Orphan Drug Development Research Articles

G.P.284 - The TREAT-NMD Advisory Committee for Therapeutics (TACT): An innovative de-risking model to foster orphan drug development

G.P.284 - The TREAT-NMD Advisory Committee for Therapeutics (TACT): An innovative de-risking model to foster orphan drug development

The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development.

Despite multiple publications on potential therapies for neuromuscular diseases (NMD) in cell and animal models only a handful reach clinical trials. The ability to prioritise drug development according to objective criteria is particularly critical in rare diseases with large unmet needs and a limited numbers of patients who can be enrolled into clinical trials. TREAT-NMD Advisory Committee for Therapeutics (TACT) was established to provide independent and objective guidance on the preclinical and development pathway of potential therapies (whether novel or repurposed) for NMD.We present our experience in the establishment and operation of the TACT. TACT provides a unique resource of recognized experts from multiple disciplines. The goal of each TACT review is to help the sponsor to position the candidate compound along a realistic and well-informed plan to clinical trials, and eventual registration. The reviews and subsequent recommendations are focused on generating meaningful and rigorous data that can enable clear go/no-go decisions and facilitate longer term funding or partnering opportunities. The review process thereby acts to comment on viability, de-risking the process of proceeding on a development programme.To date TACT has held 10 review meeting and reviewed 29 program applications in several rare neuromuscular diseases: Of the 29 programs reviewed, 19 were from industry and 10 were from academia; 15 were for novel compounds and 14 were for repurposed drugs; 16 were small molecules and 13 were biologics; 14 were preclinical stage applications and 15 were clinical stage applications. 3 had received Orphan drug designation from European Medicines Agency and 3 from Food and Drug Administration. A number of recurrent themes emerged over the course of the reviews and we found that applicants frequently require advice and education on issues concerned with preclinical standard operating procedures, interactions with regulatory agencies, formulation, repurposing, clinical trial design, manufacturing and ethics.Over the 5 years since its establishment TACT has amassed a body of experience that can be extrapolated to other groups of rare diseases to improve the community’s chances of successfully bringing new rare disease drugs to registration and ultimately to market.

Orphan drug incentives in the pharmacogenomic context: policy responses in the US and Canada.

Advances in pharmacogenomic research and increasing industry interest in personalized medicine have important implications for the way that orphan drug policies are interpreted and applied. Concerns have been raised about the potential impact of pharmacogenomics and new genomic technologies on our understanding of how disease categories are delineated, and subsequently, how the concept of rare disease should be defined for the purposes of orphan drug policies. This article considers whether orphan drug legislation can be drafted in a way that will maximize benefits and minimize concerns relating to the impact of pharmacogenomics on orphan drug research and development. After reviewing the issues that may arise at the intersection of orphan drug policies and pharmacogenomics, this article will discuss the potential impact of pharmacogenomics at two critical points: orphan designation and approval of the drug product. At each of these points, the relevant aspects of current US orphan drug legislation are examined, focusing on the extent to which recent amendments may address concerns that have been raised previously. This analysis will then provide the foundation for a critical review and recommendations regarding the proposed new Canadian orphan drug framework.

Pressure for drug development in lysosomal storage disorders - a quantitative analysis thirty years beyond the US orphan drug act.

BackgroundLysosomal storage disorders are a heterogeneous group of approximately 50 monogenically inherited orphan conditions. A defect leads to the storage of complex molecules in the lysosome, and patients develop a complex multisystemic phenotype of high morbidity often associated with premature death. More than 30 years ago the Orphan Drug Act of 1983 passed the United States legislation intended to facilitate the development of drugs for rare disorders.We directed our efforts in assessing which lysosomal diseases had drug development pressure and what distinguished those with successful development and approvals from diseases not treated or without orphan drug designation.MethodsAnalysis of the FDA database for orphan drug designations through descriptive and comparative statistics.ResultsBetween 1983 and 2013, fourteen drugs for seven conditions received FDA approval. Overall, orphan drug status was designated 70 times for 20 conditions. Approved therapies were enzyme replacement therapies (N = 10), substrate reduction therapies (N = 1), small molecules facilitating lysosomal substrate transportation (N = 3). FDA approval was significantly associated with a disease prevalence higher than 0.5/100,000 (p = 0.00742) and clinical development programs that did not require a primary neurological endpoint (p = 0.00059). Orphan drug status was designated for enzymes, modified enzymes, fusion proteins, chemical chaperones, small molecules leading to substrate reduction, or facilitating subcellular substrate transport, stem cells as well as gene therapies.ConclusionsDrug development focused on more common diseases. Primarily neurological diseases were neglected. Small clinical trials with either somatic or biomarker endpoints were successful. Enzyme replacement therapy was the most successful technology. Four factors played a key role in successful orphan drug development or orphan drug designations: 1) prevalence of disease 2) endpoints 3) regulatory precedent, and 4) technology platform. Successful development seeded further innovation.

