ORPHAN DRUGS IN INDIA AND CHALLENGES (2022)

Orphan drugs are medicinal products intended for diagnosis, prevention or treatment of life-threatening or very serious diseases or disorders that are rare. In USA, the Orphan Drug Act was passed in 1983 to give drug companies incentives to develop treatments for rare diseases. India does not have laws on orphan drugs though CDSCO defined orphan drugs as those intended to treat a condition which affects fewer than 200,000 people.

WHO defines rare disease as a disease or condition with a prevalence of ≤1/1000 population. Other definitions are diseases affecting <1/2000 population in European union, whereas USFDA defines it as any disorder affecting <200,000 population at a single time point.

It is estimated that globally around 6000 to 8000 rare diseases exist with new rare diseases being reported in the medical literature regularly. However, 80% of all rare disease patients are affected by approximately 350 rare diseases.

(Video) ORPHAN DRUGS AND CHALLENGES

ORPHAN DRUGS IN INDIA AND CHALLENGES (1)

India, like many other developing countries, currently has no standard definition of rare diseases and data on prevalence. Since there is no epidemiological data, there are no figures on burden of rare diseases and morbidity and mortality associated with them. If we apply the international estimate of 6% to 8% of population being affected by rare diseases, to India, we have between 72 to 96 million people affected by rare diseases in the country, which is a significant number. However, this is at best a general estimate and India will need to arrive at its own estimate and definition of rare diseases, derived chiefly from prevalence data, which is currently lacking. So far only about 450 rare diseases have been recorded in India from tertiary care hospitals.

The most common rare diseases include Haemophilia, Thalassemia, Sickle-cell Anaemia and Primary Immuno Deficiency in children, auto-immune diseases, Lysosomal storage disorders such as Pompe disease, Hirschsprung disease, Gaucher’s disease, Cystic Fibrosis, Hemangiomas and certain forms of muscular dystrophies.

Lack of epidemiological data
Data on how many people suffer from different rare diseases in India is lacking. The cases identified so far have been diagnosed at tertiary hospitals. The lack of epidemiological data on incidence and prevalence of rare diseases impedes understanding of the extent of the burden of rare diseases and development of a definition. It also hampers efforts to arrive at correct estimation of the number of persons suffering from these diseases and describe their associated morbidity and mortality. In such a scenario, the economic burden of most rare diseases is unknown and cannot be adequately estimated from the existing data sets. Although extremely challenging, considering the complexity of various diseases and the difficulty in diagnosis, there is a clear need to undertake systematic epidemiological studies to ascertain the number of people suffering from rare diseases in India.

Diagnosis of rare diseases
Diagnosis of a rare condition may take upto several years, owing to difficulty in diagnostic modalities and lack of awareness among doctors. For many rare diseases, no diagnostic method exists, or diagnostic facilities are unavailable. Traditional genetic testing can only address a few genes at a time. As a result, physicians must often provide their best guess on which genes to investigate. If the test is negative, further testing will be required, which is an expensive and time- consuming process. There is a lack of awareness about rare diseases in general public as well as in the medical profession. Many doctors lack appropriate training and awareness to be able to correctly and timely diagnose and treat these conditions. According to a recent report, it takes patients in United States (US) an average of 7.6 years and patients in United Kingdom (UK) an average of 5.6 years to receive an accurate diagnosis, typically involving as many as eight physicians (four primary care and four specialists). In addition, two to three misdiagnoses are typical before arriving at a final diagnosis. Delay in diagnosis or a wrong diagnosis increases the suffering of the patients exponentially. There is an immediate need to create awareness among general public, patients and their families and doctors, training of doctors for better diagnosis, standardisation of diagnostic modalities, developing of new diagnostic tools and investment in gene therapy.

(Video) The lucre of orphan drugs from an Indian context

Challenges in research and development
A fundamental challenge in research and development for the majority of rare diseases is that there is relatively little known about the pathophysiology or the natural history of these diseases. Rare diseases are difficult to research upon as the patient pool is very small and it often results in inadequate clinical experience. Therefore, the clinical explanation of rare diseases may be skewed or partial. The challenge becomes even greater as rare diseases are chronic in nature, where long term follow up is particularly important. As a result, rare diseases lack published data on long-term treatment outcomes and are often incompletely characterised.

