. Another, specific area for resource savings relates to Priority Review. In 2014, for the first time, all 17 orphan drugs approved had been granted Priority Review status. We consider that there is now a case for waiving the Priority Review assessment process for all drugs granted orphan status and suggest that they should be granted Priority Review status automatically. A more unified system that reduces the current duplication of assessment processes would reduce resources required by the FDA, which is important as use of the 4 expedited approval methods is increasing. It should at the same TIme be advantageous to companies making submissions, particularly those developing orphan drugs, whose resources—personnel, time, and financial—are often particularly limited.
We wish to thank our colleagues at TranScrip Partners, Paul Branthwaite, Dr. Bob Milsted, and Louise Whitley, for their helpful comments, as well as Nicolette Theriault for graphics preparation.
CONFLICT OF INTEREST STATEMENT
JA and GT are both employees of Transcrip Partners; they do not have any financial relationship with the companies or products mentioned in this article.
Priority Review: Just over two‐thirds (17/25) of all Priority Review approvals were orphans. Fast Track: Just over half (10/17) of all Fast Track approvals were of orphan drugs. Accelerated Approval: All 8 of the Accelerated approvals were orphans. Breakthrough Therapy: Over two‐thirds (7/9) of all Breakthrough approvals were orphans.
The 4 Different Expedited Approval Methods
Priority Review status is granted when the Center for Drug Evaluation and Research determines that a drug could provide a significant advance in medical care; it sets a target to review the drug within 6 months instead of the standard 10 months. There has to be potential for significant improvement in safety or efficacy. Unlike the other 3 methods, the US Food and Drug Administration (FDA) classifies all original new drug applications (NDAs) and biologics license applications (BLAs) for Priority Review whether or not the sponsor requests Priority Review. Fast Track can speed new drug development and review, for instance, by increasing the level of communication FDA allocates to drug developers and by enabling CDER to review portions of a drug application ahead of the submission of the complete application. An unmet need has to be demonstrated to qualify. Accelerated Approval allows early approval of a drug for a serious or life‐threatening illness that offers a benefit over current treatments. To qualify, there must be a likelihood of meaningful advantage over available therapies. Breakthrough Therapy designaTIon includes all of the Fast Track program features as well as more intensive FDA guidance on an efficient drug development program. It is granted for a subset of fast‐tracked products. Breakthrough status is designed to help shorten the development time of a promising new therapy. To gain this status, preliminary clinical evidence must indicate a substantial improvement over available therapies on a clinically significant endpoint. This method first appeared as an option taken for orphan drugs approved in 2013.
Table 1. Orphan drugs approved by the FDA in 2014
|Brand||Name||Generic Name Indication Company Fast||Track||Breakthrough||Priority||Review||Accelerated||Approval|
|Blincyto||blinatumomab||Acute lymphoblastic leukemia||Amgen|
|Cyramza||ramucirumab||Non‐small cell lung cancer||Eli Lilly|
|Esbriet||pirfenidone||Idiopathic pulmonary fibrosis||Roche|
|Keytruda||pembroli‐zumab||Melanoma||Merck & Co|
|Northera||droxidopa||Neurogenic orthostatic hypotension||Chelsea|
|Ofev||nintedanib||Idiopathic pulmonary fibrosis||Boehringer Ingelheim|
|Opdivo||nivolumab||Metastatic melanoma||Bristol Myers Squibb|
|Sylvant||siltuximab||Multicentric Castleman’s disease||Janssen Biotech|
|Vimizim||elosulfase alfa||Mucopolysaccharidosis type IVA||BioMarin|
|Zydelig||idelalisib||B‐cell blood cancer||Gilead|
|Zykadia||ceritinib||Non‐small cell lung cancer||Novartis|
Chronic lymphocyTIc leukemia, follicular B‐cell non‐Hodgkin lymphoma and relapsed small lymphocytic lymphoma. Source: FDA3 and other sources.
- Developing products for rare diseases & condiTIons. US Food and Drug AdministraTIon Web site. http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesCondiTIons/ucm2005525.htm. Updated February 26, 2015. Accessed June 3, 2015.
- Orphan drugs in the EU: a record‐breaking year. Regulatory Affairs Professional Society Web site. http://www.raps.org/Regulatory‐Focus/News/2015/01/13/21063/Orphan‐Drugs‐in‐the‐EU‐A‐Record‐Breaking‐Year/. Accessed June 3, 2015.
- Kepplinger EE. FDA’s expedited approval mechanisms for new drugs products. Biotechnol Law Rep. 2015;34 (1):15‐37.
- Clinical development. TotalOrphanDrugs Web site. http://www.orphan‐drugs.org/category/clinical‐ development/ Accessed June 3, 2015.
- Jae VS. Simplifying FDASIA: the “fast track” to expedited drug approval efficiency. Admin Law Rev. 2014;66(1):173‐198.