Delayed access to treatments for rare diseases: who's to blame?

The development and commercialization of drugs for rare diseases, termed 'orphan drugs', has historically been economically unattractive. However, because of the introduction of legislation that provides financial and regulatory incentives for the development of orphan drugs, new developments are making their way through the regulatory approval processes. Unfortunately, delays in availability of new drugs for treating rare disease continue to persist. This paper reviews the approach of several regulatory jurisdictions to orphan drugs in an effort to determine their relative effectiveness in providing patient access. Generally speaking, regulatory authorities across jurisdictions have recognized the need to enhance timely access to safe, effective treatment for patients with rare diseases and have been able to shift the approval timelines for access to new care. The greater impediment to orphan drug access appears to be funding, particularly in publicly sponsored health-care systems. Redundancies in federal and provincial reviews of orphan drugs can result in significant delays in access to new drugs. Clearly, more must be done to accelerate access to the treatments so desperately needed by patients. Public payers must be held accountable for their process and decisions--especially for rare disease therapies.

Developing a patient-directed policy framework for managing orphan and ultra-orphan drugs throughout their lifecycle.

IntroductionPolicy decisions related to orphan and ultra-orphan drugs challenge traditional decision-making processes and often frustrate those affected by them. In general, these drugs are associated with significant uncertainties around clinical benefit, ‘value for money’, affordability, and ‘adoption/diffusion’, all of which arise from a lack of available high-quality evidence. Increasingly, patients with rare diseases and their families are looking for opportunities to contribute to initiatives aimed at reducing these uncertainties. Therefore, a policy framework for guiding their involvement is needed to optimize the impact of any evidence generated.ObjectivesThe aims of this study were (1) to explore opportunities for patient involvement in reducing decision uncertainties throughout the lifecycle of orphan and ultra-orphan drugs from the perspectives of patients within the Canadian rare disease community; and (2) to develop a policy framework for patient input that maximizes the impact of their involvement on decision uncertainties around orphan and ultra-orphan drugs.MethodsTwo one-day conferences and four workshops involving patients and/or families from rare disease communities in Canada were held to discuss issues around orphan and ultra-orphan drug development, access, and coverage, and identify opportunities for patient input to reduce related decision uncertainties. Their feedback and the findings from a recent literature review on patient involvement in rare diseases were combined into a draft policy framework based upon Kingdon’s multiple streams model of decision making. The framework was presented to a group of patients and other stakeholders, including providers, pharmaceutical drug plan managers, and industry representatives, and then revised accordingly.ResultsPatients and family members/caregivers identified tangible ways of contributing to the generation of information at all stages of the drug lifecycle. However, the proximity of that information to the reduction of a specific decision uncertainty varied. While the scope of possible ways mentioned was less broad when compared with the findings of the literature review, the focus was similar—capturing the clinical benefit of an orphan or ultra-orphan drug. A policy framework comprising three stages, each with a key question and corresponding set of sub-questions to be asked by patients, was developed. The three main sequential questions were as follows. (1) What uncertainties need to be addressed? (2) What roles should patients play? (3) Is each role feasible?ConclusionsReducing decision uncertainties around orphan and ultra-orphan drugs requires a policy framework that explicates when and what type of information needs to be generated, and recognizes the role of patients as important sources of such information throughout the lifecycle of these drugs.

Innovative drugs at the 21th century

Currently, new drugs have being developed with a conceptually new research design based on human genome findings. Mainly, new drugs are intend ed for treatment of chronic noninfectious diseases, primarily, cardiovascular diseases, malignancies and respiratory diseases. One of the fundamental research directions is searching for target molecules including lipid metabolism regulators, anticoagulants, fibrinolytics, and monoclonal antibodies. The former are considered as target therapy in cardiology, dermatology, oncology, rheumatology, allergology, pulmonology, ophthalmology, etc. Another important direction is development of orphan drugs. Further basic knowledge is needed for development of new drug generations.