This makes it necessary to explore international and regional collaborations for research, collaborations with the physicians who work on any rare disease and with patient groups and families dealing with the consequences of these disorders. This will help gain a better understanding of the pathophysiology of these diseases, and the therapeutic effects that would have a meaningful impact on the lives of patients. There is also a need to review and where possible modify, clinical trial norms keeping in mind the particular challenges in rare diseases, without compromising on the safety and quality of the drugs or diagnostic tools.

CHALLENGES IN TREATMENT

Unavailability of treatment
Availability and access to medicines are important to reduce morbidity and mortality associated with rare diseases. Despite progress in recent years, effective or safe treatment is not available for most of the rare diseases. Hence, even when a correct diagnosis is made, there may not be an available therapy to treat the rare disease. There are between 7000 - 8000 rare diseases, but less than 300 have therapies available to treat them. About 95% rare diseases have no approved treatment and less than 1 in 10 patients receive disease specific treatment. Where drugs are available, they are prohibitively expensive, placing immense strain on resources of families, health systems and donor agencies alike.

Prohibitive cost of treatment
As the number of persons suffering from individual rare diseases is small, they do not constitute a significant market for drug manufacturers to develop and bring to market drugs for them. For this reason, rare diseases are also called ‘orphan diseases’ and drugs to treat them are called “orphan drugs”. Where, they do make drugs to treat rare diseases, they sell them at extremely high costs, to recoup the cost of research and development. At present very few pharmaceutical companies are manufacturing drugs for rare diseases globally and there are no domestic manufacturers in India. Due to the high cost, the government has not been able to provide these drugs for free. It is estimated that for a child weighing 10 kg, the annual cost of treatment for some rare diseases, may vary from Rupees 18 Lakhs to 1 crore 70 lakhs.

(Video) The challenges of developing treatments for rare diseases

Further, the methodology for evaluating treatment of rare diseases is often still in experimental phase, impeding assessment of clinical relevance and cost effectiveness. Several countries have through legislation like Orphan Drug Act (ODA), provided incentives to drug manufactures to encourage them to manufacture drugs for rare diseases. The most powerful incentive for drug manufacturers is the grant of 7 to 10 years of exclusive marketing rights including protection from imports, if a drug gets ‘orphan drug’ status. This means that pharmaceutical companies can price their drugs without fear of competition. This has changed the nature of drug development. The number of requests for orphan drug designation has quadrupled since 2000. The result has been a boom in drug sales and profits. In fact, almost a third of drugs for rare diseases now exceed £1bn in annual sales. The global orphan drugs market is expected to reach £144bn by 2020, and account for 19% of total branded prescription drug sales. The average price of a drug for rare diseases exceeds $100,000 a year.

According to reports, while legislations on orphan drug development, like the US Orphan Drug Act, have facilitated development of orphan drugs, they have not been able to check the prices of these drugs, which has led to grave concerns for health system sustainability. For instance, the cystic fibrosis drug, Kalydeco (ivacaftor) is priced at £14,000 per patient per month. In fact, each one of the world's 10 most expensive drugs, is a drug to treat some rare disease, with Soliris (eculizumab) being the most expensive at £340,000 per patient per year.

Although these drugs are prescribed to fewer patients, their extremely high prices can result in revenues equivalent to traditional blockbuster drugs. Publicly listed pharmaceutical companies that are orphan drug market authorisation holders are associated with higher market value and greater profits than companies not producing drugs for rare diseases.

Over the years many drugs have won ‘orphan drugs’ status even if they are not new or represent a scientific breakthrough. An inexpensive off-patent drug approved by the FDA for one condition, but widely prescribed as an “off-label” treatment for a rare disease, can be transformed into a big moneymaker. The accompanying 7 to 10 years of monopoly status can mean large price hikes for a drug that was already in wide use. For instance, older medicines for rare diseases like imatinib used to treat chronic myloid luekemia can cost over USD 100,000 a year in developed countries. In India, public health safeguards in the patent law that restrict evergreening patents have allowed Indian generic manufacturers to manufacture and supply this drug at far lower prices. The exorbitant prices of drugs for rare diseases has led to concerns even in the developed countries about maintaining sustainability of the rare diseases funding/reimbursement programmes.