- New drugs at FDA: CDER’s new molecular entities and new therapeutic biological products. US Food
and Drug Administration Web site. http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovaTIon/ucm20025676.htm. Updated April 14, 2015. Accessed June 3, 2015.
The Fundamental Diseases Partnership
Press Release: Findacure launches Online Toolkit for Rare Disease Patients For immediate release.
UK charity Findacure is pleased to announce that this week it has launched an innovative online toolkit- a series of interactive modules, which are aimed at rare disease patient groups to help them develop the skills and knowledge they need to grow their organisations. The toolkit has been built by expert clinicians, fundraisers, drug developers and academics. The modules cover a broad range of topics pertaining to rare disease patient advocacy; there are guides on fundraising, on patient recruitment, on getting involved in clinical trials, on working with academia and on effective PR and communications. We would like to invite rare disease patients to sign up for use of this tookit, which can be accessed at findacure.gather.io/d/sign-up Nick Sireau, the founder of Findacure and father of two boys with ultra rare disease Alkaptonuria (or ‘Black Bone Disease’) said “There are around 7,000 rare diseases and only around half of them have patient groups dedicated to helping them. The remainder of them are isolated, with little hope of cures or little hope of a future. Those that do have patient groups are often ‘kitchen table groups’, set up by patients, their friends and families, desperately striving to make a difference. That’s why we’ve launched this online toolkit for rare disease patient groups – it will provide a range of resources where rare disease patients can go if they’re in need of advice.” Currently, rare disease patient groups are chronically underfunded. They do not usually have access to the skills required to move forward with their aims of funding research and finding treatments. Findacure was set up in 2012, following founder Nick Sireau’s experience of running a patient group for the rare disease affecting his two sons, Alkaptonuria. He was faced with an uphill struggle of raising funds, setting up research, and learning how to run clinical trials and support patients, and realised there was a huge need for practical, hands on advice for patient groups to achieve their aims. Through this online toolkit, Findacure aim to do exactly that.
International research team develops new methods for therapy evaluation in rare diseases
Aachen, 02.09.2013 – In the EU diseases that affect on average not more than 5 in 10,000 people are called rare. Worldwide, more than 7,000 such rare diseases are registered. The resulting groups of patients who are affected by a specific rare disease can be very small. A significant number of diseases occur in only 1-2 patients (e.g. obesity due to prohormone convertase-I deficiency), Orphanet (2012). In the EU 6 to 8 % of the population – that is, from 27 to 36 million people – suffer from at least one of the 5,000 to 8,000 different rare diseases. According to information from the European Society of paediatric oncology, 75% of rare diseases affect children, of whom 30% die before reaching their fifth birthday.
The ability of conventional statistical methods to evaluate new therapeutic approaches for any given rare diseases is limited due to the small number of patients concerned. This means that established statistical approaches to demonstrate the efficacy and safety of therapies may fail in this situation. Thus, there is an urgent need not only to develop new therapeutic approaches to treat diseases, but also to develop new statistical methods to establish which approaches work. The aim is to use and bring together all possible sources of information in order to optimize the process. This is the point of departure for IDEAL (“Integrated Design and Analysis of small population group trials”) research project. The project will explore new methods for design and analysis of clinical studies and to integrate and and synthesize these into an effective strategy, so that the efficiency of clinical trials evaluating therapies for rare diseases can be significantly increased. An international researcher-team under the coordination of Professor Ralf-Dieter Hilgers of the Uniklinik RWTH Aachen, will jointly develop new designs and sophisticated analysis methods for the evaluation of therapies for rare diseases, supported by the EU Projektmanagement Office der RWTH Aachen. The research is funded by the 7.th Framework Programme of the European Union (FP7-HEALTH2013-INNOVATION-1, No. 602 552) with 3 million. €. The consortium consist of Professor Ralf-Dieter Hilgers, Uniklinik RWTH Aachen, Professor Holger Dette, Ruhr University Bochum, Franz König, Medical University of Vienna, Professor France Mentré, Institut National de la Santé et de la Recherche Medicale in Paris, Professor Stephen Senn, Centre de Recherche Public de la Santé Luxembourg, Professor Mats Karlson, Uppsala University, Uppsala, Professor Malgorzata Bogdan, Polytechnika Wroclawska, Warsaw, Dr. Carl-Fredrik Burman, Chalmers Tekniska Hoekskola AB, Goeteburg, Professor Geert Molenberghs, University Hasselt, Hasselt and Professor Christoph Male, Medical University of Vienna. The research programme is divided into 11 work packages. The work packages focus on the assessment of randomization, the extrapolation of dose-response information, the study of adaptive trial designs, the development of optimal experimental designs in mixed models, as well as pharmacokinetic and individualized designs, simulation of clinical studies, the involvement and identification of genetic factors, decision-theoretic considerations, as well as the evaluation of biomarkers.
