Orphan Drugs and Mesothelioma


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The Role of Orphan Drugs in Mesothelioma Treatment

An orphan drug is medicine aimed at treating rare diseases and disorders in the public but does not get made through large pharmaceutical companies because of their rarity. In the almost 40 years the Office of the Orphan Product Development program has been around, the overall success of orphan drugs is positive. Orphan drugs for mesothelioma treatment could be the answer to finding an efficient treatment for those who are suffering from the disease.

Orphan drugs can receive designation after submitting a formal request to the FDA with data supporting their cause through the CDER NextGen portal. In the past year, mesothelioma has had one drug receive designation. Verismo Therapeutics’ SynKIR-110 is a form of immunotherapy that will target the solid mesothelioma tumor. Trials have begun in the beginning of 2023 to investigate the success of the drug for mesothelioma, mesothelin-expressing ovarian cancer, and cholangiocarcinoma cancer. If success is seen with this drug, late-stage mesothelioma patients could see their tumors shrink in size.

Challenges & Opportunities of Mesothelioma Orphan Drug Development

Developing a drug for a disease that doesn’t have many known cases can be difficult. There may not be many people for studies to take place, and if your clinical trial has specific requirements, it could become even more difficult. Many mesothelioma patients are diagnosed in stage 3 or 4 and the prevalence of painful symptoms may push them not to be able to participate. Plus, most people living with mesothelioma are older and may not be able to handle rigorous drug treatments.

Incentives for those who sponsor orphan drugs are available, too. Benefits such as tax credits for qualified clinical trials, exemption from user fees, or up to seven years of market exclusivity after approval are on the table. The FDA wants to support the funding of treatment drugs for many rare conditions but may not have all the fund money. The creation of the Office of Orphan Product Development (OOPD) allowed for the support of grant money. By working with other groups and organizations that focus on research and medical treatment of rare diseases, the OOPD has given a new light to those suffering from rare diseases.

Access to Orphan Drugs for Mesothelioma Patients

The importance of mesothelioma patients having access to life-changing orphan drugs is self-explanatory. People want to cure mesothelioma. There are a few barriers that are halting their access. The price of the drugs can often be expensive and aren’t affordable to the population the drug is trying to target. Insurance may not cover the costs of experimental drugs, causing patients to dig into their own pockets. The number of people diagnosed with mesothelioma isn’t necessarily increasing, but the demand for viable treatment is. Therefore, the orphan drugs that are working to find a cure are becoming harder to attain.

More research is needed to assess the state of orphan drugs and their accessibility accurately, but patient advocate groups are fighting for accessibility. Not only can patient advocates help you navigate treatment during your journey, but they can also help you retrieve financial compensation, emotional support, and answer any questions you may have surrounding your diagnosis.

The Future of Orphan Drugs in Mesothelioma Care

Research into finding successful treatments for rare diseases may only continue to grow. With the help of the FDA and other organizations looking to support finding a cure for rare conditions, more mesothelioma drugs may soon be coming to clinical trials. There is always room to improve and grow within mesothelioma care as eager medical professionals and specialist look to find better treatment options.

About 40 percent of all new medicines are coming from orphan drugs. Finding a treatment for mesothelioma is imperative since a lot of patients find out about their diagnosis when treatment options are minimal. If the orphan drug SynKIR-110 can show success, late-stage mesothelioma patients may now have a better option in treating their cancer. The benefits of these drugs may not be known for a while, but once they are, the impact may be huge. Getting involved in orphan drug trials may benefit those searching for new options. With the public’s continued support for the OOPD program, we could see more successful mesothelioma drug therapies coming soon. Ask us how we can help you, if you think an orphan drug treatment plan may be right for you.

Last updated on September 26th, 2023 at 11:04 pm

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