COLORADO SPRINGS — Colorado House Minority Leader Rose Pugliese knows all too well the struggles and pain of raising a child with a rare genetic disorder.
The Republican, who represents House District 14 in El Paso County, is always hoping for a treatment to help with her daughter’s condition. So, when Pugliese met another woman who had two daughters also suffering from a rare condition — in this case a terminal one — and heard the mother’s tragic story, she decided it was time to act, introducing House Bill 25-1270, “Patients’ Right to Try Individualized Treatments,” which Governor Polis signed into law on Monday.
“She had two daughters who had a rare terminal disorder and the drug to treat it was not available through the FDA,” Pugliese said, adding that after one of the daughters died in hospice waiting for approval, the mother decided she was going to take her other daughter elsewhere for treatment she couldn’t get here. That daughter received treatment in Italy and is alive today.
The bill is based off of the federal “Right to Try” Act which allows patients who meet certain eligibility criteria to try investigational drugs that have also met certain criteria.
“I thought if we were able to allow doctors to prescribe some of these drugs, more patients would live,” she said. “And that is where my heart is.”
However, Pugliese’s bill takes the “right to try” concept one step further. Under the federal law, the investigational drug is one that is under review to treat a group of people. Under HB 1270 a Colorado patient can request from the drug manufacturer a “drug, biological product, or device that is unique to and produced exclusively for use by an individual patient based on the patient’s own genetic profile.”
Requirements under HB 1270 stipulate that the eligible patient has:
- A life-threatening or severely debilitating illness, as attested to by the patient’s treating physician;
- Considered all other treatment options currently approved by the United States food and drug administration;
- Received a recommendation from the patient’s treating physician;
- Given written, informed consent for the use of the individualized investigational drug, biological product, or device; and
- Documentation from the treating physician that the individual meets the definition of “eligible patient”.
The bill does not require that a manufacturer individualize a drug for a single patient, but allows one to do so, as well as to do so at no additional cost (though payment to cover the cost of manufacturing the drug can be charged).
The bill also exempts insurance companies from being required to pay for the drug.
Pugliese enlisted her long-time friend Sen. Barbara Kirkmeyer, R-Brighton to sponsor the legislation in the Senate.
Kirkmeyer said after hearing testimony from parents and other patients, that decision was easy.
“The testimony we heard in committee was heartbreaking,” Kirkmeyer said, “People with rare diseases face significant — often insurmountable hurdles — to access innovative individualize therapies. Hearing moms speak about all they go through to give their child an opportunity to live and then live a quality life, had everyone in tears.”
The bill, which passed out both chambers of the legislature unanimously, included a pair of Democrat sponsors as well: Rep. Lindsay Gilchrest (Denver) and Senator Lindsey Daugherty (Jefferson County).
“The Governor was very supportive of it,” Pugliese said, adding that his support is likely what pushed the bill through unanimously. “He actually wanted the bill to go further. I’m not sure what that would look like, but there may be a part two.”