Two work packages provide support for project management and the dissemination of results. The IDEAL project is accompanied by an advisory board of international experts with different professional backgrounds, representing both patients’ interests, the views of the pharmaceutical industry as well as clinical and regulatory aspects. The ability to use mathematical and statistical techniques for the evaluation of new treatments where standard methods fail to be successful is a challenge and motivation for all of us. We all look forward to this exciting collaboration.
Über die Uniklinik RWTH Aachen (AöR)
Die Uniklinik RWTH Aachen verbindet als Supramaximalversorger patientenorientierte Medizin und Pflege, Lehre sowie Forschung auf internationalem Niveau. Mit 34 Fachkliniken, 25 Instituten und fünf fachübergreifenden Einheiten deckt die Uniklinik das gesamte medizinische Spektrum ab. Hervorragend qualifizierte Teams aus Ärzten, Pflegern und Wissenschaftlern setzen sich kompetent für die Gesundheit der Patienten ein. Die Bündelung von Krankenversorgung, Forschung und Lehre in einem Zentralgebäude bietet beste Voraussetzungen für einen intensive interdisziplinären Austausch und eine enge klinische und wissenschaftliche Vernetzung. Rund 6.000 Mitarbeiterinnen und Mitarbeiter sorgen für patientenorientierte Medizin und eine Pflege nach anerkannten Qualitätsstandards. Die Uniklinik versorgt mit 1.240 Betten rund 47.000 stationäre und 153.000 ambulante Fälle im Jahr. Weitere Informationen bei: Universitätsklinikum Aachen (AöR) Prof. Dr. rer. nat. Ralf-Dieter Hilgers Institut für Medizinische Statistik
An Open Access Journal
New Methods for Evaluation of Therapy
By: Rare Disorders
Back to News
Aachen, 9/2/2013-In the EU diseases that affect 5 in 10,000 people or less are defined as rare. Worldwide more than 7,000 such rare diseases are registered. The resulting groups of patients who are affected by a specific rare disease can be very small. The ability of conventional statistical methods to evaluate new therapeutic approaches for any given rare disease is limited due to the small number of patients. Thus, there is an urgent need not only to develop new therapeutic approaches to treat diseases, but also to develop new statistical methods to establish which approaches work.
The Kids Cancer Centre (KCC) is supported by many generous community, corporate and family groups. The work the Centre undertakes would not be possible without the dedication of these groups; including the following major supporters.
Sydney Children’s Hospital Foundation
SCHF supports the Kids Cancer Centre (KCC) so they are able to provide essential fellowship positions, nursing staff, play therapy, purchase new equipment and conduct vital research to help fight the battle against childhood cancer.
How to get involved
Find out how you can help!
How to Donate
There are a number of different ways to donate and make a real difference to the Hospital. Donations will be directed to the Kids Cancer Fund.
How to Donate
There are a number of different ways to donate and make a real difference to the Hospital. You can make an online donation via Sydney Children’s Hospital Foundation who support the Kids Cancer Centre.
Click here to make an online donation.
How to get involved
The support the Foundation provides to the KCC would not be possible without the help of the community.
Each year we run a number of campaigns and events, from the Gold Telethon in June to the Little Lights Appeal at Christmas, which you can become a part of, either by attending or donating. You can direct all money you raise to the Kids Cancer Fund at SCHF.
You can also run your own fundraising event, such as a bake sale, world record attempt or charity dinner. If this is something you are interested in, please contact us for more information and we can help you to create a great event and raise as much money as possible.
Other ways to help include:
One-off or regular donations
Providing pro bono goods or services
Placing a collection tin at your retail outlet or organisation
Leaving a gift in your Will
Funds will be directed to the Kids Cancer Fund at SCHF, a dedicated fund to support the KCC’s needs.
Sydney Children’s Hospital Foundation
The Sydney Children’s Hospital Foundation (SCHF) is the principal fundraising body for Sydney Children’s Hospital, Randwick (SCH). Our core purpose is to provide significant financial assistance for the support of equipment, research and clinical assistance at SCH.
The SCHF supports the Kids Cancer Centre (KCC) so they are able to provide essential fellowship positions, nursing staff, play therapy, purchase new equipment and conduct vital research to help fight the battle against childhood cancer. Donations are directed to the Kids Cancer Fund, a dedicated fund created by SCHF to support the KCC’s needs.
Community support has enabled the opening of the Sydney Cord and Marrow Transplant Facility, renovations to the Day Cancer Ward, funding for social workers, pharmacists and research initiatives – to name a few.
Community support through SCHF is crucial to provide children with cancer the best possible care and to help the Hospital reach its goal of curing all children with cancer.