The Budget Impact of Orphan Drugs in the U.S.: A 2007-2013 MIDAS Sales Data Analysis

Introduction: The Orphan Drug Act (ODA) of 1983 established incentives for the development of drugs that treat rare or orphan diseases. The ODA targets therapies for diseases affecting fewer than 200,000 patients in the US. As a consequence, orphan drug development was stimulated and therapies have been developed to treat a variety of previously underserved conditions including acute myeloid leukemia, chronic lymphocytic leukemia, multiple myeloma, myelodysplastic syndromes, Gaucher disease and Hunter syndrome. Between 1973 to 1983, prior to the ODA, less than 10 drugs for orphan conditions were available to patients, while in 2013 the FDA website listed more than 400 approved orphan drugs (this includes both new molecular entities and supplemental applications/indications). Due to the perceived high therapy costs and therefore potential impact on payer’s drug budgets, orphan drugs are facing increasing scrutiny from payers. The objective of this study was to estimate the total annual expenditures on orphan drugs in the US from 2007 to 2013, and to evaluate orphan drug expenditures as a percentage of total pharmaceutical expenditures.Methods: A list of drugs which have been approved by the FDA and given orphan status in the US since the establishment of the ODA through the end of 2013 was generated from the US FDA Orphan Drug Product designation website (N=366 unique products). Only branded products (N=357) were considered in this analysis. The IMS MIDAS database, the gold standard for audited biopharmaceutical sales, was used to assess orphan drug and total drug expenditures. The MIDAS database integrates and extends the IMS National Prescription Audits, which detail estimated sales by product and therapy class through retail and non-retail channels at the unit of the product pack level. The analysis focused on orphan drugs with expenditures measured between 2007 and 2013 in the MIDAS database. Only products with a single orphan indication or with multiple indications which were all orphan were included in the analysis (N=303). Total orphan drug expenditures were calculated annually from 2007 to 2013 and further reported as a proportion of total annual pharmaceutical drug expenditures. All expenditures were adjusted to 2014 USD using the Bureau of Economic Analysis GDP deflator. Expenditures for orphan drugs with cancer indications were evaluated as a subset of all orphan drugs.Results: Between 2007 and 2013, expenditures were measured for a final N=266 branded orphan drugs in the MIDAS database; 37 out of 303 were either not present or had no sales in MIDAS. Orphan drug expenditures totaled $15.0 billion (B) in 2007 and $25.6B in 2013, representing 4.8% to 7.6% of total U.S. pharmaceutical drug expenditures in 2007 and 2013, respectively (Figure 1). There were 74 (27.8%) orphan drugs identified with an orphan cancer indication with expenditures representing 26.7% (2007) and 34.8% (2013) of total orphan drug expenditures. Over the same period (2007–2013), a total of 145 orphan drugs were granted approval (both new molecular entities and supplemental applications/indications).Conclusions: Although the ODA has led to an increase in the number of approved orphan drugs and the annual expenditures on orphan drugs, the overall budget impact of orphan drugs is relatively small and has remained fairly stable as a proportion of total pharmaceutical expenditures. Concerns that growth in orphan drug expenditures may lead to unsustainable drug expenditures do not appear to be justified. [Display omitted] DisclosuresDivino:IMS Health: Employment. Dekoven:IMS Health: Employment. Wang:MKTXS: Employment. Kleinrock:IMS Health: Employment. Harvey:Celgene, Millennium, Onyx and Novartis: Research Funding. Wade:IMS Health: Employment. Kaura:Celgene: Employment, Equity Ownership.

Value-based reimbursement decisions for orphan drugs: a scoping review and decision framework.