The exorbitant prices have led to calls for transparency in setting prices of drugs and for price control and have even prompted scrutiny and congressional inquiries. This should be a matter of careful consideration for any legislative or policy measures adopted in India for promotion of drug development for rare diseases. There is a pressing need for multipronged strategies to make drugs more affordable – by taking measures to lower the prices of drugs, encourage generic and local manufacturing of drugs and encourage PSUs to manufacture drugs for rare diseases.

(Video) Rare Diseases: A Global Challenge

Skyscraping Pricing – Increasing opportunities
Every time, high drug prices are not due to patent of medicines. Just remember Infamous Martin Shkreli, who purchased Turing Pharmaceuticals which is involved in production of Daraprim and subsequently hiked price of medicine by 5500 percent. The world took note on that price hike. It became a hike controversy when Turing raised the price of a dose of Daraprim in the U.S. market from US$13.50 to US$750.

Patients whose liver does not produce one of the two primary bile acids and results in a critical condition that causes neurological problems, seizures and even dementia. For the past half century, it was relatively easy to treat such patients at a cost of €500 per annum by using Chenofalk (chenodeoxycholic acid), an off-label drug. In 2008, Leadiant acquired the rights to Chenofalk, and developed its own version, known as CDCA Leadiant. Then, very smartly in 2014, it got the EU to classify its version as an “orphan drug”, giving Leadiant exclusive marketing rights for 10 years. Immediately thereafter, it jacked up the price of CDCA Leadiant to about €150,000 for a year’s treatment.

Cydan, which helps launch startups focused on orphan-drug development, have decided to steer clear of them. That strategy is beginning to pay off. According to Adams, the company has successfully spun out three startups. The first of these, Vtesse, launched in January 2015 to develop a treatment for Niemann-Pick type C, a rare lysosomal storage disorder, using sugar molecules called cyclodextrins. It was acquired by Sucampo Pharmaceuticals for $200 million last April 2017.

India does not have laws on orphan drugs as yet but the draft rules released by the health ministry on a regulatory framework for clinical trials. Higher numbers of patients from India should make the pharma companies to invest more in orphan drugs. So far, USA has developed over 200 drugs and biological products to treat orphan diseases since orphan drugs act passed.

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FAQs

What is orphan drug India? ›

An orphan drug is a treatment designed for a rare disease, or one which has no satisfactory method of diagnosis, prevention or treatment. Although individually uncommon, with an average prevalence of fewer than 40 to 50 cases per 100,000 people, around 350 million patients worldwide are living with a rare disease.

What are orphan drugs and examples? ›

An orphan drug can be defined as one that is used to treat an orphan disease. For example, haem arginate, used to treat acute intermittent porphyria, variegate porphyria, and hereditary coproporphyria [12], is an orphan drug.

What role do orphan drugs have in the drug process? ›

An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases.

Why are orphan drugs important? ›

The so-called 'orphan drugs' are intended to treat diseases so rare that sponsors are reluctant to develop them under usual marketing conditions.

What does Orphan Drug mean? ›

Listen to pronunciation. (OR-fun …) A drug used to treat, prevent, or diagnose an orphan disease. An orphan disease is a rare disease or condition that affects fewer than 200,000 people in the United States.

Why are there so many rare diseases in India? ›

India is home to culturally and genetically diverse populations, which are burdened by genetic diseases. Due to the high prevalence of recessive alleles owing to endogamous practices, rare diseases form a significant burden in India.

Why orphan drug is so called? ›

An orphan drug is a medication or other medicinal product used to treat a rare disease or disorder. It is called an “orphan drug” because due to its limited market, it is unlikely to be adopted by a pharmaceutical company.

How many orphan drugs are currently on the market today? ›

Specifically, in 2020, the agency approved 32 novel drugs and biologics with orphan drug designation.

Are orphan drugs profitable? ›

Pharmaceutical companies are seeking “orphan” status to develop blockbuster drugs used to treat other common medical conditions. They reap the benefits of orphan status – subsidies, tax credits, and waivers – while generating billions of dollars in profits.

Does orphan drug status expire? ›

New chemical entity (NCE) exclusivity expires after five years (sometimes four); orphan drug exclusivity ends after seven years; and even Reference Product exclusivity expires eventually – after a whopping 12 years.

What are examples of orphan diseases? ›

Orphan and rare diseases include more familiar conditions such as cystic fibrosis, Lou Gehrig's disease and Tourette's syndrome, as well as less familiar conditions such as Duncan's Syndrome, Madelung's disease and acromegaly/gigantism. The prevalence of rare diseases is often an estimate and may change over time.

