What is an Orphan Drug?
An "Orphan Drug" is a drug created for an obscure disease that affects such a small number of individuals residing in the United States that the disease or condition is considered rare. The Orphan Drug Act provides changes in applicable law to incentivize a pharmaceutical company to take on creating a drug that does not appear to have the potential to provide ROI (Return on Investment). The Act strives to ensure that potential drugs that might not otherwise be developed would be developed and made available to those that need them most.
My medications are delivered to my home while I am at work. They look almost melted when the mail order pharmacy delivers them. Is it safe to take them?
The question has become very important this season as record temperatures combined with a greater number of people relying on mail order and specialty pharmacies increases.
There is not evidence that the temporary exposure to heat causes the medication to be ineffective or unsafe. But the questions of "how hot is too hot?" and "how long is too long?" have not been answered. Almost all clinical trials involving medications require that the medication be maintained, stored and shipped in temperature controlled environments. Some exceptions are made for brief excursions when being shipped, moved, stored or dispensed. And it is these stringent clinical trials that our current knowledge of medication storage and shipping practices and recommendations is based.
There are pharmacists and clinical researchers at the major drug companies and mail order pharmacies who are attempting to answer the question now, but we don't have the necessary answers yet.
What can you do? You should submit a request in writing to the company that ships your medication, for the drug(s) to be sent in a protective container with a freezer pack or cold pack. If any medication shows signs of melting, sweating, cracking, discoloration or other physical change to the outside of the pill or capsule, you should not take the medication. If you have any concerns that the effectiveness of the medication you have taken has been compromised, please share those concerns with your hematologist, oncologist or CML specialist. They will be able to arrange more frequent testing to confidently determine if your condition has changed in any way.
Strategies you can use during warm weather months:
1. Have your medications delivered to your office or workplace, instead of to your empty front porch.
2. Request in writing that your mail order pharmacy pack the medication in a layered or insulated container or cooler, with the addition of a freezer pack or cold pack.
3. Request in writing that your mail order pharmacy send your shipment overnight to avoid long layovers in warmer climates.
4. If there is any delay in getting your medication on time, please contact your CML specialist, hematologist or oncologist so that they can help you make arrangements to obtain your drug on schedule. Skipping doses is NOT an option.
Can I have Pets?
You have leukemia, CML to be specific, and you also have wonderful pets that you would like to keep and protect. This is a great question and one faced by many individuals and families.
As you know, there are four primary types of leukemia: Chronic Myelogenous Leukemia (CML), Chronic Lymphocytic Leukemia (CLL), Acute Myelogenous Leukemia (AML) and Acute Lymphocytic Leukemia (ALL). CML behaves somewhat differently than CLL, AML and ALL. CML compromises your immune system's ability to protect you. In untreated CML, leukemic cells gradually replace healthy, mature cells and the result is that you become vulnerable to many infections.
When treated with tyrosine kinase inhibitors and other effective treatments, the leukemic cells are reduced and hopefully eliminated, leaving the environment available for the production of healthy cells which mature and are fully functional. Depending on your condition and phase of disease when diagnosed, your immune system will be compromised to different degrees. Once treatment begins, your immune system will be restored over time and protective functions will resume. When you achieve a deep response to treatment and maintain it over time, your immune system will function essentially like any person without CML.
If your CML progresses to accelerated or blast phase, your immune system will once again be in trouble and will not be able to protect your body as fully as it should.
So the answer to the question, "Can I have pets if I have CML?" is, "It depends." If you are in treatment for CML with a tyrosine kinase inhibitor or other effective treatment, and you have achieved and maintained a good response to that treatment, there are no contraindications to you having pets. However, if you are newly diagnosed, are transitioning treatments due to disease progression, or considering stem cell transplant please talk with your CML specialist about your pets. He or she will be able to tell you if your immune system is healthy enough to safely care for an live with pets.
There are several websites which cover precautions for individuals with compromised immune systems. Please make sure that the source of your information is credible and reliable.
Tips to Keep You and Your Pet Safe and Healthy General
- Research your pet, its breed and special needs.
- Keep all immunizations up to date and visit your veterinarian regularly.
- Keep animals as clean as possible.
- Consult your veterinarian should your pet experience coughing, sneezing, discharge from eyes, vomiting, diarrhea or weight loss.
- Keep nails trimmed to avoid scratches to your skin.
- Feed only commercially prepared pet food. This prevents your pet from contracting toxoplasmosis (cats) or other illnesses from eating undercooked food or wild animals.
- Prevent your pet from drinking water from the toilet.
- Use safe and effective flea and tick protection all year.
- Avoid contact with outdoor areas where animal droppings, urine or feces may be.
Keep your cat's litter box in a separate area from your living and eating space. Use gloves and a mask when changing litter and then dispose of same. Scoop litter daily. Always wash your hands after handling litter box.
Make sure testing for feline leukemia and feline immunodeficiency has been done. Although you can't "catch" these viruses, which may compromise your pet's immune system making them vulnerable to illnesses that might be harmful to you.
Keep nails trimmed to avoid scratches.
Minimize boarding or kennel visits as dogs can share "kennel cough" with their family.
Birds Wear gloves and a mask when cleaning cage or disturbing the contents of the cage. Take care when handling your bird to avoid being scratched. Reptiles Wear gloves when handling your reptiles and when cleaning their tanks and cages. People can be exposed to salmonella from reptiles representing a risk to patients.
Please remember to enjoy your pets too! They are faithful friends and members of our families. Having pets in your life can be an enjoyable and helpful therapy itself.
I have leftover medication because my doctor switched me to another drug. Can I give it to someone in need? What can I do?
This has been a challenge for our community for a long time. Currently, the NCMLS (and many other organizations) recommend that you take any unused CML medications (drugs such as Gleevec, Sprycel, Tasigna, Busulif, and Iclusig/Ponatinib) to your oncologist. Doing so provides the greatest opportunity for others to benefit from it if they cannot afford or have difficulty accessing their CML medication.
As you know, precription medications are "controlled substances" and therefore only intended for the person for whom they're precribed. Laws vary by state regarding possession of these medications, and while each State's law may be slightly different, they call for some strict penalties for those found to be abusing them by illegally distributing or transporting these drugs.
While we know that it happens and is probably done with the best of intentions, penalites can be quite severe. For example, according to the Drug Enforcement Agency of the United States charged and convicted violators can lose access to government programs such as Medicare and Medicaid. Depending upon the degree of violation, a lifetime ban can be imposed.
Use of the US Postal Service can increase any penalties as the violation then involves using a government service as a means of transport and distribution of a controlled substance if discovered. Shipping companies such as UPS or Fedex have strict rule on what can be shipped via their services. Please review their respective websites for specifs information on shipping policies.
When it comes to selling unused medications, one is subjected to even more risk as the violation moves from one of "wanting to help" and perhaps not knowing the regulations governing prescription drugs, to on of profiting from the sale of a substance that is regulated by a federal agency.
Giving your leftover CML medications to your oncologist not only eliminates the risks associated with distritribution of these drugs, it provides the greatest opportunity for the medication to be used to help someone who may otherwise not be able to afford or access treatment for their disease.
The National CML Society has contemplated ways in which to address this issue, including discussions with key CML physicians, pharaceutical companies, and regulatory agencies. Hopefully, one day we will have a viable, safe, and legal option for disposing of our unused CML medication.