BackgroundThe rate of development of new orphan drugs continues to grow. As a result, reimbursing orphan drugs on an exceptional basis is increasingly difficult to sustain from a health system perspective. An understanding of the value that societies attach to providing orphan drugs at the expense of other health technologies is now recognised as an important input to policy debates.ObjectivesThe aim of this work was to scope the social value arguments that have been advanced relating to the reimbursement of orphan drugs, and to locate these within a coherent decision-making framework to aid reimbursement decisions in the presence of limited healthcare resources.MethodsA scoping review of the peer reviewed and grey literature was undertaken, consisting of seven phases: (1) identifying the research question; (2) searching for relevant studies; (3) selecting studies; (4) charting, extracting and tabulating data; (5) analyzing data; (6) consulting relevant experts; and (7) presenting results. The points within decision processes where the identified value arguments would be incorporated were then located. This mapping was used to construct a framework characterising the distinct role of each value in informing decision making.ResultsThe scoping review identified 19 candidate decision factors, most of which can be characterised as either value-bearing or ‘opportunity cost’-determining, and also a number of value propositions and pertinent sources of preference information. We were able to synthesize these into a coherent decision-making framework.ConclusionOur framework may be used to structure policy discussions and to aid transparency about the values underlying reimbursement decisions for orphan drugs. These values ought to be consistently applied to all technologies and populations affected by the decision.Electronic supplementary materialThe online version of this article (doi:10.1007/s40273-014-0235-x) contains supplementary material, which is available to authorized users.

A methodological framework for drug development in rare diseases.

IntroductionDeveloping orphan drugs is challenging because of their severity and the requisite for effective drugs. The small number of patients does not allow conducting adequately powered randomized controlled trials (RCTs). There is a need to develop high quality, ethically investigated, and appropriately authorized medicines, without subjecting patients to unnecessary trials.Aims and ObjectivesThe main aim is to develop generalizable framework for choosing the best-performing drug/endpoint/design combinations in orphan drug development using an in silico modeling and trial simulation approach. The two main objectives were (i) to provide a global strategy for each disease to identify the most relevant drugs to be evaluated in specific patients during phase III RCTs, (ii) and select the best design for each drug to be used in future RCTs.Methodological approachIn silico phase III RCT simulation will be used to find the optimal trial design and was carried out in two steps: (i) statistical analysis of available clinical databases and (ii) integrative modeling that combines mathematical models for diseases with pharmacokinetic-pharmacodynamics models for the selected drug candidates.ConclusionThere is a need to speed up the process of orphan drug development, develop new methods for translational research and personalized medicine, and contribute to European Medicines Agency guidelines. The approach presented here offers many perspectives in clinical trial conception.

PSY108 - Top 20 Orphan Drugs Availability, Pricing and Reimbursement In Slovakia: 2005-2012 Review

Orphan drugs are highly priced and top 20 orphan drugs create almost 2,5% of total drug expenditure in Slovakia. We conducted 8 years review of government and literature sources to provide insight into pricing, reimbursement and availability situation surrounding top 20 orphan drugs in Slovakia from the health care payer perspective. We provide analysis of official prices, reimbursement status and availability of top 20 orphan drugs in Slovakia from 2005 till 2012. Data were obtained from government sources. We considered orphan drugs list (Cote and Keating, 2012) that exceeded 1 billion $ sales in 2008 (globally) and compared molecules' availability in Slovakia. Same molecules are among best selling 20 orphan drugs in Slovakia, with highest sale of 95 million EUR (Bevacizumab, 2005-2012) compared to lowest sale of 19 million EUR (Tacrolimus, 2005-2012). It took from 1 (Imatinib) to 19 years (Glatiramer acetate) to be launched in Slovakia after orphan designation. Top 20 orphan drugs had average DOT 472 EUR compared to total pharma market DOT average 0,43 EUR. From selected orphan drugs 60% had full (100%) reimbursement status and 40% were fully covered by hospital budgets. Only 4 of them were launched in Slovakia since 2005 (included), 16 of them were launched from 1990 till 2004. Prices ranged from 330 EUR to 5800 EUR (ex-factory, one package, 2012). There are highly valuable incentives for industry to invest in to development of orphan drugs in EU. Current context of economic constraints in EU however justifies the need to pay close attention to the rationale of maintaining such incentives in the context of potential return on investments of companies offering high priced drugs. Top 20 orphan drugs in Slovakia have prices high above average and also full reimbursement status. We expect more restrictive drug policy measures in this field.

The pooling of manpower and resources through the establishment of European reference networks and rare disease patient registries is a necessary area of collaboration for rare renal disorders.