Are orphan drugs FDA approved? ›

The FDA has authority to grant orphan drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition. Orphan drug designation qualifies sponsors for incentives including: Tax credits for qualified clinical trials.

Who created the Orphan Drug Act? ›

The Orphan Drug Act (ODA) was signed into law in 1983 by President Ronald Reagan. This major piece of legislation was the first-of-its-kind for rare diseases and its success has helped to encourage similar legislation in other parts of the world.

What is orphan trial? ›

Orphan products clinical trials grants are a proven method of successfully fostering and encouraging the development of new safe and effective medical products for rare diseases/conditions.

Are orphan drugs covered by insurance? ›

Medicare Plans' Coverage of Orphan Drugs—Stand-Alone PDPs and MA-PDs. The coverage rate (percentage of plans covering a drug) for orphan drugs among Medicare prescription drug plans is high. On average, an orphan drug is covered by 84 percent (standard deviation: 24 percent) of stand-alone PDPs.

Who is considered an orphan? ›

An orphan is a child whose parents have died. The term is sometimes used to describe any person whose parents have died, though this is less common. A child who only has one living parent is also sometimes considered an orphan.

How do you qualify for orphan drugs? ›

Criteria for orphan drug designation
  1. The product must be intended for use in a rare disease or condition.
  2. Adequate documentation or prevalence data must demonstrate that the intended condition is rare.
17 Feb 2021

What happens after orphan drug designation? ›

After orphan designation

Developing medicines intended for small numbers of patients has little commercial incentive under normal market conditions. Therefore, the EU offers a range of incentives to encourage the development of designated orphan medicines.

Why do so many kids in India have birth defects? ›

Indian people are living in the midst of risk factors for birth defects, e.g., universality of marriage, high fertility, large number of unplanned pregnancies, poor coverage of antenatal care, poor maternal nutritional status, high consanguineous marriages rate, and high carrier rate for hemoglobinopathies.

Which country has most genetic disorders? ›

The Arab world has one of the highest rates of genetic disorders globally; some 906 pathologies are endemic to the Arab states, including thalassaemia, Tourette's syndrome, Wilson's disease, Charcot-Marie-Tooth disease, mitochondrial encephalomyopathies, and Niemann-Pick disease.

What are 7 genetic disorders? ›

Genetic Disorders
  • Genetic Disorders. Sickle Cell Disease.
  • Cystic fibrosis. Cystic Fibrosis Liver Disease.
  • Brain, Nerves and Spine. Huntington's Disease.
  • Cleft lip and palate. Cleft Lip and Palate.

Why are rare diseases called orphan? ›

In some parts of the world, an orphan disease is a rare disease whose rarity means there is a lack of a market large enough to gain support and resources for discovering treatments for it, except by the government granting economically advantageous conditions to creating and selling such treatments.

Is amphotericin an orphan drug? ›

Oral Amphotericin B Formulation Gets Orphan Drug Status for Cryptococcosis. The Food and Drug Administration (FDA) has granted Orphan Drug designation to MAT2203 (Matinas BioPharma) for the treatment of cryptococcosis. Cryptococcosis is a rare, fatal infection caused by the fungus Cryptococcus neoformans.

Is Zolgensma an orphan drug? ›

Currently, since the orphan drug Zolgensma is without generic variants, instead of gambling with threats, the Government must proactively endeavour to issue Compulsory Licenses for the drug or enact any measures that reduces the status of receiving Zolgensma treatment, from fiction to reality.

How many people use orphan drugs? ›

According to the Orphan Drug Act, the US FDA considers a rare disease to be one that affects fewer than 200,000 Americans. Orphan drug designation by the European Medicines Agency (EMA) uses a condition prevalence in the European Union (EU) of not greater than five in 10,000 people affected (i.e., one in 2,000).

What companies have orphan status? ›

Pages in category "Orphan drug companies"
  • AbbVie.
  • Actelion.
  • Alexion Pharmaceuticals.
  • Amgen.
  • Amicus Therapeutics.
  • AstraZeneca.

How are orphan drugs priced? ›

Pricing of orphan drugs follows the same economic logic as drug pricing in general: the price of an orphan drug is set by a manufacturer in an effort to recoup research and development (R&D) costs and to attain a certain profit margin.

