How to Import Drugs That are Still on Clinical Trial but Life Saving?

One can import and manufacture new drugs currently in Phase III clinical trials under the new Compassionate Use Program. These new drugs can be prescribed to patients with critical diseases.

Highlights :

‘Compassionate Use Program to benefit patients’ with spinal muscular atrophy (SMA) and severe COVID-19
A team of mothers whose children were diagnosed with SMA fight with the government to introduce the new rule
Allows import and manufacture of new drugs currently in Phase III clinical trials in other countries
These drugs on trial maybe the only hope for a patient suffering from an incurable disease

The compassionate use program in India was initiated by a group of mothers whose kids were diagnosed with spinal muscular atrophy (SMA). This program enables the use of new drugs that are not available in the country but under phase III clinical trials in the country or in another country.

With the approval of the Central Licensing Authority, the new drug can be imported or manufactured to treat patients diagnosed with life-threatening diseases. These drugs can be useful where there are no current cures available for a condition like cancer or rare diseases. Currently patients diagnosed with SMA and severe COVID-19 may benefit from the new rule.

Many patients in India suffer from rare diseases. Most patient advocacy groups and clinicians cite between 5,000 and 8,000 rare diseases in the world that have no known cure. There are also some infections cancers, especially when in advanced stages where there are no known cures. It is in such situations of desperation that cancers that ‘Compassionate Use Program’ to procure medication can kick in to benefit patients.One of those rare diseases is spinal muscular atrophy (SMA)
. What brought hope recently to many patients and their families was the new amendment by the government of India. It allowed the use of untested drugs that are currently under clinical trials in other countries to be brought to India under the term “Compassionate Use” program.

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‘New Drugs currently in Phase III Clinical Trials can be imported or manufactured in India under the Compassionate Use Program. Patients diagnosed with rare diseases like spinal muscular atrophy and the current pandemic COVID-19 to benefit from the new rule.’
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How does a Clinical Trial take place?

The Center for clinical trial needs to get registered

The respective companies announce the details on their websites
The drug controller evaluates the proposal and approves it
The respective companies also need to decide the countries for the clinical trial to confirm the tests, required expertise in conducting global clinical trials and, the experience of the investigator.
The companies will also decide the country in which the clinical trials are conducted. Once the drug gets approved, there should be patent protection.

In India, we don’t have strict patent protection for drug manufacturing, and many generic drugs have been manufactured in India and have often been looked at as patent violation by international bodies, but this has changed in recent years.

A new promising drug that shows early encouraging results in a clinical trial raises hopes of patients and to participate in such global clinical trials; an Indian patient hasto travel to the clinical trial center abroad and can sometimes be included in it if they meet the inclusion criteria.

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Clinical trials serve as a vital component for improving the treatment of medical conditions as they lead to higher standards of patient care.

How did the Compassionate Use Program begin in India?

Archana Panda, a mother of a child diagnosed with SMA, started interacting with Roche after finding out that Roche had developed a new molecule that could help children with Type I SMA. The company had also identified 34 children in India who would be given the medicine. However, the drug could only be imported unless the government introduced guidelines for compassionate use.

After numerous meetings with the Health Ministry and Drugs Controller General of India, a team of mothers contacted different advocacy groups in Europe and the US. They compiled guidelines from different countries and pleaded with the government to bring the Compassionate Use to India.For many years families and patients have been battling to get this scheme introduced in India.

There was Dr.Razeerna, whose 14-year-old son Jewel has SMA had to go to the court to get access, and it took about 10-12 working days for the order to get approved. Though around six children benefitted from the order, one child had lost his life while waiting for the order to be approved. Between January and June, seven children passed away while waiting for the drug. In critical conditions and diseases, time plays a vital role.

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What is the Compassionate Use Program?

On June 5th, 2020, the Ministry of Health and Family Welfare notified a draft to amend the New Drugs and Clinical Trials Rules of 2019. The new draft included the rules to import and manufacture unapproved new drugs for Compassionate Use for the treatment of patients by medical institutions or hospitals.

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What are the eligibility conditions for Compassionate Use of New Drugs?

Patients who are suffering from

Life-threatening diseases
Diseases that cause permanent severe disability
Diseases which require unmet medical need which has not been permitted in the country previously.

Which drugs can be Prescribed under the Compassionate Use of New Drugs?

A medical officer of a medical institution or hospital may be able to import new drugs that are currently under Phase III clinical trial in the country or another country.

What are the Procedures to be Followed to Import New Drugs?

To import new drugs, the Medical Superintendent of the hospital or Head of the Medical Institution must submit a certified application to the Central Licensing Authority in Form CT-28.

The form CT-28 must be also be accompanied with details regarding the

The rationale for the use of the new drug
Criteria for patient selection with disease description and other details
Method of administration of the drug, dose including the duration of the therapy
Description of manufacturing facility
CMC Information
Pharmacology and toxicology information
Description of clinical and laboratory tests to evaluate the effects of the drug and minimize its risks.

What are the limitations to manufacture a new drug under the Compassionate Use Program?

To manufacture a drug for the compassionate use program, similar rules and criteria for importing need to be met. The quantity of the new drug manufactured under the Compassionate Use Program will always be under 100 doses other than a few rare exceptional cases where permission is required.

How does the Compassionate Use Program Help Patients with Spinal Muscular Atrophy?

The Compassionate Use Program gives a ray of hope to patients diagnosed with Type Ispinal muscular atrophy.

Subhrojyoti Bhowmick, a clinical research doctor at Peerless Hospital, treating a two-year-old boy Abhineet diagnosed with Type I SMA suggested a drug Risdiplam.

Risdiplam is a drug from Roche which is currently under clinical trials. The ethics committee of the hospital met in May and discussed the participation of the family in the Roche’s compassionate use program. They are currently in the process of signing up for the program with Roche India along with all the other documents required.

Another doctor in Hyderabad, Ramesh Konanki at Rainbow Hospital has also prescribed Risdiplam for two patients with Type I SMA.

What are the benefits of Compassionate Use Program in COVID-19?

In the current COVID-19 pandemic, there is urgent need for medications for severely ill patients. The compassionate use program would be highly beneficial for patients as they can be prescribed drugs currently in phase III of clinical trials.

Newer drugs or vaccines as they are introduced and show benefit in a clinical trial could be imported to treat severely ill patients diagnosed with COVID-19.

Importing many drugs like Spinraza and Zolgensma may help treat SMA. “These drugs are not available in India as we do not have an insurance model. So,the compassionate use program would encourage more pharma companies to bring their drugs to India” Archana Panda said.

Compassionate Use Program would also have a significant impact on treating patients severely infected by the COVID-19.

References:

The Gazette of India - (https://cdsco.gov.in/opencms/opencms/system/modules/CDSCO.WEB/elements/download_file_division.jsp?num_id=NTk3Mw==)
Health Ministry publishes New Drugs and Clinical Trials (Amendment) Rules - (https://www.expresshealthcare.in/news/health-ministry-publishes-new-drugs-and-clinical-trials-amendment-rules/421517/)
Mothers’ struggle behind nod for ‘compassionate use’ drugs - (https://economictimes.indiatimes.com/industry/healhcare/biotech/mothers-struggle-behind-nod-for-compassionate-use-drugs/articleshow/76309851.cms)
’Compassionate use’ means life for a fortunate few - (https://economictimes.indiatimes.com/industry/healthcare/biotech/healthcare/compassionate-use-means-life-for-a-fortunate-few/articleshow/76903892.cms)

Source-Medindia