This review aims to provide guidance on emerging concepts and policy related to European reference networks (ERNs) for rare diseases (RDs) and the development and management of RD patient registries. A major problem facing many RDs including rare renal disorders is that patients do not have a specialist centre that they can attend where clinicians, working as a multidisciplinary team, are experts in the particular disease. Furthermore, for most RDs, no single centre, and in many cases no single country, has sufficient numbers of patients and resources to fully understand the natural history or to conduct clinical and translational research. Therefore, the pooling of manpower and resources through the establishment of ERN and RD patient registries is a common and necessary area of collaboration. The concept of European networks for RDs dates back to the early 2000s and the Commission launch of a call for European pilot reference networks for RDs. These networks of expert centres have been brought together through the desire for further knowledge and innovation in RD areas. Networks demand a holistic approach and long-term vision with close collaboration between clinicians, diagnostic laboratories, scientists, patients and their families. The development of legal measures for ERNs is in progress at the Commission and these networks will be a shared responsibility of the Commission and member states. In the context of ERNs, an essential activity is the patient registries. Patient registries are organized databases where patient information, including demographic, medical and family history, are collected, stored and available for retrieval via standardized and secure methods. Patient registries are increasingly recognized as crucial tools for RD research for which international collaboration is absolutely essential to understand the pathogenesis of rare genotypes, achieve a unified collection of phenotypic data, foster natural history studies providing the foundation for successful orphan drug development, facilitate studies to identify appropriate clinical endpoints or biomarkers, identify participants for research and clinical trials and support discussions with regulators including the safety and efficacy evaluation of potential therapies. Furthermore, patient registries are often used as part of regulatory decisions and post-marketing surveillance requirements. Data can be entered into a registry by patients, clinicians, researchers or directly imported from patient's health records. The major concern in maintaining the dynamic of these networks and registries is sustainability, as the infrastructures and coordination have a cost.

A Economia das Doenças Raras: Incentivos e Regulação

The objective of this paper is to review theoretical and empirical about the economics of rare diseases and orphan drugs and to analyze their main implications, identifying the magnitude of the problem, its current importance and describe the main regulatory instruments for this market. Rare diseases present a low prevalence in a given population, they are usually degenerative, chronically debilitating, and require long-term treatment, affecting the physical, mental, sensory and behavioral capabilities of the patients. Orphan drugs are used for diagnosis, prevention and treatment of rare diseases. The rarity implies difficulties for proof of their clinical efficacy. The regulatory systems for rare diseases in the United States and European Union will be presented and their influence for development of orphan drugs. It was concluded that the regulatory mechanisms are able to stimulate the development of orphan drugs.

Comparative effectiveness research and the rise of orphan indications

Purpose – The purpose of this paper is to show the link between the comparative effectiveness research (CER) and the interest in developing drugs for rare disease by the pharmaceutical industry. Total healthcare spending is on the rise without having a corresponding better health outcome. As such, with the growing role of government in healthcare, measuring and demonstrating value is beginning to expand beyond the private sector to metrics applied in the public sector. A modern approach to comparative effective research began its rapid rise in the USA when the American Recovery and Reinvestment Act of 2009 allocated $1.1 billion for CER. This paper analyzes the implication and impact on the pharmaceutical industry. Design/methodology/approach – The paper reviews the basic definitions of CER and its areas of strength and weakness. It used real examples of drugs to show the impact of the Reinvestment Act of 2009 on the rise of orphan drugs. Findings – The study shows that the act encouraged the development of orphan drugs, mainly because of the low budget impact due to a smaller patient base. Provisions of the Affordable Care Act provide incentives for such rare disease indications, as no one can be denied coverage with pre-existing conditions. Research limitations/implications – The study is limited by the number of available rare drugs and the ongoing process of implementation of the Affordable Care Act. Practical implications – The study shows the cost-effective method of treating medical conditions. Social implications – Development of orphan drugs opens up access to care for many patients at a cost-effective price. Originality/value – This paper shows the link between the CER and the interest in developing drugs for rare disease by the pharmaceutical industry. It also brings out the possible implication of the Affordable Care Act on the pharmaceutical industry with respect to its strategies for drug development and drug portfolio.

The evolving drug development landscape: from blockbusters to niche busters in the orphan drug space.

Strategy, Management and Health Policy Large pharmaceutical companies have traditionally focused on the development of blockbuster drugs that target disease states with large patient populations. However, with large-scale patent expirations and competition from generics and biosimilars, anemic pipelines, escalating clinical trial costs, and global health-care reform, the blockbuster model has become less viable. Orphan drug initiatives and the incentives accompanied by these have fostered renewed research efforts in the area of rare diseases and have led to the approval of more than 400 orphan products. Despite targeting much smaller patient populations, the revenue-generating potential of orphan drugs has been shown to be huge, with a greater return on investment than non-orphan drugs. The success of these "niche buster" therapeutics has led to a renewed interest from "Big Pharma" in the rare disease landscape. This article reviews the key drivers for orphan drug research and development, their profitability, and issues surrounding the emergence of large pharmaceutical firms into the orphan drug space.