Is it profitable to develop drugs for rare diseases? ›

Hundreds of new rare-disease treatments have entered the market over the past few decades, and orphan drug development has become a highly profitable industry.

Is Botox an orphan drug? ›

Botox, stocked in most dermatologists' offices, started out as a drug to treat painful muscle spasms of the eye and now has three orphan drug approvals. It's also approved as a mass market drug to treat a variety of ailments, including chronic migraines and wrinkles.

Which of the following is not necessary in patient teaching concerning the excretion of medications quizlet? ›

Which of the following is NOT necessary in patient teaching concerning the excretion of medication? Exercise has no effect on the excretion of medications.

What is the fairness in orphan drug exclusivity act? ›

1629, the Fairness in Orphan Drug Exclusivity Act: “The bipartisan Fairness in Orphan Drug Exclusivity Act will help address soaring prescription drug costs by closing a loophole that has been used to unfairly block generic drugs from coming to market,” Pallone and Eshoo said.

What is orphan market exclusivity? ›

Market exclusivity is an orphan incentive awarded by the European Commission to a specific clinical indication with an orphan designation. Each indication with an orphan designation confers ten years' market exclusivity for the particular indication.

What's the rarest disorder? ›

According to the Journal of Molecular Medicine, Ribose-5 phosphate isomerase deficiency, or RPI Deficinecy, is the rarest disease in the world with MRI and DNA analysis providing only one case in history.

What are rare diseases in India? ›

The most common rare diseases include Haemophilia, Thalassemia, Sickle-cell Anaemia and Primary Immuno Deficiency in children, auto-immune diseases, Lysosomal storage disorders such as Pompe disease, Hirschsprung disease, Gaucher's disease, Cystic Fibrosis, Hemangiomas and certain forms of muscular dystrophies.

Why do you think the FDA promotes orphan drugs? ›

An orphan drug is a drug for a rare disease or condition. Some rare disease treatments have been “orphaned” or discontinued because there was not enough financial incentive to continue development or production. The Orphan Drug Act incentivizes drug development for rare diseases.

Who is subject to orphan drug exclusion? ›

A: The exclusion of drugs with orphan indications applies to free-standing cancer hospitals, rural referral centers, sole community hospitals, and critical access hospitals. The exclusion does not apply to covered entities that meet the 340B Program eligibility requirements and are enrolled under secti...

Is it hard to get orphan drug designation? ›

The orphan application process is arduous

Actually, the orphan application process is quite simple. In the US, the argument hinges on disease prevalence of under 200,000. If the number can be established and there is a “scientific rationale” for the use of the drug, it will most likely achieve orphan status.

Can orphan drugs have generics? ›

An orphan drug is a product that treats a rare disease that affects fewer than 200,000 people. Exclusivity is granted for 7 years on a designated orphan drug. If a new drug is the only solution, then it does not apply to generics.

What was the first orphan drug? ›

The first Orphan drug to be approved under the new law was Hematin (approved in 1984) for acute intermittent porphyria. Acute intermittent porphyria has a prevalence of 10,000–15,000 patients in the United States.

Who does the Orphan Drug Act impact? ›

For thirty-five years the Orphan Drug Act of 1983 has provided incentives for pharmaceutical manufacturers to develop drugs to treat rare diseases—conditions that affect fewer than 200,000 people in the US.

When was Orphan Drug Act was started? ›

The Orphan Drug Act of 1983 grants 7 years of market exclusivity for products to treat rare diseases and conditions affecting fewer than 200 000 patients in the US.

What are examples of orphan drugs? ›

An orphan drug can be defined as one that is used to treat an orphan disease. For example, haem arginate, used to treat acute intermittent porphyria, variegate porphyria, and hereditary coproporphyria [12], is an orphan drug.

Does insurance cover rare disease? ›

Kids with rare diseases can be covered by the Children's Health Insurance Program (CHIP). Apply for Medicare. People under age 65 with certain diseases qualify for Medicare with no waiting period. These conditions are amyotrophic lateral sclerosis (ALS) and end-stage renal disease (ESRD).

Does Medicare cover rare diseases? ›

One model for paying for rare diseases is Medicare coverage for end-stage renal disease (ESRD) and amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease).