Patient database and orphan drug development

There is no concept of rare disease (RD) but Nambyo (intractable disease) since 1972. In 1995 the definition of Nambyo included the concept of rareness and the frequency for a Nambyo is less than 50000 in Japanese population. Currently Nambyo are selected for special research support, and special treatment of medical expenses. The budget for research is 10 billion yen. The approximate number of medical recipients is estimated to be 700000. The measures already developed for Nambyo are not comprehensive, therefore currently several additional measures are being envisaged. We are now planning to join the Orphanet. The expectations for Orphanet Japan are to: Enhance international collaboration of RD, providing international up-to-date information of RD in Japanese, inform historical and up-to-date research of Nambyo, and promote information exchange, joint research and network establishment. It is necessary to make a Patient Registry for rare disease, and hopefully have a structure to integrate worldwide registry with same concept. Recently "International Rare Disease Research Consortium (IRDiRC)" was formed. The purpose of this consortium is to make an international coordination of the rare disease research, and to integrate the knowledge of the rare disease research. We will also talk about the Patient Registry by Patient Advocacy group, including Patient Reported Outcome (PRO).

PRM139 - A comprehensive economic and pricing modeling framework for understanding orphan drug development

PRM139 - A comprehensive economic and pricing modeling framework for understanding orphan drug development

New Financial and Research Models for Pediatric Orphan Drug Development - Focus on the NCATS TRND Program.

While there are approximately 7,000 identified human diseases considered as "rare" based on population prevalence or incidence, the cumulative impact runs into the millions of patients globally. Although the genetic underpinnings of more than 2,000 rare diseases have been elucidated, there remains a paucity of therapeutic options, frequently due to lack of commercial interest. Development programs suffer high attrition within the so-called "Valley of Death," in which the risks of scientific failure are still too high to justify the increasing development costs. This problem is common to any drug development campaign, but it is particularly exacerbated in the rare diseases, many of which arise in childhood. To stimulate development of therapeutics for these otherwise underserved patient populations, a number of regulatory incentives and research initiatives have been established. Extended patent protections, expedited regulatory reviews for qualified drug sponsors, and clinical trial grant support aim to foster interest in completing development programs. To stimulate researchers to embark on rare disease drug development campaigns, earlier-stage preclinical research resources have been created, as well, such as the Therapeutics for Rare and Neglected Diseases (TRND) program at the U.S. National Institutes of Health (NIH). TRND is a unique NIH program created to support drug development through formation of public-private partnerships. These partnerships leverage the robust biopharmaceutical industry experience of the TRND staff scientists and the deep disease area expertise of the collaborating partners. Each project adopted into the TRND portfolio aims to satisfy two broad goals: developing a novel therapy for a rare or otherwise neglected disease, and exploring ways to accelerate the drug development process overall so that lessons learned can be disseminated to the wider community undertaking translational research. This article discusses common obstacles and opportunities for therapeutic development, and provides examples of the types of projects TRND has undertaken across a broad range of pediatric rare disorders.

Orphan regulations for orphan drug development in India

Through this review article an attempt has been made to put forward the challenges faced by rare disease drug development and the current scenario of orphan drug legislations in India. An orphan drug is a pharmaceutical agent that is used to treat a rare medical condition (viz., glioblastoma multiforme, nocardiosis, Tourette syndrome, etc). Developed countries such as United States (US), Europe, Japan, and Australia have laid down legal framework for combating rare diseases. A path breaking legislation was formulated by the US government way back in 1983, known as “Orphan Drugs Act (ODA).” The key purpose of ODA was to incentivize R and D initiatives for such drugs to treat millions of population suffering from “orphan diseases.” Though the percentage of patients suffering from “rare diseases” in India is reportedly higher than the world average, unfortunately even today such cases get little help from our government. Indian government should also encourage its domestic pharmaceutical industry to get engaged in research for orphan drugs by putting an “ODA” in place and extending financial support, and regulatory concessions like smaller and shorter clinical trials, without further delay. Thus, India could well‑demonstrate that the concept of orphan drugs for orphan diseases is really not orphan in India. Key words: Orphan diseases, orphan drugs act, orphan drug

Orphan regulations for orphan drug development in India

Orphan regulations for orphan drug development in India