How do you qualify for orphan drug designation? ›

Criteria for orphan drug designation
  1. The product must be intended for use in a rare disease or condition.
  2. Adequate documentation or prevalence data must demonstrate that the intended condition is rare.
17 Feb 2021

What is considered an orphan disease? ›

An orphan disease is defined as a condition that affects fewer than 200,000 people nationwide.

Are orphan drugs FDA approved? ›

The FDA has authority to grant orphan drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition. Orphan drug designation qualifies sponsors for incentives including: Tax credits for qualified clinical trials.

What are examples of orphan diseases? ›

Orphan and rare diseases include more familiar conditions such as cystic fibrosis, Lou Gehrig's disease and Tourette's syndrome, as well as less familiar conditions such as Duncan's Syndrome, Madelung's disease and acromegaly/gigantism. The prevalence of rare diseases is often an estimate and may change over time.

Are orphan drugs profitable? ›

Pharmaceutical companies are seeking “orphan” status to develop blockbuster drugs used to treat other common medical conditions. They reap the benefits of orphan status – subsidies, tax credits, and waivers – while generating billions of dollars in profits.

Does orphan drug status expire? ›

New chemical entity (NCE) exclusivity expires after five years (sometimes four); orphan drug exclusivity ends after seven years; and even Reference Product exclusivity expires eventually – after a whopping 12 years.

How long does orphan drug status last? ›

Orphan drug status (designated by the FDA) gives a company exclusive marketing rights for a seven-year period, along with other benefits to recoup the costs of researching and developing drugs to treat rare diseases.

Why orphan drug is so called? ›

An orphan drug is a medication or other medicinal product used to treat a rare disease or disorder. It is called an “orphan drug” because due to its limited market, it is unlikely to be adopted by a pharmaceutical company.

Why is it called orphan disease? ›

Orphan disease: A disease that has not been adopted by the pharmaceutical industry because it provides little financial incentive for the private sector to make and market new medications to treat or prevent it.

What are rare diseases in India? ›

The most common rare diseases include Haemophilia, Thalassemia, Sickle-cell Anaemia and Primary Immuno Deficiency in children, auto-immune diseases, Lysosomal storage disorders such as Pompe disease, Hirschsprung disease, Gaucher's disease, Cystic Fibrosis, Hemangiomas and certain forms of muscular dystrophies.

How many orphan drugs are currently on the market today? ›

Specifically, in 2020, the agency approved 32 novel drugs and biologics with orphan drug designation.

Who is subject to orphan drug exclusion? ›

A: The exclusion of drugs with orphan indications applies to free-standing cancer hospitals, rural referral centers, sole community hospitals, and critical access hospitals. The exclusion does not apply to covered entities that meet the 340B Program eligibility requirements and are enrolled under secti...

Are orphan drugs covered by insurance? ›

Medicare Plans' Coverage of Orphan Drugs—Stand-Alone PDPs and MA-PDs. The coverage rate (percentage of plans covering a drug) for orphan drugs among Medicare prescription drug plans is high. On average, an orphan drug is covered by 84 percent (standard deviation: 24 percent) of stand-alone PDPs.

Why do you think the FDA promotes orphan drugs? ›

An orphan drug is a drug for a rare disease or condition. Some rare disease treatments have been “orphaned” or discontinued because there was not enough financial incentive to continue development or production. The Orphan Drug Act incentivizes drug development for rare diseases.

What are 5 genetic diseases? ›

What are common genetic disorders?
  • Down syndrome (Trisomy 21).
  • FragileX syndrome.
  • Klinefelter syndrome.
  • Triple-X syndrome.
  • Turner syndrome.
  • Trisomy 18.
  • Trisomy 13.
20 Aug 2021

Videos

1. Orphan Drug Innovation Needs and Priorities: CeBIL Symposium 2021
(Cambridge Law Faculty)
2. Strategic Regulatory and Operational Challenges of Rare Disease Studies
(WorldwideClinical)
3. 8-Year-Old Girl Battling A Rare Brain Disease She Calls ‘Awesome’ | TODAY
(TODAY)
4. Driving Improved Outcomes in Orphan Diseases: Challenges of Treating Rare/Orphan Diseases
(DKBmed)
5. Early access to orphan drugs – the rationale, challenges and options available
(Orphan Drugs)
6. #RareDisease : Roadmap For Orphan Drugs in India by Dy Drugs Controller (I), CDSCO Annam Visala
(Indian Organization for Rare Diseases)

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